Pipeline Watchlist
Company Pipelines
Companies
62assets
- LutatheraAAA601 / Lutathera / SSTR / Radioligand therapy / Oncology: Solid TumorsGastroenteropancreatic neuroendocrine tumorsPhase 3Supplementary; filing 2028GEPNET, pediatricsPhase 2
- 177Lu-NeoBAAA603 / GRPR / Radioligand therapy / Oncology: Solid TumorsBreast cancerPhase 1Multiple solid tumorsPhase 2Lead; filing >= 2029
- AAA614FAP / Radioligand therapy / Oncology: Solid TumorsSolid tumorsPhase 2Lead
- PluvictoAAA617 / Pluvicto / PSMA / Radioligand therapy / Oncology: Solid TumorsOligometastatic prostate cancerPhase 3Supplementary; filing >= 2029Metastatic hormone sensitive prostate cancer (mHSPC)RegistrationSupplementary
- 225Ac-PSMA-617AAA817 / PSMA / Radioligand therapy / Oncology: Solid TumorsMetastatic castration-resistant prostate cancer (mCRPC)Phase 3Supplementary; filing >= 2029post Lu Metastatic castration-resistant prostate cancer (mCRPC)Phase 3Lead; filing 2028
- ScemblixABL001 / Scemblix / BCR-ABL / Inhibitor / Oncology: HematologyChronic myeloid leukemia, pediatricsPhase 2Supplementary; filing 2027
- CosentyxAIN457 / Cosentyx / IL17A / Inhibitor / ImmunologyPolymyalgia rheumaticaRegistrationSupplementary; filing 2026
- AimovigAMG334 / Aimovig / CGRPR / Antagonist / In-market Brands and Global HealthMigraine, pediatricsPhase 3Supplementary; filing 2028
- AMO959DNA PK / Inhibitor / Oncology: Solid TumorsProstate cancerPhase 1Lead
- MayzentBAF312 / Mayzent / S1P1,5 receptor modulator / NeuroscienceMultiple sclerosis, pediatricsPhase 3Supplementary; filing 2027
- VijoyceBYL719 / Vijoyce / PI3K-alpha / Inhibitor / Oncology: Solid TumorsLymphatic malformationsPhase 3Supplementary; filing >= 2029
- farabursenCYX082 / MIR17 / Inhibitor / Cardiovascular, Renal and MetabolicAutosomal dominant polycystic kidney diseasePhase 1Lead
- pelabresibDAK539 / BET / Inhibitor / Oncology: HematologyMyelofibrosisPhase 3Lead; filing 2026
- DFT383CTNS gene delivery / NeuroscienceCystinosisPhase 1Lead
- DII235siRNA targeting Lp(a) MRNA / Cardiovascular, Renal and MetabolicCVRR-Lp(a)Phase 2Lead; filing >= 2029
- del-braxDWH213 / siRNA DUX4 knockdown / NeuroscienceFacioscapulohumeral muscular dystrophyPhase 3Lead; filing 2028
- tulmimetostatDZR123 / EZH1, EZH2 / Inhibitor / Oncology: Solid TumorsMetastatic prostate cancerPhase 1Solid tumors & lymphomasPhase 2Lead
- ECI830CDK2 / Inhibitor / Oncology: Solid TumorsBreast cancerPhase 1Lead
- EDI048CpPI(4)K / Inhibitor / In-market Brands and Global HealthCryptosporidiosisPhase 2Lead
- EDK060Lead Indication / NeuroscienceCharcot-Marie-Tooth diseasePhase 1
- ESP359DLL3 / Radioligand therapy / Oncology: Solid TumorsSolid tumorsPhase 1Lead
- del-desiranEWF980 / siRNA DMPK knockdown / NeuroscienceMyotonic dystrophy type 1Phase 3Lead; filing 2027
- EYU688NS4B / Inhibitor / In-market Brands and Global HealthDengue feverPhase 2Lead
- zigakibartFUB523 / Anti-APRIL / Cardiovascular, Renal and MetabolicIgA nephropathyPhase 3Lead; filing 2027
- 177Lu-NNS309FXX489 / FAP / Radioligand therapy / Oncology: Solid TumorsSolid tumorsPhase 1Lead
- GCJ904Lead Indication / Oncology: Solid TumorsSolid tumorsPhase 1
- GHZ339Lead Indication / ImmunologyAtopic dermatitisPhase 2filing >= 2029
- GIA632IL-15 mAb / ImmunologyAtopic dermatitisPhase 2Lead; filing >= 2029VitiligoPhase 2Supplementary; filing >= 2029
- GXV813M4 / Agonist / NeuroscienceSchizophreniaPhase 2Lead
- HJB647Lead Indication / Cardiovascular, Renal and MetabolicHeart failurePhase 1
- votoplamHTT227 / Huntingtin modulator / NeuroscienceHuntington’s diseasePhase 3Lead; filing >= 2029
- INE963Plasmodium falciparum / Inhibitor / In-market Brands and Global HealthMalariaPhase 2Lead
- IPX643Lead Indication / ImmunologyInflammation-driven diseasesPhase 1
- ITU512HbF inducing agent / In-market Brands and Global HealthSickle cell diseasePhase 1Lead
- luxdegalutamideJSB462 / Androgen receptor protein degrader / Oncology: Solid TumorsMetastatic hormonal sensitive prostate cancerPhase 2Metastatic castration resistant prostate cancerPhase 2Lead; filing >= 2029
- cipargaminKAE609 / PfATP4 / Inhibitor / In-market Brands and Global HealthMalaria, uncomplicatedPhase 2Supplementary; filing >= 2029Malaria severePhase 2Lead; filing >= 2029
- LeqvioKJX839 / Leqvio / siRNA (regulation of LDL-C) / Cardiovascular, Renal and MetabolicCVRR (primary prevention)Phase 3Supplementary; filing >= 2029CVRR (secondary prevention)Phase 3Supplementary; filing 2027
- Ganaplacide + lumefantrineKLU156 / Non-artemisinin plasmodium falciparum / Inhibitor / In-market Brands and Global HealthMalaria, uncomplicatedPhase 3Lead; filing 2026
- del-zotaKPE179 / PMO mediated exon 44 skipping / NeuroscienceDuchenne muscular dystrophyPhase 2Lead; filing 2026
- FabhaltaLNP023 / Fabhalta / CFB / Inhibitor / Neuroscience / Cardiovascular, Renal and Metabolic / Oncology: Hematology / OthersGeneralized myasthenia gravisPhase 3Supplementary; filing 2027C3 glomerulopathy, pediatricsPhase 3IgAN, pediatricsPhase 3
- RhapsidoLOU064 / Rhapsido / BTK / Inhibitor / ImmunologyChronic spontaneous urticaria, pediatricsPhase 3Hidradenitis suppurativaPhase 3Supplementary; filing 2027Chronic inducible urticaria (CINDU)Phase 3Supplementary; filing 2026
- remibrutinibLOU064 / BTK / Inhibitor / NeuroscienceMultiple sclerosis, secondary progressivePhase 3Supplementary; filing >= 2029Myasthenia gravisPhase 3Supplementary; filing 2028Multiple sclerosis, relapsing remittingPhase 3Supplementary; filing 2027
- LTP001SMURF1 / Inhibitor / Cardiovascular, Renal and MetabolicIdiopathic pulmonary fibrosisPhase 2Pulmonary arterial hypertensionPhase 2Lead; filing >= 2029
- LXE408Proteasome / Inhibitor / In-market Brands and Global HealthChagasPhase 2Visceral leishmaniosisPhase 2Lead; filing >= 2029
- abelacimabMAA868 / FXI / Inhibitor / Cardiovascular, Renal and MetabolicAtrial fibrillationPhase 3Lead; filing 2028
- MAS825IL18 Inhibitor, IL1B / ImmunologyStill’s diseasePhase 2Lead
- NIO752Tau antisense oligonucleotide / NeuroscienceAlzheimer’s diseasePhase 1Progressive supranuclear palsyPhase 1Lead
- OJR520Lead Indication / Cardiovascular, Renal and MetabolicCKDPhase 1
- KesimptaOMB157 / Kesimpta / CD20 / Antagonist / NeuroscienceMultiple sclerosis, new dosing regimenPhase 3Supplementary; filing 2027Multiple sclerosis, pediatricsPhase 3Supplementary; filing 2026
- PAC001Anti-IL-6 mAb / Cardiovascular, Renal and Metabolic / OthersASCVD pacibekitugPhase 2Lead; filing >= 2029Thyroid eye disease (TED) pacibekitugPhase 2
- PIT565Anti-CD19, Anti-CD2, Anti-CD3 / ImmunologySystemic lupus erythematosusPhase 1Rheumatoid arthritisPhase 1
- RydaptPKC412 / Rydapt / Multi-targeted kinase / Inhibitor / In-market Brands and Global HealthAcute myeloid leukemia, pediatricsPhase 2Supplementary; filing 2027
- PKN605HDAC6 / Inhibitor / Cardiovascular, Renal and MetabolicAtrial FibrillationPhase 2Lead
- QCZ484Lead Indication / Cardiovascular, Renal and MetabolicHypertensionPhase 2filing >= 2029
- AtecturaQMF149 / Atectura / LABA + ICS / In-market Brands and Global HealthAsthma, pediatricsPhase 3Supplementary; filing >= 2029
- AdakveoSEG101 / Adakveo / P-selectin / Inhibitor / In-market Brands and Global HealthSickle cell disease, pediatricsPhase 3Supplementary; filing >= 2029
- TIN816ATP modulator / Cardiovascular, Renal and MetabolicAcute kidney injuryPhase 2Lead
- pelacarsenTQJ230 / ASO targeting Lp(a) / Cardiovascular, Renal and MetabolicCVRR (secondary prevention) in patients with elevated Lp(a)Phase 3Lead; filing 2026
- ianalumabVAY736 / BAFF-R inhibitor, ADCC-mediated B-cell depletor / Immunology / Oncology: HematologyLupus NephritisPhase 3Supplementary; filing 2028Systemic lupus erythematosus (SLE)Phase 3Supplementary; filing 2028Systemic sclerosisPhase 2Supplementary; filing 2028
- VHB937TREM2 stabilizer and activator / NeuroscienceAmyotrophic lateral sclerosisPhase 2Lead; filing >= 2029Alzheimer's diseasePhase 2Supplementary; filing >= 2029
- YMI024Lead Indication / Cardiovascular, Renal and MetabolicCVRRPhase 1
- rapcabtagene autoleucelYTB323 / CD19 CAR-T / Immunology / Neuroscience / Oncology: HematologyRheumatoid arthritis and severe, refractory Sjögren’s diseasePhase 1Systemic sclerosisPhase 2Supplementary; filing >= 2029MyositisPhase 2Supplementary; filing >= 2029
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- ABBV-8736ABBV-8736 / TREM1 / Biologic / ImmunologyCrohn's DiseasePhase 2
- TavapadonD1/D5 partial agonist / Small molecule / NeuroscienceParkinson's DiseaseSubmitted
- ABBV-525ABBV-525 / MALT1 / Small molecule / OncologyHematologic TumorsPhase 1
- Telisotuzumab vedotinABBV-399 / EMRELIS / c-Met / Antibody-drug conjugate / OncologyNon-Small Cell Lung CancerApprovedUS
- EpcoritamabGen3013 / EPKINLY/TEPKINLY / CD3, CD20 / Bispecific antibody / OncologyRelapsed/Refractory DLBCLApprovedUSEURelapsed/Refractory Follicular LymphomaApprovedRelapsed/Refractory CLLPhase 2
- ABBV-466 (Skyrizi + ABBV-382)ABBV-466 / SKYRIZI / IL-23 + anti-a4b7 mAb / Biologic combination / ImmunologyCrohn's DiseasePhase 2
- BudigalimabABBV-181 / PD1 / Biologic / OncologySolid and Hematologic TumorsPhase 2
- ABBV-701ABBV-701 / TL1A / Biologic / ImmunologyInflammatory Bowel DiseasePhase 1
- ABBV-932ABBV-932 / DRD2/DRD3 modulator / Small molecule / NeuroscienceBipolar DepressionPhase 2Generalized Anxiety DisorderPhase 2
- ABBV-9832 (Lutikizumab + Ravagalimab)ABBV-9832 / IL-1a/1b + CD40 antagonist / Biologic combination / ImmunologyRheumatoid ArthritisPhase 2
- ABBV-6628ABBV-6628 / C5 / Biologic / Eye CareGeographic AtrophyPhase 1
- ABBV-619ABBV-619 / CD19 / Biologic/CAR-T / ImmunologyAutoimmune diseasesPhase 1
- LivmoniplimabABBV-151 / GARP-TGF-beta1 / Biologic / OncologyHepatocellular CarcinomaPhase 2Solid TumorsPhase 2Non-Small Cell Lung CancerPhase 2
- Armour ThyroidArmour Thyroid / T3T4 / Biologic / Other SpecialtiesHypothyroidismPhase 3
- ABBV-319ABBV-319 / CD19 / Biologic ADC / ImmunologySjogren's diseasePhase 1Systemic Lupus ErythematosusPhase 1
- ABBV-722ABBV-722 / LPAR1 / Small molecule / ImmunologyIdiopathic Pulmonary FibrosisPhase 1Systemic SclerosisPhase 1
- ABBV-142ABBV-142 / LPAR1 / Biologic / ImmunologyIdiopathic Pulmonary FibrosisPhase 1
- ABBV-324ABBV-324 / Biologic ADC / OncologySolid TumorsPhase 1
- ABBV-295ABBV-295 / Long-Acting Amylin Analog / Large molecule / Other SpecialtiesObesityPhase 1
- SurzetoclaxABBV-453 / BCL-2 / Small molecule / OncologyRelapsed/Refractory Multiple MyelomaPhase 1
- ABBV-2002ABBV-2002 / PAR-2 / Biologic / NeuroscienceMigrainePhase 2
- IcalcaprantABBV-1354 / KOR antagonist / Small molecule / NeuroscienceBipolar DepressionPhase 2Major Depressive DisorderPhase 2
- ABBV-1042ABBV-1042 / M4 receptor / Small molecule / NeuroscienceBipolar ManiaPhase 1
- Surabgene LomparvovecABBV-RGX-314 / VEGF / Gene therapy / Eye Carewet AMD (subretinal)Phase 3wet AMD (suprachoroidal)Phase 2Diabetic Retinopathy (suprachoroidal)Phase 2
- UbrogepantUBRELVY / CGRP receptor / Small molecule / NeuroscienceMenstrual MigrainePhase 3
- ABBV-2001ABBV-2001 / BCMA, CD38, CD3 / Biologic trispecific T-cell engager / OncologyRelapsed/Refractory Multiple MyelomaPhase 1
- Pivekimab SunirinePVEK / CD123 / Biologic ADC / Oncology
- Mirvetuximab soravtansineELAHERE / FR-alpha / Biologic ADC / OncologyPlatinum-Resistant Ovarian CancerApprovedPlatinum-Sensitive Ovarian CancerPhase 3
- AtogepantQULIPTA / CGRP receptor / Small molecule / NeuroscienceAcute MigraineSubmittedOUSMenstrual MigrainePhase 3OUS
- EmraclidineM4 PAM / Small molecule / NeuroscienceSchizophreniaPhase 2PsychosisPhase 1
- UpadacitinibABT-494 / RINVOQ / JAK1 / Small molecule / ImmunologyAlopecia AreataSubmittedUSEUHidradenitis SuppurativaPhase 3Systemic Lupus ErythematosusPhase 3
- ABBV-166 (Skyrizi + Lutikizumab)ABBV-166 / SKYRIZI / IL-23 + IL-1a/1b / Biologic combination / ImmunologyPsoriatic ArthritisPhase 2
- ABBV-848ABBV-848 / IRAK4 / Small molecule / ImmunologyRheumatoid ArthritisPhase 1
- ABBV-706ABBV-706 / SEZ6 / Biologic ADC / OncologyFirst Line Small Cell Lung CancerPhase 2Relapsed/Refractory Small Cell Lung CancerPhase 3Neuroendocrine NeoplasmsPhase 1
- ABBV-1480ABBV-1480 / PD-1, VEGF / Biologic bispecific antibody / OncologySolid TumorsPhase 1
- LutikizumabABT-981 / IL-1a/1b / Biologic / ImmunologyHidradenitis SuppurativaPhase 3
- GemibotulinumtoxinAGemibotA / SNAP-25 / Biologic / NeuroscienceVentral Hernia RepairPhase 2
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- camizestrant + CDK4/6ingSERD + CDK4/6i / small molecule combination / Oncology1L HR+ HER2- ESR1m advanced breast cancerPhase IIISubmitted
- camizestrant + palbociclibngSERD + CDK4/6i / small molecule combination / Oncology1L HR+ HER2- advanced breast cancerPhase III
- camizestrant +/- abemaciclibngSERD + CDK4/6i / small molecule combination / Oncologyadjuvant HR+ HER2- early breast cancerPhase III
- camizestrantngSERD / small molecule / Oncologyadjuvant switch HR+ HER2- early breast cancerPhase III
- AZD0120AZD0120 / CD19/BCMA CAR-T / cell therapy / Oncology/Rare Disease/Respiratory & Immunologyrelapsed/refractory multiple myelomaPhase IIINew to Phase IIIamyloid light-chain amyloidosisPhase Isystemic lupus erythematosusPhase I
- puxitatug samrotecanB7-H4 TOP1i ADC / antibody-drug conjugate / Oncology2-3L B7-H4+ endometrial cancerPhase III
- rilvegostomigPD-1/TIGIT bispecific mAb / bispecific antibody / OncologyHCC; biliary tract; NSCLC; gastric cancerPhase III
- saruparibPARP1 inhibitor / small molecule / Oncologyprostate cancer; breast cancerPhase III
- sonesitatug vedotinCLDN18.2 MMAE ADC / antibody-drug conjugate / OncologyCLDN18.2+ gastric cancerPhase III
- surovatamigAZD0486 / CD19/CD3 T-cell engager / bispecific antibody / Oncology/Respiratory & Immunology1L elderly DLBCL; follicular lymphomaPhase IIIB-cell driven autoimmune diseasePhase I
- torvutatug samrotecanAZD5335 / anti-FRα TOP1i ADC / antibody-drug conjugate / Oncologyovarian cancerPhase III
- volrustomigPD-1/CTLA-4 bispecific mAb / bispecific antibody / Oncologycervical cancer; HNSCC; NSCLC; mesotheliomaPhase IIIcolorectal cancer; NSCLC; other solid tumoursPhase II
- balcinrenone/dapagliflozinMR antagonist/modulator + SGLT2 inhibitor / small molecule combination / Cardiovascular, Renal and Metabolismheart failure with CKDPhase IIICKDPhase II
- baxdrostataldosterone synthase inhibitor / small molecule / Cardiovascular, Renal and MetabolismhypertensionPhase IIIAcceptedprimary aldosteronismPhase III
- baxdrostat/dapagliflozinaldosterone synthase inhibitor + SGLT2 inhibitor / small molecule combination / Cardiovascular, Renal and MetabolismCKD; prevention of heart failurePhase III
- laroprovstatPCSK9 inhibitor / small molecule / Cardiovascular, Renal and MetabolismdyslipidaemiaPhase III
- WainuaWainua / ligand-conjugated antisense / antisense oligonucleotide / Cardiovascular, Renal and MetabolismATTRv-PNPhase IIILaunchedATTR CMLCM
- zibotentan/dapagliflozinendothelin A receptor antagonist/SGLT2 inhibitor / small molecule combination / Cardiovascular, Renal and MetabolismCKD with high proteinuriaPhase III
- elecoglipronoral GLP-1 receptor agonist / small molecule / Cardiovascular, Renal and MetabolismT2D/chronic weight managementPhase II
- AZD6234AZD6234 / peptide / peptide / Cardiovascular, Renal and Metabolismchronic weight management in overweight or obesityPhase II
- tozorakimabIL-33 mAb / monoclonal antibody / Respiratory & ImmunologyCOPDPhase IIIsevere viral lower respiratory tract diseasePhase IIIasthmaPhase II
- TezspireTezspire / TSLP mAb / monoclonal antibody / Respiratory & Immunologysevere uncontrolled asthmaPhase IIILaunchedChinaeosinophilic esophagitis; COPD; nasal polypsLCM
- SaphneloSaphnelo / type I IFN receptor mAb / monoclonal antibody / Respiratory & Immunologysystemic lupus erythematosusPhase IIILaunchedChinacutaneous lupus erythematosus; SLE SCLCM
- anselamimabfibril-reactive mAb / monoclonal antibody / Rare Diseaseamyloid light-chain amyloidosisPhase IIISubmitted
- cliramitugtransthyretin depleter / large molecule / Rare Diseasetransthyretin amyloid cardiomyopathyPhase III
- efzimfotase alfanext generation TNSALP ERT / enzyme replacement therapy / Rare DiseasehypophosphatasiaPhase III
- eneboparatideparathyroid hormone receptor 1 / large molecule / Rare DiseasehypoparathyroidismPhase III
- gefurulimabanti-C5 dual-binding nanobody / nanobody / Rare Diseasegeneralised myasthenia gravisPhase IIISubmitted
- KavigaleKavigale / SARS-CoV-2 LAAB / long-acting antibody / Infectious Diseaseprevention of COVID-19Phase IIILaunched
- AZD0292AZD0292 / pseudomonas Psl-PcrV bispecific mAb / bispecific antibody / Infectious DiseasebronchiectasisPhase II
- AZD5148AZD5148 / anti-Clostridioides difficile TcdB mAb / monoclonal antibody / Infectious Diseasereduction of C. diff recurrencePhase II
- AZD7760AZD7760 / S. aureus virulence factors / mAb combination / Infectious Diseaseprevention of Staph aureus infectionPhase II
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- erenumab-aooeAIMOVIG / CGRP receptor / Monoclonal antibody / NeurosciencePediatric migraineInvestigational
- blinatumomabBLINCYTO / CD19 x CD3 / BiTE molecule / Hematology/Oncology; InflammationNewly diagnosed B-cell precursor acute lymphoblastic leukemiaInvestigationalRelapsed/refractory acute lymphoblastic leukemia, subcutaneous administrationPhase 2InvestigationalSystemic lupus erythematosus with and without nephritisInvestigational
- dazodalibepCD40L / Fusion protein / Rare DiseaseSjögren’s diseaseInvestigational
- romosozumab-aqqgEVENITY / Sclerostin / Monoclonal antibody / BonePediatric osteogenesis imperfectaInvestigational
- tarlatamab-dlleIMDELLTRA / DLL3 x CD3 / BiTE molecule / Hematology/OncologySmall cell lung cancer, limited-stage and extensive-stage settingsInvestigationalSmall cell lung cancer, combination studiesInvestigationalSmall cell lung cancer, subcutaneous administrationPhase 1Investigational
- pegloticaseKRYSTEXXA / Uric acid / PEGylated uricase / Rare DiseaseUncontrolled gout, subcutaneous administration with methotrexateInvestigational
- sotorasibLUMAKRAS / KRAS G12C / Small molecule / Hematology/OncologyKRAS G12C-mutated metastatic colorectal cancer, first line combinationInvestigationalPreviously treated advanced KRAS G12C-mutated non-small cell lung cancerInvestigationalNewly diagnosed advanced KRAS G12C-mutated NSCLC, combinationInvestigational
- maridebart cafraglutideAMG 133 / MariTide / GLP-1 receptor; GIP receptor / Antibody-peptide conjugate / Cardiometabolic; DiabetesObesityInvestigationalCardiovascular risk reduction in ASCVD with obesity or overweightInvestigationalHeart failure events and cardiovascular risk reduction in HFpEF/HFmrEF with obesityInvestigational
- romiplostimNPLATE / Thrombopoietin receptor / Peptibody / Hematology/OncologyChemotherapy-induced thrombocytopeniaInvestigational
- olpasiranAMG 890 / Lp(a) / siRNA / CardiometabolicAtherosclerotic cardiovascular diseaseInvestigational
- apremilastOTEZLA / PDE4 / Small molecule / InflammationJuvenile psoriatic arthritisInvestigationalPediatric Behcet’s diseaseInvestigational
- etelcalcetidePARSABIV / Calcium-sensing receptor / Peptide / NephrologyPediatric secondary hyperparathyroidism in CKD receiving hemodialysisInvestigational
- evolocumabREPATHA / PCSK9 / Monoclonal antibody / CardiometabolicHypercholesterolemia in high cardiovascular risk without prior MI or strokeInvestigational
- avacopanTAVNEOS / C5aR1 / Small molecule / Rare DiseasePediatric ANCA-associated vasculitisInvestigational
- teprotumumab-trbwTEPEZZA / IGF-1R / Monoclonal antibody / Rare DiseaseThyroid eye disease, subcutaneous administrationInvestigational
- tezepelumab-ekkoTEZSPIRE / TSLP / Monoclonal antibody / InflammationChronic obstructive pulmonary diseaseInvestigationalEosinophilic esophagitisInvestigational
- xaluritamigAMG 509 / STEAP1 x CD3 / XmAb bispecific T-cell engager antibody / Hematology/OncologyProstate cancerInvestigationalEwing sarcomaInvestigational
- ABP 206ABP 206 / PD-1 / Monoclonal antibody biosimilar / Hematology/OncologyInvestigational biosimilar to OPDIVO (nivolumab)Investigational biosimilar
- ABP 234ABP 234 / PD-1 / Monoclonal antibody biosimilar / Hematology/OncologyInvestigational biosimilar to KEYTRUDA (pembrolizumab)Investigational biosimilar
- ABP 692ABP 692 / CD20 / Monoclonal antibody biosimilar / InflammationInvestigational biosimilar to OCREVUS (ocrelizumab)Investigational biosimilar
- daxdilimabILT7 / Monoclonal antibody / Rare DiseaseDermatomyositis and anti-synthetase inflammatory myositisInvestigationalDiscoid lupus erythematosusInvestigational
- inebilizumabUPLIZNA / CD19 / Monoclonal antibody / InflammationSystemic lupus erythematosus with nephritisInvestigational
- carfilzomibKYPROLIS / Proteasome / Small molecule / Hematology/OncologyPediatric acute lymphoblastic leukemiaInvestigational
- AMG 104AMG 104 / TSLP / Monoclonal antibody Fab / InflammationAsthmaInvestigational
- AMG 732AMG 732 / IGF-1R / Monoclonal antibody / Rare DiseaseThyroid eye diseaseInvestigational
- AMG 355AMG 355 / CCR8 / Monoclonal antibody / Hematology/OncologyAdvanced solid tumor malignanciesInvestigational
- AMG 410AMG 410 / Small molecule / Hematology/OncologySolid tumorsInvestigational
- AMG 436AMG 436 / Small molecule / Hematology/OncologyAdvanced solid tumor malignanciesInvestigational
- AMG 513AMG 513 / CardiometabolicObesityInvestigational
- AMG 691AMG 691 / Monoclonal antibody / InflammationAsthmaInvestigational
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- Glucose sensitive insulinNN1845 / Analogue / DiabetesType 1 and 2 diabetesPhase 1
- Ventus NLRP3iNN6022 / NLRP3 / Inhibitor / DiabetesCVDPhase 1
- siRNA GalXC-GYS2siRNA / DiabetessiRNA GalXC-GYS2Phase 1
- CNPCNP / Analogue / DiabetesHeart failurePhase 1
- Amylin 355Analogue / ObesityObesityPhase 1
- Amylin 1213Analogue / ObesityObesityPhase 1
- SLC25A5SLC25A5 / siRNA / ObesityMASHPhase 1
- Inno8Antibody fragment / Rare Blood DisordersHaemophiliaPhase 1
- Subcutaneous ZenagamtideGLP-1 / amylin / Co-agonist / Diabetes / ObesitySubcutaneous ZenagamtidePhase 2ObesityPhase 2
- Monlunabant in DKDCB1 receptor / Small molecule / DiabetesDiabetic Kidney Disease (DKD)Phase 2
- Oral ZenagamtideGLP-1 / amylin / Co-agonist / Diabetes / ObesityDiabetesPhase 2ObesityPhase 2
- CDR132LmiR-132 / Antisense oligonucleotide / DiabetesHeart failure with reduced ejection fraction (HFrEF)Phase 2
- UBT251 (GGG tri-agonist)ObesityObesityPhase 2
- TripleGLP1 / GIP / amylin / Tri-agonist / ObesityObesityPhase 2
- MonlunabantINV-202 / CB1 receptor / Small molecule / ObesityObesityPhase 2
- NDecEPI01 / Rare Blood DisordersSickle cell disease / An oral combination of decitabine and tetrahydrouridine. Project is developed in collaboration with EpiDestiny.Phase 2
- EtavopivatRare Blood DisordersThalassemiaPhase 2
- CagriSema in T2DNN9388 / Analogue / DiabetesType 2 diabetesPhase 3
- Ziltivekimab ASCVDNN6018 / DiabetesAtherosclerotic cardiovascular disease (ASCVD) with chronic kidney disease (CKD) and inflammationPhase 3
- CoramitugPRX004 / DiabetesATTR CardiomyopathyPhase 3
- Ziltivekimabin HFpEFDiabetesHeart failure with preserved ejection fraction (HFpEF)Phase 3
- Ziltivekimab AMIDiabetesAcute Myocardial infarction (AMI)Phase 3
- CagrilintideAnalogue / ObesityObesityPhase 3
- Efruxifermin in MASHFGF21 / Analogue / ObesityMASHPhase 3
- Etavopivat (Sickle Cell Disease)Small molecule / Rare Blood DisordersSickle Cell DiseasePhase 3
- IcosemaNN1535 / Analogue / DiabetesType 2 diabetes / A combination of GLP-1 analogue semaglutide and insulin icodec intended for once-weekly treatment.Filed
- Insulin icodecNN1436 / Analogue / DiabetesDiabetesFiled
- CagriSemaNN9898 / Analogue / ObesityObesityFiled
- Semaglutide 7.2 mgAnalogue / ObesityObesityFiled
- DenecimigMim8 / Inhibitor / Rare Blood DisordersHaemophilia A with or without inhibitors / A next generation FVIII mimetic bispecific antibody for subcutaneous prophylaxis of haemophilia A regardless of inhibitor status.Filed
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- ArexvyArexvy / RSV F protein / Recombinant protein, adjuvanted vaccine / Infectious diseasesRSV adults (50-59 years of age at increased risk)Phase III/Registration
- gepotidacinGSK2140944 / Bacterial topoisomerase inhibitor / Small molecule / Infectious diseasesUrogenital gonorrhoeaPhase III/Registration
- bepirovirsenGSK3228836 / HBsAg / Antisense oligonucleotide / Infectious diseasesChronic HBV infectionPhase III
- BexseroBexsero / MenB / Recombinant protein, OMV vaccine / Infectious diseasesMeningitis B (infants US)Phase III/RegistrationUS
- MenABCWY vaccineGSK3536819 / Meningococcal ABCWY / Recombinant protein, OMV, conjugated vaccine / Infectious diseasesMeningococcal diseasePhase III/Registration
- Nucala (mepolizumab)Nucala / IL-5 / Monoclonal antibody / Respiratory/ImmunologyCOPD with eosinophilic phenotypePhase III/Registration
- depemokimabGSK3511294 / IL-5 / Monoclonal antibody / Respiratory/ImmunologyAsthma with type 2 inflammationPhase III/RegistrationChronic rhinosinusitis with nasal polypsPhase III/RegistrationEGPAPhase III
- camlipixantGSK4523199 / P2X3 / Small molecule antagonist / Respiratory/ImmunologyRefractory chronic coughPhase III
- linerixibatGSK2330672 / IBAT / Small molecule / Respiratory/ImmunologyCholestatic pruritus in primary biliary cholangitisPhase III
- Blenrep (belantamab mafodotin)GSK2857916 / Blenrep / BCMA / Antibody-drug conjugate / OncologyRelapsed/refractory multiple myelomaPhase III/Registration
- Jemperli (dostarlimab)Jemperli / PD-1 / Monoclonal antibody / OncologyEndometrial cancerPhase III/RegistrationRectal cancer (dMMR)Phase IINon-small cell lung cancerPhase III
- Zejula (niraparib)Zejula / PARP / Small molecule / OncologyOvarian cancerPhase III/Registration
- belrestotugGSK4428859 / TIGIT / Monoclonal antibody / OncologyNSCLC and other solid tumorsPhase III
- GSK5764227GSK5764227 / B7-H3 / Antibody-drug conjugate / OncologyExtensive-stage small-cell lung cancerPhase IIIAdvanced GI solid tumorsPhase I/II
- cobolimabGSK4069889 / TIM-3 / Monoclonal antibody / OncologyNon-small cell lung cancerPhase II/III
- GSK4532990GSK4532990 / HSD17B13 / RNA interference / Opportunity drivenNASH/MASHPhase II
- GSK4172239GSK4172239 / DNMT1 / Small molecule inhibitor / Opportunity drivenSickle cell diseasePhase I
- GSK3882347GSK3882347 / FimH / Small molecule antagonist / Infectious diseasesUncomplicated UTIPhase I
- GSK3923868GSK3923868 / PI4K beta / Small molecule inhibitor / Infectious diseasesRhinovirus diseasePhase I
- GSK3965193GSK3965193 / PAPD5/PAPD7 / Small molecule inhibitor / Infectious diseasesChronic HBV infectionPhase I
- GSK2556286GSK2556286 / Mtb cholesterol-dependent / Small molecule inhibitor / Infectious diseasesTuberculosisPhase I
- GSK3772701GSK3772701 / P. falciparum whole cell / Small molecule inhibitor / Infectious diseasesMalariaPhase I
- GSK4024484GSK4024484 / P. falciparum whole cell / Small molecule inhibitor / Infectious diseasesMalariaPhase I
- GSK3536867GSK3536867 / Salmonella typhoid + paratyphoid A / Bivalent conjugate vaccine / Infectious diseasesSalmonella (typhoid + paratyphoid A)Phase I
- AGN-151586 / progranulin program (latozinemab)AL001 / Progranulin/sortilin / Monoclonal antibody / Opportunity drivenFrontotemporal dementia with progranulin gene mutationPhase III
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- ZanubrutinibBTK / Inhibitor / Hematologic Malignancies / Immunology & InflammationTreatment-Naive Chronic Lymphocytic Leukemia / Small Lymphocytic LymphomaApprovedRelapsed/Refractory Chronic Lymphocytic Leukemia / Small Lymphocytic LymphomaApprovedRelapsed/Refractory Waldenström Macroglobulinemia and Treatment-Naïve Waldenström MacroglobulinemiaApproved
- SonrotoclaxBCL2 / Inhibitor / Hematologic MalignanciesTreatment-Naive Chronic Lymphocytic LeukemiaPhase 3Treatment-Naive Chronic Lymphocytic LeukemiaPhase 3Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic LymphomaApproved
- TislelizumabPD-1 / Monoclonal Antibody / Lung Cancers / Gastrointestinal Cancers / Other CancersFirst-Line Extensive-Stage Small Cell Lung CancerApprovedFirst-Line Non-Squamous Non-Small Cell Lung CancerApprovedFirst-Line Squamous Non-Small Cell Lung CancerApproved
- BGB-16673BTK Chimeric Degradation Activation Compound [CDAC] / Hematologic Malignancies / Immunology & InflammationRelapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic LymphomaPhase 3Relapsed/Refractory Chronic Lymphocytic Leukemia/Small Lymphocytic LymphomaPhase 3Relapsed/Refractory Mantle Cell LymphomaPhase 2
- BlinatumomabCD19 x CD3 / BiTE® [Bispecific T-Cell Engager] / Hematologic MalignanciesRelapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (subcutaneous formulation)Phase 2
- TarlatamabDLL3 x CD3 / BiTE® [Bispecific T-cell Engager] / Lung CancersFirst-Line Extensive-Stage Small Cell Lung CancerPhase 3First-Line Extensive-Stage Small Cell Lung CancerPhase 3Second-Line Small Cell Lung CancerPhase 3
- ZanidatamabHER2 Bispecific / Antibody / Gastrointestinal CancersFirst-Line HER2 Positive Gastroesophageal AdenocarcinomaPhase 3Second-Line HER2 Positive Biliary Tract CancerApprovedFirst-Line HER2 positive Biliary Tract CancerPhase 3
- BG-68501CDK2 / Inhibitor / Breast/Gynecologic CancersBreast Cancer and Solid TumorsPhase 1
- BGB-21447BCL2 / Inhibitor / Breast/Gynecologic CancersHormone Receptor Positive Breast CancerPhase 1
- BGB-43395CDK4 / Inhibitor / Breast/Gynecologic CancersBreast Cancer and Solid TumorsPhase 1
- BGB-B455Claudin 6 x CD3 Bispecific / Antibody / Breast/Gynecologic CancersGynecologic and Solid TumorsPhase 1
- BG-C9074B7-H4 Antibody Drug Conjugate / Breast/Gynecologic CancersBreast Cancer and Solid TumorsPhase 1
- BG-75202KAT6A/B / Inhibitor / Breast/Gynecologic CancersBreast CancerPhase 1
- BG-75098CDK2 Chimeric Degradation Activation Compound [CDAC] / Breast/Gynecologic CancersBreast CancerPhase 1
- BGB-B2033GPC3 x 4-1BB Bispecific / Antibody / Gastrointestinal CancersSolid TumorsPhase 1
- BG-C137FGFR2b Antibody Drug Conjugate / Gastrointestinal CancersGastrointestinal CancersPhase 1
- BGB-53038Pan-KRAS / Inhibitor / Gastrointestinal CancersLung Cancers and GI CancersPhase 1
- BG-C477CEA Antibody Drug Conjugate / Gastrointestinal CancersLung Cancers and GI CancersPhase 1
- BG-C0902EGFR x MET x MET Antibody Drug Conjugate / Lung CancersLung Cancers and GI CancersPhase 1
- BG-T187EGFR x MET x MET Trispecific / Antibody / Lung CancersLung Cancers and GI CancersPhase 1
- BGB-58067MTA Cooperative PRMT5 / Inhibitor / Lung CancersLung Cancers and GI CancersPhase 1
- BG-89894MAT2A / Inhibitor / Lung CancersLung Cancers and Solid TumorsPhase 1
- BG-60366EGFR Chimeric Degradation Activation Compound [CDAC] / Lung CancersLung CancersPhase 1
- BGB-26808HPK1 / Inhibitor / Gastrointestinal CancersSolid Tumors/Pan- TumorPhase 1
- XaluritamigSTEAP1 x CD3 Bispecific T-cell Engager (XmAb®) / Other CancersMetastatic Castration-Resistant Prostate CancerPhase 1
- BGB-45035IRAK4 Chimeric Degradation Activation Compound [CDAC] / Immunology & InflammationRheumatoid ArthritisPhase 2Skin DiseasesPhase 1
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- NucresiranRNAi therapeutic / Transthyretin Amyloidosis (TTR)ATTR Amyloidosis with CardiomyopathyPhase 3hATTR Amyloidosis with PolyneuropathyPhase 3
- ZilebesiranRNAi therapeutic / CardiovascularHypertensionPhase 3REVERSIR HypertensionPhase 1
- RapirosiranALN-HSD / HSD / RNAi therapeutic / MetabolicMetabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 2
- ALN-ANG3ALN-ANG3 / ANG3 / RNAi therapeutic / MetabolicDiabetic Kidney DiseasePhase 2
- ALN-4324ALN-4324 / GRB14 / RNAi therapeutic / MetabolicType 2 Diabetes MellitusPhase 1
- ALN-2232ALN-2232 / ACVR1C / RNAi therapeutic / MetabolicObesity & Weight ManagementPhase 1
- ALN-PNPALN-PNP / PNP / RNAi therapeutic / MetabolicNon-Alcoholic Fatty Liver Disease (NAFLD)Phase 1
- ALN-APOC3ALN-APOC3 / APOC3 / RNAi therapeutic / MetabolicDyslipidemiaPhase 1
- ALN-CIDEBALN-CIDEB / CIDEB / RNAi therapeutic / MetabolicMetabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 1
- Cemdisiran(+/- Pozelimab)RNAi therapeutic / Neuroscience/OtherMyasthenia GravisPhase 3Geographic AtrophyPhase 3
- MivelsiranALN-APP / APP / RNAi therapeutic / NeuroscienceCerebral Amyloid Angiopathy (CAA)Phase 2Alzheimer’s DiseasePhase 1
- ALN-HTT02ALN-HTT02 / HTT / RNAi therapeutic / NeuroscienceHuntington’s DiseasePhase 1
- ALN-5288ALN-5288 / MAPT / RNAi therapeutic / NeuroscienceAlzheimer’s DiseasePhase 1
- ALN-SODALN-SOD / SOD1 / RNAi therapeutic / NeuroscienceSOD1 Amyotrophic Lateral Sclerosis (ALS)Phase 1
- ALN-SNCAALN-SNCA / SNCA / RNAi therapeutic / NeuroscienceParkinson’s DiseasePhase 1
- Cemdisiran (+Pozelimab)RNAi therapeutic / HematologyParoxysmal Nocturnal HemoglobinuriaPhase 3
- ALN-6400ALN-6400 / PLG / RNAi therapeutic / HematologyBleeding DisordersPhase 2
- AG-236ALN-TMP / TMP / RNAi therapeutic / HematologyPolycythemia VeraPhase 1
- ALN-CFBALN-CFB / CFB / RNAi therapeutic / HematologyParoxysmal Nocturnal HemoglobinuriaPhase 1
- Elebsiran (+Tobevibart)RNAi therapeutic / OtherHepatitis D Virus InfectionPhase 2
- ALN-BCATALN-BCAT / BCAT / RNAi therapeutic / OtherHepatocellular CarcinomaPhase 1
- ALN-4285ALN-4285 / RNAi therapeutic / OtherHealthy VolunteersPhase 1
- ALN-4915ALN-4915 / RNAi therapeutic / OtherHealthy VolunteersPhase 1
- ALN-F1202ALN-F1202 / RNAi therapeutic / OtherHealthy VolunteersPhase 1
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- PumitamigPD-L1/VEGF-A / immunomodulator / OncologyMultiple solid tumors; NSCLC; SCLC; HCC; CRC; ES-SCLC; EGFRm NSCLC; glioblastoma; MPM; NEN; TNBC; PDACclinical
- BNT325/DB-1305BNT325/DB-1305 / targeted therapy / OncologyMultiple solid tumorsclinical
- BNT324/DB-1311BNT324/DB-1311 / targeted therapy / OncologyMultiple solid tumorsclinical
- BNT321BNT321 / targeted therapy / OncologyMultiple solid tumors; HCC combinationclinical
- Autogene cevumeranmRNA cancer immunotherapy / OncologyAdjuvant CRC; adjuvant PDACclinical
- BNT116BNT116 / mRNA cancer immunotherapy / OncologyAdvanced NSCLC; 1L advanced NSCLCclinical
- BNT113BNT113 / HPV16 / mRNA cancer immunotherapy / Oncology1L HPV16+ HNSCCclinical
- GotistobartCTLA-4 / immunomodulator / OncologyMetastatic NSCLC; metastatic CRPC; PROC; multiple solid tumorsclinical
- BNT211BNT211 / cell therapy / OncologyMultiple solid tumorsclinical
- BNT314/GEN1059BNT314/GEN1059 / immunomodulator / OncologyMultiple solid tumors; metastatic CRC combinationclinical
- BNT329BNT329 / immunomodulator / OncologyMultiple solid tumorsclinical
- BNT326/YL202BNT326/YL202 / targeted therapy / OncologyMultiple solid tumors; NSCLC combination; advanced/metastatic breast cancerclinical
- BNT317BNT317 / immunomodulator / OncologyMultiple solid tumorsclinical
- Trastuzumab pamirtecanHER2 / targeted therapy / OncologyMetastatic breast cancer; advanced/metastatic breast cancer combination; 2L endometrial cancer; multiple solid tumorsclinical
- BNT162 + BNT161BNT162 + BNT161 / mRNA vaccine combination / Infectious diseasesCOVID-19-influenza combinationclinical
- BNT163BNT163 / HSV / mRNA vaccine / Infectious diseasesHSVclinical
- BNT166BNT166 / Mpox / mRNA vaccine / Infectious diseasesMpoxclinical
- BNT162BNT162 / COMIRNATY / SARS-CoV-2 / mRNA vaccine / Infectious diseasesCOVID-19Commercialmarketed
- BNT351BNT351 / HIV / antibody mRNA / Infectious diseasesHIVclinical
- BNT164BNT164 / Tuberculosis / mRNA vaccine / Infectious diseasesTuberculosisclinical
- BNT165BNT165 / Malaria / mRNA vaccine / Infectious diseasesMalariaclinical
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- LenacapavirHIV capsid inhibitor / Long-acting injectable / VirologyHIV Prevention (PrEP, PURPOSE 365)Phase 3HIV Treatment (in combinations)Phase 3HIV Treatment (with bNAbs)Phase 2
- Bictegravir/lenacapavir oral combinationINSTI + capsid inhibitor / Oral / VirologyHIV Treatment (virologically suppressed, ARTISTRY-1 & ARTISTRY-2)Phase 3
- Islatravir/lenacapavir oral combinationLong-acting oral / VirologyHIV Treatment (virologically suppressed, ISLEND-1 & ISLEND-2)Phase 3
- Lenacapavir + teropavimab + zinlirvimabCapsid + bNAbs / Long-acting injectable / VirologyHIV Treatment (virologically suppressed)Phase 2
- GS-1720/GS-4182GS-1720/GS-4182 / INSTI/capsid inhibitor / Long-acting oral / VirologyHIV Treatment (WONDERS-1 & -2)Phase 2
- Teropavimab + zinlirvimabbNAbs / Broadly neutralizing antibodies / VirologyHIV CurePhase 2
- VesatolimodVirologyHIV Cure (FRESH)Phase 2
- SelgantolimodVirologyHBV CurePhase 2
- HepcludexHepcludex / VirologyChronic HDV with compensated liver disease (MYR301)FiledBLA pending (US); Approved (EU)USEU
- EdecesertibInflammationLupus (COSMIC)Phase 2
- Tilpisertib fosmecarbilInflammationInflammatory bowel disease (PALEKONA)Phase 2
- α4β7 inhibitorα4β7 / InflammationInflammatory Bowel Disease (SWIFT)Phase 2
- Sacituzumab govitecan-hziyAntibody-drug conjugate / Oncology1L metastatic triple-negative breast cancer (PD-L1-, ASCENT-03)FiledsBLA pendingUS1L metastatic triple-negative breast cancer (PD-L1+, with pembrolizumab, ASCENT-04)FiledsBLA pendingUSHigh risk adjuvant triple-negative breast cancer (with pembrolizumab, ASCENT-05)Phase 3
- Axicabtagene ciloleucelCD19 CAR / Cell therapy / Oncology2L+ high-risk follicular lymphoma (ZUMA-22)Phase 31L high-risk large B-cell lymphoma (ZUMA-23)Phase 3
- Brexucabtagene autoleucelCD19 CAR / Cell therapy / OncologyPediatric acute lymphocytic leukemia/non-Hodgkin lymphoma (ZUMA-4)Phase 2
- Anitocabtagene autoleucelCell therapy / Oncology2-4L relapsed/refractory multiple myeloma (iMMagine-3)Phase 34L+ relapsed/refractory multiple myeloma (iMMagine-1)FiledBLA pendingUS
- KITE-363KITE-363 / CD19/CD20 bicistronic / Cell therapy / OncologyRelapsed/refractory diffuse large B-cell lymphomaPhase 1RMAT designationRheumatology (autoimmune)Phase 1
- DenikitugGS-1811 / OncologyAdvanced cancersPhase 1
- Domvanalimab + zimberelimabAnti-TIGIT + anti-PD1 / Combination therapy / Oncology1L metastatic non-small cell lung cancer (with chemo, STAR-121)Phase 31L head and neck squamous cell carcinoma (VELOCITY-HNSCC)Phase 2
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- ZilganersenION373 / GFAP / RNA-Targeted Therapeutic / NeurologyAlexander DiseasePhase 1/3ActiveGlobal
- ION582ION582 / UBE3A-ATS / RNA-Targeted Therapeutic / NeurologyAngelman SyndromeClinicalActive
- ION269ION269 / APP / RNA-Targeted Therapeutic / NeurologyAlzheimer's DiseaseClinicalActive
- ION717ION717 / PRNP / RNA-Targeted Therapeutic / NeurologyPrion DiseaseClinicalActive
- ION356ION356 / PLP1 / RNA-Targeted Therapeutic / NeurologyPelizaeus-Merzbacher DiseaseClinicalActive
- ION859ION859 / LRRK2 / RNA-Targeted Therapeutic / NeurologyParkinson's DiseaseClinicalActive
- ION464ION464 / SNCA / RNA-Targeted Therapeutic / NeurologyMultiple System AtrophyClinicalActiveParkinson's DiseaseClinicalActive
- ION440ION440 / MECP2 / RNA-Targeted Therapeutic / NeurologyMECP2 Duplication SyndromeClinicalActive
- OlezarsenApoC-III / RNA-Targeted Therapeutic / CardiologySevere HypertriglyceridemiaClinicalActive
- DonidalorsenPKK / RNA-Targeted Therapeutic / Specialty/RareHereditary AngioedemaClinicalActive
- SapablursenTMPRSS6 / RNA-Targeted Therapeutic / Specialty/RarePolycythemia VeraClinicalActive
- UlefnersenFUS / RNA-Targeted Therapeutic / NeurologyAmyotrophic Lateral SclerosisClinicalActive
- TofersenSOD1 / RNA-Targeted Therapeutic / NeurologyAmyotrophic Lateral Sclerosis (Presymptomatic SOD1)ClinicalActive
- IONIS-MAPTRxBIIB080 / TAU / RNA-Targeted Therapeutic / NeurologyAlzheimer's DiseaseClinicalActive
- TominersenIONIS-HTTRx / HTT / RNA-Targeted Therapeutic / NeurologyHuntington's DiseasePhase 2Active
- ION306ION306 / SMN2 / RNA-Targeted Therapeutic / NeurologySpinal Muscular AtrophyClinicalActive
- EplontersenTTR / RNA-Targeted Therapeutic / CardiologyTransthyretin-Mediated Amyloid CardiomyopathyPhase 3Active
- ION904ION904 / Angiotensinogen / RNA-Targeted Therapeutic / CardiologyTreatment-Resistant HypertensionClinicalActive
- BepirovirsenHepatitis B Virus / RNA-Targeted Therapeutic / Other MedicinesHepatitis B Virus InfectionPhase 3Active
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- zolbetuximabIMAB362 / VYLOY / Claudin 18.2 / Antibody / OncologyGastric and gastroesophageal junction adenocarcinoma, combo with pembrolizumab and chemotherapyFiledEuropeJapan
- gilteritinibASP2215 / XOSPATA / FLT3 / Small molecule / OncologyNewly diagnosed acute myeloid leukemia with high intensity induction chemotherapyPhase 3Post-chemotherapy maintenance acute myeloid leukemiaPhase 3Post-hematopoietic stem cell transplant maintenance acute myeloid leukemiaPhase 3
- enfortumab vedotinASG-22ME / PADCEV / Nectin-4 / Antibody-drug conjugate (ADC) / OncologyCisplatin-eligible muscle-invasive bladder cancer, combo with pembrolizumabFiledEuropeUSCisplatin-ineligible muscle-invasive bladder cancer, combo with pembrolizumabFiledEuropeJapanBladder-sparing muscle-invasive bladder cancer, combo with pembrolizumabPhase 2
- fezolinetantESN364 / VEOZAH / NK3 receptor / Small molecule / Women's HealthVasomotor symptoms due to menopausePhase 3ChinaVasomotor symptoms in breast cancer patients on adjuvant endocrine therapyPhase 2
- zocaglusagene nuzaparvovecAT845 / GAA gene replacement / Gene therapy (AAV-based gene therapy) / Genetic RegulationPompe diseasePhase 1
- ASP2957ASP2957 / MTM1 gene replacement / Gene therapy (AAV-based gene therapy) / Genetic RegulationX-linked myotubular myopathyPhase 1
- setidegrasibKRAS G12D / Small molecule degrader / Targeted Protein DegradationPancreatic ductal adenocarcinomaPhase 3
- ASP7317ASP7317 / Retinal pigment epithelial cells / Cell therapy / OphthalmologyGeographic atrophy secondary to age-related macular degenerationPhase 1
- ASP1002ASP1002 / Claudin 4/CD137 / T-cell engager bispecific antibody / OncologyGastric and gastroesophageal junction adenocarcinoma; pancreatic adenocarcinomaPhase 1
- ASP5834ASP5834 / Pan-KRAS / Small molecule degrader / OncologyCancerPhase 1
- VIR-5500VIR-5500 / PSMA/CD3 / T-cell engager / OncologyProstate cancerPhase 1
- ASP2998ASP2998 / TROP2 / Antibody-drug conjugate (ADC) / OncologyCancerPhase 1
- ASP2138ASP2138 / Claudin 18.2/CD3 / T-cell engager bispecific antibody / OncologyCancerPhase 2
- ASP546CASP546C / Claudin 18.2 / Antibody-drug conjugate (ADC) / OncologyCancerPhase 2Europe
- abiraterone decanoateASP5541/PRL-02 / CYP17 lyase / Small molecule / OncologyProstate cancerPhase 2
- mirabegronYM178 / Beta-3 receptor / Small molecule / UrologyNeurogenic detrusor overactivity in pediatric patients aged 6 months to less than 3 yearsPhase 3Europe
- roxadustatASP1517/FG-4592 / HIF-PH / Small molecule / NephrologyAnemia associated with chronic kidney disease in pediatric patientsPhase 3Europe
- pudexacianinium chlorideASP5354 / Drug-device combination / OtherIntraoperative ureter visualization for minimally invasive and open abdominopelvic surgeriesPhase 3
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- Mesalamine DRAsacol HD / generic small molecule / Gastroenterology
- EverolimusAfinitor / mTOR / generic small molecule / Oncology
- Prednisolone acetatePred-Forte / generic small molecule / Ophthalmology
- EltrombopagPromacta / TPO receptor agonist / generic small molecule / Hematology
- EpinephrineAdrenalin / adrenergic receptors / generic small molecule / Emergency/Critical Care
- LenalidomideRevlimid / generic small molecule / Oncology
- Risperidone ERRisperdal Consta / dopamine/serotonin receptors / generic long-acting injectable / Psychiatry
- Romidepsin injectionRomidepsin / HDAC / generic small molecule / Oncology
- CyclosporineRestasis / calcineurin / generic small molecule / Ophthalmology
- Beclomethasone dipropionateQVAR / glucocorticoid receptor / generic inhaled corticosteroid / Respiratory
- Sodium BicarbonateNeut / generic injectable / Critical Care
- Lanreotide injectionSomatuline Depot / somatostatin receptors / generic peptide injectable / Endocrinology/Oncology
- RifaximinXifaxan / generic small molecule / Gastroenterology
- Omalizumab biosimilarXOLAIR / IgE / biosimilar monoclonal antibody / Immunology/Allergy
- Sodium oxybateXyrem / generic small molecule / Neurology
- BimatoprostLumigan / prostaglandin receptor / generic small molecule / Ophthalmology
- Denosumab biosimilarsPROLIA & XGEVA / RANKL / biosimilar monoclonal antibody / Osteoporosis/Bone Cancer
- Albuterol sulfateProAir HFA / beta-2 adrenergic receptor / generic inhaled small molecule / Respiratory
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- PlozasiranARO-APOC3 / REDEMPLO / APOC3 / RNAi therapeutic / CardiometabolicFamilial chylomicronemia syndromeIn MarketSevere hypertriglyceridemiaPhase 3ActiveHypertriglyceridemiaPhase 3Active
- ZodasiranARO-ANG3 / ANGPTL3 / RNAi therapeutic / CardiometabolicHomozygous familial hypercholesterolemiaPhase 3Recruiting
- OlpasiranAMG 890; ARO-LPA / LPA / RNAi therapeutic / Cardiovascular diseaseElevated lipoprotein(a)/cardiovascular riskPhase 3Recruiting/Active
- GSK4532990ARO-HSD / HSD17B13 / RNAi therapeutic / Liver diseaseMASH/NASHPhase 2Recruiting
- ARO-PNPLA3JNJ-75220795 / PNPLA3 / RNAi therapeutic / Liver diseaseMASH/NASHPhase 1
- ARO-INHBEINHBE/Activin E / RNAi therapeutic / Obesity/metabolic diseaseObesity with or without type 2 diabetesPhase 1
- ARO-ALK7ACVR1C/ALK7 / RNAi therapeutic / Obesity/metabolic diseaseObesity with or without type 2 diabetesPhase 1Recruiting
- ARO-MAPTMAPT/tau / RNAi therapeutic / CNSAlzheimer's diseasePhase 1Recruiting
- ARO-ATXN2ATXN2 / RNAi therapeutic / CNSSpinocerebellar ataxia 2Phase 1
- SRP-1005ARO-HTT / HTT / siRNA therapeutic / CNSHuntington's diseasePhase 1
- ARO-RAGERAGE / RNAi therapeutic / PulmonaryInflammatory pulmonary diseasesPhase 2Active
- ARO-MMP7MMP7 / RNAi therapeutic / PulmonaryIdiopathic pulmonary fibrosisPhase 1
- FazirsiranTAK-999; ARO-AAT / Z-AAT / RNAi therapeutic / Liver diseaseAlpha-1 antitrypsin deficiency-associated liver diseasePhase 3Recruiting
- ARO-HBVJNJ-3989; JNJ-73763989 / HBV gene products / RNAi therapeutic / Infectious diseaseChronic hepatitis BPhase 2
- ARO-DUX4DUX4 / RNAi therapeutic / Muscular diseaseFacioscapulohumeral muscular dystrophyPhase 2
- ARO-DM1DMPK mRNA / RNAi therapeutic / Muscular diseaseMyotonic dystrophy type 1Phase 2Recruiting
- ARO-C3Complement component 3 / RNAi therapeutic / Complement mediated diseaseComplement-mediated renal diseasePhase 1RecruitingC3 glomerulopathyPhase 1RecruitingIgA nephropathyPhase 1Recruiting
- ARO-CFBComplement factor B / RNAi therapeutic / Complement mediated diseaseComplement mediated diseasePhase 1
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- Vabysmofaricimab-svoa / VABYSMO / VEGF/Ang-2 / Bispecific antibody / OphthalmologyNeovascular age-related macular degeneration (nAMD) and Diabetic macular edema (DME)Approvedapproved
- Ojemdatovorafenib / OJEMDA / OncologyOncology (undisclosed approved indication)Approvedapproved
- Miplyffaarimoclomol / MIPLYFFA / Rare Diseases / NeurologyNiemann-Pick disease type C (NPC)ApprovedapprovedUS
- Xaciatoclindamycin phosphate vaginal gel 2% / XACIATO / Small molecule / Gynecology / Infectious DiseaseBacterial VaginosisApprovedapproved
- Ixinitycoagulation factor IX (recombinant) / IXINITY / Factor IX / Recombinant protein / HematologyHemophilia BApprovedapproved
- Dsuviasufentanil sublingual tablet / DSUVIA / Opioid receptor / Small molecule / Pain ManagementPainApprovedapproved
- Dare to Playsildenafil cream / DARE to PLAY / PDE5 / Small molecule / Gynecology / Sexual HealthFemale Sexual Arousal DisorderApprovedapproved
- ersodetugRZ358 / INSR / Antibody / Metabolic DisordersHypoglycemia due to congenital hyperinsulinism (cHI)Phase 3ongoingHypoglycemia due to tumor hyperinsulinismPhase 3ongoing
- RZ402RZ402 / OphthalmologyDiabetic macular edema (DME)Phase 2ongoing
- D-FiFCX-007 / Gene Therapy / Dermatology / Rare DiseasesDystrophic epidermolysis bullosa (DEB)Phase 3ongoing
- seralutinibGB002 / PDGFR, CSF1R, and c-KIT / Inhaled tyrosine kinase inhibitor / Respiratory / CardiovascularPulmonary arterial hypertension (PAH)Phase 3ongoingPulmonary Hypertension Associated With Interstitial Lung Disease (PH-ILD)Phase 2ongoing
- cetrelimabJNJ-63723283 / PD-1 / Antibody / Oncology / Infectious DiseaseMuscle-invasive bladder cancer (MIBC)Phase 2ongoingChronic Hepatitis B Virus InfectionPhase 2ongoing
- volixibatLUM-002 / Ileal bile acid transporter (IBAT) / Small molecule / GastroenterologyPrimary sclerosing cholangitis (PSC)Phase 2bongoingPrimary biliary cholangitis (PBC)Phase 2ongoing
- osavampatorNeurology / PsychiatryMajor depressive disorderPhase 3ongoing
- OHB-607OHB-607 / Recombinant human IGF-I/IGFBP-3 / Recombinant protein / Respiratory / NeonatologyPrevention of bronchopulmonary dysplasia in extremely premature infantsPhase 2ongoing
- REC-4881REC-4881 / Allosteric MEK1/2 / Small molecule / OncologyFamilial adenomatous polyposisPhase 2ongoing
- NIS793NIS793 / Antibody / OncologySolid tumorsPhase 3ongoing
- QTORIN rapamycinQTORIN / mTOR / Small molecule / Dermatology / Rare DiseasesCutaneous venous malformationsPhase 3ongoingClinically significant angiokeratomasPhase 2ongoing
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- direclidineM4 / Small Molecule / PsychiatrySchizophreniaPhase 3Bipolar ManiaPhase 2
- NBI-570M1/M4 / Small Molecule / PsychiatrySchizophreniaPhase 2
- osavampatorAMPA / Small Molecule / PsychiatryMajor Depressive DisorderPhase 3
- NBI-986M4 / Small Molecule / NeurologyMovement DisordersPhase 1
- NBI-890VMAT2 / Small Molecule / PsychiatryTardive DyskinesiaPhase 2
- NBI-569M1/M4 / Small Molecule / PsychiatryAlzheimer's PsychosisPhase 1
- NBI-567M1 / Small Molecule / NeurologyAlzheimer's CognitionPhase 1
- NBI-355Nav1.2/1.6 / Small Molecule / NeurologyEpilepsyPhase 1
- NBIP-1435CRF1 / Peptide / EndocrinologyClassic Congenital Adrenal HyperplasiaPhase 1
- NBIP-2118CRF2 / Peptide / EndocrinologyObesityPhase 1
- NBIP-1968TG + NBIP-2118 Combo / Peptide / EndocrinologyObesityPhase 4
- NBIB-223Frataxin / Gene Therapy / NeurologyFriedreich's AtaxiaPhase 4
- NBI-675VMAT2 / Small Molecule / PsychiatryCNS IndicationsPhase 1
- NBI-188CRF1 / Small Molecule / EndocrinologyWomen's HealthPhase 4
- NBIM-1748ImmunologyUndisclosedPhase 4
- NBIM-1008ImmunologyUndisclosedPhase 4
- NBIM-1112ImmunologyUndisclosedPhase 1
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- FianlimabLAG3 / Monoclonal Antibody / Oncology1L metastatic melanoma, adjuvant melanomaPhase 3ActiveGlobal
- CemiplimabLibtayo / PD-1 / Monoclonal Antibody / OncologyCancerPhase 2/3ActiveGlobal
- DavutamigREGN5093 / METxMET / Bispecific Antibody / OncologyCancerPhase 2ActiveGlobal
- ItepekimabIL-33 / Monoclonal Antibody / Immunology & Inflammation / RespiratoryCOPD, CRSwNPPhase 3ActiveGlobal
- LinvoseltamabLynozyfic / BCMAxCD3 / Bispecific Antibody / OncologyMultiple myelomaPhase 2/3ActiveGlobal
- MarlotamigREGN7075 / EGFRxCD28 / Bispecific Antibody / OncologySolid TumorsPhase 2ActiveGlobal
- OdronextamabOrdspono / CD20xCD3 / Bispecific Antibody / OncologyLymphomaPhase 2ActiveGlobal
- UbamatamabREGN4018 / MUC16xCD3 / Bispecific Antibody / OncologyOvarian CancerPhase 2ActiveGlobal
- NezastomigREGN5678 / PSMAxCD28 / Bispecific Antibody / OncologyProstate CancerPhase 2ActiveGlobal
- RapirosiranALN-HSD / HSD17B13 / siRNA / Cardiovascular & MetabolicNASHPhase 2ActiveGlobal
- ALN-ANG3ALN-ANG3 / ANGPTL3 / siRNA / Cardiovascular & MetabolicDyslipidemiaPhase 2ActiveGlobal
- CemdisiranC5 / siRNA / Ophthalmology / ImmunologyGeographic atrophyClinicalActiveGlobal
- PozelimabC5 / Monoclonal Antibody / Ophthalmology / ImmunologyGeographic atrophyClinicalActiveGlobal
- BetV1REGN5713-5715 / BetV1 / Monoclonal Antibody / Immunology & InflammationBirch AllergyClinicalActiveGlobal
- DB-OTOOtarmeni / Otoferlin / Gene Therapy / Neurology / Hearing LossOtoferlin-related hearing lossPhase 1/2ActiveGlobal
- MibavademabUnknown / Biologic / OncologyCancerPhase 2ActiveGlobal
- TrevogrumabREGN1033 / Myostatin / Monoclonal Antibody / MetabolicMuscle weakness/wastingPhase 2ActiveGlobal
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- mezigdomidecereblon E3 ligase / CELMoD; oral targeted protein degrader / Hematologyrelapsed/refractory multiple myelomaPhase IIIactive
- iberdomidecereblon E3 ligase / CELMoD; oral targeted protein degrader / Hematologymultiple myelomaPhase IIIactive
- golcadomidecereblon E3 ligase / CELMoD; oral targeted protein degrader / Hematologyaggressive B-cell lymphomaPhase IIactive
- pumitamigBNT327/BMS-986545 / PD-L1; VEGF-A / bispecific antibody / Oncologysolid tumorsPhase IIIactive
- iza-brenantibody-drug conjugate / Oncologysolid tumorsPhase I/IIactive
- nivolumabOPDIVO / PD-1 / monoclonal antibody / Oncologymultiple oncology indicationsPhase IIIactive LCM
- nivolumab and relatlimab HDPD-1; LAG-3 / antibody combination / Oncologyoncology indicationsPhase IIIactive LCM
- ipilimumabYERVOY / CTLA-4 / monoclonal antibody / Oncologymultiple oncology indicationsPhase IIIactive LCM
- KarXTCOBENFY / muscarinic receptors / small molecule / Neuroscienceneuropsychiatric indicationsPhase IIIactive LCM
- milvexianFactor XIa / small molecule anticoagulant / Cardiovascularthrombotic disordersPhase IIIactive
- BMS-986278BMS-986278 / LPA1 / small molecule antagonist / Fibrosisidiopathic pulmonary fibrosisPhase IIIactive
- BMS-986495BMS-986495 / MTBR tau / antibody / NeuroscienceAlzheimer's diseasePhase IIactive
- imzokitugCCR8 / monoclonal antibody / Oncologysolid tumorsPhase I/IIactive
- obexelimabCD19; FcγRIIb / bifunctional antibody / Immunologyautoimmune diseasesPhase IIIactive
- luspatercept-aamtREBLOZYL / TGF-beta superfamily ligands / recombinant fusion protein / Hematologyanemia indicationsPhase IIIactive LCM
- OncoACP3OncoACP3 / prostate cancer target / radiopharmaceutical therapeutic/diagnostic agent / Oncologyprostate canceractive
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- guselkumabTREMFYA / IL-23 / Monoclonal Antibody / ImmunologyPsoriasisPsoriatic ArthritisCrohn's Disease
- ibrutinibIMBRUVICA / BTK / Small Molecule / OncologyMantle Cell LymphomaOther B-cell malignancies
- icotrokinraJNJ-2113 / IL-23R / Targeted Oral Peptide / ImmunologyPsoriasisPsoriatic Arthritis
- JNJ-4804JNJ-4804 / Co-antibody / Co-antibody Therapy / ImmunologyUlcerative Colitis
- nipocalimabIMAAVY / FcRn / Monoclonal Antibody / Immunology / NeuroscienceGeneralized Myasthenia GravisRheumatoid ArthritisWarm Autoimmune Hemolytic Anemia
- aticaprantKOR antagonist / Small Molecule / NeuroscienceAdjunctive Treatment for Major Depressive Disorder with Anhedonia
- RPGR Gene TherapyRPGR / Gene Therapy / Vision / OphthalmologyRetinitis Pigmentosa
- teclistamabTECVAYLI / BCMA x CD3 / Bispecific Antibody / OncologyMultiple Myeloma
- sCD59JNJ-1887 / sCD59 / Gene Therapy / Vision / OphthalmologyGeographic Atrophy
- gemcitabine intravesical delivery systemTAR-200 / INLEXZO / DNA synthesis / Drug Delivery System / OncologyHigh Risk Non-Muscle Invasive Bladder Cancer
- niraparib / abirateroneAKEEGA / PARP / CYP17 / Small Molecule Combination / OncologyMetastatic Castration-Sensitive Prostate CancerMetastatic Castration-Resistant Prostate Cancer
- daratumumabDARZALEX / CD38 / Monoclonal Antibody / OncologyMultiple Myeloma
- erdafitinibBALVERSA / FGFR / Small Molecule / OncologyUrothelial Carcinoma
- apalutamideERLEADA / Androgen Receptor / Small Molecule / OncologyProstate Cancer
- ciltacabtagene autoleucelCARVYKTI / BCMA / CAR-T Cell Therapy / OncologyMultiple Myeloma
- Filovirus multivalent vaccineVAC69120 / MVA-BN-Filo / Ebola Virus / Vaccine / Infectious DiseasesEbola
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- NiraparibPARP / Inhibitor / OncologyOvarian Cancer (1st line maintenance)ApprovedOvarian Cancer (Platinum sensitive relapsed maintenance)Approved
- Tumor Treating FieldsOncologyGlioblastoma (GBM)ApprovedPancreatic Cancer (1st line)Phase 3 / Pivotal
- RipretinibTKI / OncologyGastrointestinal Stromal Tumors (GIST) (4th line)Approved
- RepotrectinibROS1 / TRK / OncologyROS1+ NSCLCApprovedNTRK+ solid tumorsApproved
- Tisotumab vedotinTF / ADC / OncologyCervical Cancer (2nd line+ r/m)Phase 3 / PivotalCervical Cancer (1st line r/m, combo)Phase 2 / POC
- Zocilurtatug pelitecanZL-1310 / Zoci / DLL3 / ADC / OncologyES-SCLCPhase 3 / PivotalOther NECsPhase 2 / POC
- ZL-6201LRRC15 / ADC / OncologySarcoma & CAF+ Solid TumorPhase 1
- ZL-1311MUC17xCD3 / OncologyGI TumorsPre-clinical
- ZL-1222PD-1/IL-12 / OncologySolid TumorsPre-clinical
- EfgartigimodFcRn / ImmunologyGeneralized Myasthenia Gravis (gMG)ApprovedChronic Inflammatory Demyelinating Polyneuropathy (CIDP)ApprovedMyositisPhase 3 / Pivotal
- PovetaciceptBAFF/APRIL / ImmunologyIgA NephropathyPhase 3 / PivotalPrimary Membranous NephropathyPhase 2 / POC
- ElegrobartIGF-1R / ImmunologyTEDPhase 3 / Pivotal
- ZL-1503IL13/IL31R / ImmunologyMod-to-Sev ADPhase 1
- Xanomeline-TrospiumKarXT / NeuroscienceSchizophreniaApproved
- OmadacyclineInfectious DiseaseAcute Bacterial Skin and Skin Structure Infection (ABSSSI)ApprovedCommunity-Acquired Bacterial Pneumonia (CABP)Approved
- Sulbactam-DurlobactamInfectious DiseaseHABP/VABP caused by Susceptible Isolates of Acinetobacter Baumannii-calcoaceticus ComplexApproved
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- RelugolixTAK-385
- BrigatinibAlunbrig
- IxazomibNinlaro
- FruquintinibFruzaqla
- RusfertideTAK-121
- ElriterceptTAK-226
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- BotensilimabAGEN1181 / Anti-CTLA-4 / Fc-enhanced anti-CTLA-4 antibody / OncologyNon-MSI-H 4L+ metastatic colorectal cancerPhase 3Sites activeNon-MSI-H metastatic colorectal cancer NLMPhase 2Enrollment completePD-1 R/R melanomaPhase 2
- BalstilimabAGEN2034 / Anti-PD-1 / PD-1 inhibitor antibody / OncologyMSS metastatic colorectal cancer in combination with botensilimabPhase 3Sites activePD-1 R/R melanoma in combination with botensilimabPhase 2
- AGEN2373AGEN2373 / CD137 / CD137 agonist antibody / OncologySolid tumorsPhase 1PD-1 R/R melanoma with botensilimabPhase 2
- AGEN1571AGEN1571 / Anti-ILT-2 / Antibody / OncologySolid tumorsPhase 1
- AGEN1777AGEN1777 / Anti-TIGIT x CD96 / Bispecific antibody / OncologySolid tumors with balstilimabPhase 1
- AGEN1423AGEN1423 / Anti-CD73 x TGFB / Bispecific antibody / OncologySolid tumorsPhase 1
- INCAGN1876INCAGN1876 / Anti-GITR / Antibody / OncologySolid tumorsPhase 1
- AGEN1949AGEN1949 / OX40 / OX40 agonist antibody / OncologySolid tumorsPhase 1
- MK-4830MK-4830 / ILT4 / Anti-ILT4 antagonist antibody / OncologyNeoadjuvant ovarian cancerPhase 2Ongoing
- INCAGN2390INCAGN2390 / TIM-3 / Anti-TIM-3 antagonist antibody / OncologyMelanoma and SCCHN in combinationIn development
- INCAGN2385INCAGN2385 / LAG-3 / Anti-LAG-3 antagonist antibody / OncologyMelanoma and SCCHN in combinationIn development
- CR6086 + BalstilimabCR6086; AGEN2034 / EP4 + PD-1 / EP4 antagonist plus PD-1 antagonist / OncologypMMR-MSS colorectal cancerPhase 1/2Ongoing
- NLM001 + ZalifrelimabNLM-001 / Hedgehog + CTLA-4 / Hedgehog inhibitor plus CTLA-4 antagonist / OncologyAdvanced pancreatic cancerPhase 1/2Ongoing
- OBT076 + BalstilimabOBT076; AGEN2034 / CD205 + PD-1 / Antibody-drug conjugate plus PD-1 antibody / OncologySolid tumorsOngoing
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- High-concentration, low-volume adalimumabAVT02 / HUMIRA® / Biosimilar / ImmunologyBiosimilar to HUMIRA®ApprovedApprovedU.S.Europe (EEA, UK, Switzerland, Balkans)CanadaAustraliaSaudi ArabiaIsraelEgyptSouth AfricaMoroccoPeruBolivia
- ustekinumabAVT04 / STELARA® / Biosimilar / ImmunologyBiosimilar to STELARA®ApprovedApprovedU.S.JapanCanadaEU/EEA
- denosumabAVT03 / PROLIA®/XGEVA® / Biosimilar / Bone DiseaseBiosimilar to PROLIA®/XGEVA®ApprovedApprovedJapanEU/EEA
- golimumabAVT05 / SIMPONI®/SIMPONI ARIA® / Biosimilar / ImmunologyBiosimilar to SIMPONI®/SIMPONI ARIA®ApprovedApprovedJapanEU/EEAUnited Kingdom
- afliberceptAVT06 / EYLEA® / Biosimilar / OphthalmologyBiosimilar to EYLEA®ApprovedApprovedEU/EEAJapanUnited Kingdom
- omalizumabAVT23 / XOLAIR® / Biosimilar / Respiratory
- vedolizumabAVT16 / ENTYVIO® / Biosimilar / Immunology
- pembrolizumabAVT33 / KEYTRUDA® / Biosimilar / Oncology
- certolizumab pegolAVT10 / CIMZIA® / Biosimilar / Immunology
- ixekizumabAVT28 / TALTZ® / Biosimilar / Immunology
- canakinumabAVT48 / ILARIS® / Biosimilar / Immunology
- guselkumabAVT41 / TREMFYA® / Biosimilar / Immunology
- ofatumumabAVT65 / KESIMPTA® / Biosimilar / Immunology
- dupilumabAVT19 / DUPIXENT® / Biosimilar / Immunology
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- 177Lu rosopatamab tetraxetanTLX591-Tx / PSMA / Antibody / Urologic OncologyProstate Cancer
- 177Lu-DOTA-PSMATLX597-Tx / PSMA / Small molecule / Urologic OncologyProstate Cancer
- 177Lu-girentuximabTLX250-Tx / CAIX / Antibody / Urologic OncologyKidney Cancer / ccRCC
- 131I-IPATLX101-Tx / LAT / Small molecule / Neuro-oncologyBrain Cancer / Glioma
- 90Y-besilesomabTLX66-Tx / CD66 / Antibody / HematologyBone-marrow conditioning
- 225Ac-RADmAbTLX592-Tx / PSMA / Antibody / Urologic OncologyProstate Cancer
- 225Ac-girentuximabTLX252-Tx / CAIX / Antibody / Urologic OncologyKidney Cancer
- UndisclosedTLX400-Tx / FAP / Small molecule / Urologic OncologyBladder Cancer
- 211At-APATLX102-Tx / LAT / Small molecule / Neuro-oncologyBrain Cancer
- -olaratumab conjugateTLX300-Tx / PDGFRα / Antibody / Musculoskeletal OncologySoft Tissue Sarcoma
- 153Sm-DOTMPTLX090-Tx / Small molecule / Musculoskeletal OncologyBone metastasesPain palliation
- 68Ga-PSMA-11TLX591-CDx / Illuccix / Gozellix / PSMA / Diagnostic Radiopharmaceutical / OncologyProstate Cancer Imaging
- 18F-floretyrosineTLX101-CDx / Pixclara / Pixlumi / LAT / Diagnostic Radiopharmaceutical / Neuro-oncologyGlioma Imaging
- 89Zr-DFO-girentuximabTLX250-CDx / Zircaix / CAIX / Diagnostic Radiopharmaceutical / OncologyClear Cell Renal Cell Carcinoma (ccRCC) Imaging
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- EfgartigimodVYVGART; VYVGART Hytrulo / FcRn / antibody fragment / AutoimmuneGeneralized Myasthenia Gravis (gMG)CommercialChronic Inflammatory Demyelinating Polyneuropathy (CIDP)CommercialPrimary Immune Thrombocytopenia (ITP)CommercialJapan
- EmpasiprubartC2 / monoclonal antibody / AutoimmuneMultifocal Motor Neuropathy (MMN)RegistrationalDelayed Graft Function (DGF)Proof of ConceptChronic Inflammatory Demyelinating Polyneuropathy (CIDP)Registrational
- AdimanebartMuSK / agonist antibody / NeurologyCongenital Myasthenic Syndromes (CMS)Proof of ConceptSpinal Muscular Atrophy (SMA)Proof of Concept
- ARGX-109ARGX-109 / IL-6 / antibody / UndisclosedUndisclosedPhase 1
- ARGX-121ARGX-121 / IgA / antibody / UndisclosedUndisclosedPhase 1
- ARGX-213ARGX-213 / FcRn / antibody / UndisclosedUndisclosedPhase 1
- ARGX-118ARGX-118 / Galectin-10 / Airway InflammationAirway InflammationPreclinical
- ARGX-125ARGX-125 / bispecific antibody / UndisclosedUndisclosedPreclinical
- TSP-101TSP-101 / Fn14 / UndisclosedUndisclosedPreclinical
- LP0145ARGX-112 / IL-22R / SIMPLE Antibody / DermatologyAtopic Dermatitispartnered
- AGMB-101ARGX-114 / MET / HGF-mimetic agonist antibody
- ABBV-151ARGX-115 / GARP-TGF-beta1 / SIMPLE Antibody / OncologyTumorspartnered
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- CasdatifanAB521 / HIF-2α Inhibitor
- QuemliclustatCD73 Inhibitor
- DomvanalimabAnti-TIGIT Antibody
- ZimberelimabAnti-PD-1 Antibody
- EtrumadenantA2a/A2b Receptor Antagonist
- AB598Anti-CD39 Antibody
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- Taldefgrobep AlfaBHV-2000 / myostatin/activin receptor signaling / recombinant protein; myostatin-activin pathway inhibitor / neuromuscular; metabolicSpinal muscular atrophyPhase 3ObesityPhase 1
- OpakalimBHV-7000 / Kv7.2/Kv7.3 potassium channels / small molecule Kv7 activator / neurology; psychiatryFocal epilepsyPhase 3Generalized epilepsyPhase 3Major depressive disorderPhase 2
- TRPM3 AntagonistBHV-2100 / TRPM3 / oral small molecule antagonist / painPain disordersPhase 2
- TYK2/JAK1 InhibitorBHV-8000 / TYK2/JAK1 / brain-penetrant small molecule inhibitor / neuroinflammationParkinson's diseasePhase 3Prevention of amyloid therapy induced ARIAPhase 1Alzheimer's diseasePhase 1
- IgG DegraderBHV-1300 / IgG / MoDE extracellular protein degrader / immunologyGraves' disease / rheumatoid arthritisPhase 1
- IgG DegraderBHV-1310 / IgG / MoDE extracellular protein degrader / immunologyMyasthenia gravisPreclinical
- Gd-IgA1 TRAP DegraderBHV-1400 / galactose-deficient IgA1 / TRAP extracellular protein degrader / immunology; renalIgA nephropathyPhase 1
- β1AR Autoantibody DegraderBHV-1600 / β1AR autoantibodies / bifunctional MoDE degrader / cardiovascularPeripartum cardiomyopathyPhase 1
- Trop2 ADC +/- PD1BHV-1510 / TROP2 / antibody-drug conjugate / oncologyAdvanced or metastatic epithelial tumorsPhase 1
- FGFR3 ADCBHV-1530 / FGFR3 / antibody-drug conjugate / oncologyUrothelial cancer and other tumorsPhase 1
- CD30 ADCBHV-1500 / CD30 / antibody-drug conjugate / oncologyHodgkin lymphomaPreclinical
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- LecanemabLEQEMBI / Aβ / mAb / NeurologyPreclinical Alzheimer's diseasePhase 3Early Alzheimer's disease, SC-AI initiationRegulatory review; FDA PDUFA May 24, 2026U.S.
- DiranersenBIIB080 / tau / ASO / NeurologyEarly Alzheimer's diseasePhase 2PoC readout reported May 14, 2026
- BIIB091BIIB091 / BTK / peripheral BTK inhibitor / NeurologyMultiple sclerosisPhase 2
- Dapirolizumab pegolCD40L / pegylated antibody fragment / ImmunologySystemic lupus erythematosusPhase 3
- LitifilimabBDCA2 / mAb / ImmunologySystemic lupus erythematosusPhase 3Cutaneous lupus erythematosusPhase 3FDA Breakthrough Therapy DesignationU.S.
- BIIB142BIIB142 / IRAK4 / degrader / ImmunologyAutoimmune diseasePhase 1
- FelzartamabCD38 / mAb / Rare diseaseAntibody-mediated rejectionPhase 3IgA nephropathyPhase 3Primary membranous nephropathyPhase 3
- OmaveloxoloneSKYCLARYS / Nrf2 / activator / Rare diseasePediatric Friedreich ataxiaPhase 3
- SalanersenBIIB115 / SMN / ASO / Rare diseaseSpinal muscular atrophyPhase 3
- ZorevunersenSCN1A / ASO / Rare diseaseDravet syndromePhase 3
- Nusinersen high doseSPINRAZA / SMN2 / splice modulator / Rare diseaseSpinal muscular atrophyApproved in U.S.; under review in E.U.U.S.E.U.
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- tividenofusp alfa-eknmDNL310; ETV:IDS / AVLAYAH / IDS enzyme replacement; TfR TransportVehicle-enabled delivery / Enzyme TransportVehicle biologic / Lysosomal storage disordersHunter syndrome (MPS II)Approved; Phase 2/3 confirmatory study ongoingU.S. accelerated approval; global Phase 2/3 COMPASS ongoingUSGlobal
- DNL126ETV:SGSH / SGSH enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disordersSanfilippo syndrome Type A (MPS IIIA)Phase 1/2; Phase 3 plannedOngoing Phase 1/2; global Phase 3 confirmatory study plannedGlobal
- DNL593TAK-594/DNL593; PTV:PGRN / Progranulin replacement / Protein TransportVehicle biologic / Neurodegenerative diseaseGRN-related frontotemporal dementia (FTD-GRN)Phase 1/2Ongoing; Denali received notice that Takeda will terminate collaboration, with Denali planning to continue developmentGlobal
- DNL628OTV:MAPT / MAPT / tau / Oligonucleotide TransportVehicle therapeutic / Neurodegenerative diseaseAlzheimer's diseasePhase 1bCTA approved; study start-up underway
- DNL952ETV:GAA / GAA enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disordersPompe diseasePhase 1IND clinical hold lifted; proceeding to Phase 1US
- BIIB122/DNL151DNL151; BIIB122 / LRRK2 / Small molecule inhibitor / Neurodegenerative diseaseParkinson's diseasePhase 2bLUMA enrollment completed; readout expected 2026GlobalLRRK2-associated Parkinson's diseasePhase 2aBEACON ongoing
- eclitasertibSAR443122/DNL758 / RIPK1 / Small molecule inhibitor / Inflammatory diseaseUlcerative colitisPhase 2Developed by Sanofi; Phase 2 results expected in 1H 2026
- DNL921ATV:Abeta / Amyloid beta / Antibody TransportVehicle therapeutic / Neurodegenerative diseaseAlzheimer's diseaseIND-enablingPhase 1/1b initiation planned
- DNL111ETV:GCase / GCase / Enzyme TransportVehicle biologic / Neurodegenerative disease; lysosomal storage disordersParkinson's diseaseIND-enablingIND-enablingGaucher diseaseIND-enablingIND-enabling
- DNL622ETV:IDUA / IDUA enzyme replacement / Enzyme TransportVehicle biologic / Lysosomal storage disordersMPS IIND-enablingIND-enabling
- DNL422OTV:SNCA / SNCA / alpha-synuclein / Oligonucleotide TransportVehicle therapeutic / Neurodegenerative diseaseParkinson's diseaseIND-enablingIND-enabling
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- INCA033989INCA033989 / mutCALR / HematologyCALR-mutated essential thrombocythemia (2L)Phase 3Initiation mid-2026USCALR-mutated myelofibrosis (1L)Phase 1Data expected 2H 2026CALR-mutated myelofibrosis (2L)Phase 3Initiation 2H 2026
- INCB161734INCB161734 / KRAS G12D / Oncology1L G12D-mutated pancreatic ductal adenocarcinoma (PDAC)Phase 3Ongoing (in combo with SOC chemo)KRAS G12D-mutated solid tumorsPhase 1Data expected 2H 2026
- INCA33890INCA33890 / TGFβR2 × PD-1 / bispecific / Oncology1L microsatellite-stable colorectal cancer (MSS CRC)Phase 3Ongoing1L MSS PDAC (combo with SOC)Phase 1Data expected 2H 2026
- INCB123667INCB123667 / CDK2 / OncologyPlatinum-resistant ovarian cancer (PROC)PivotalOngoing1L maintenance ovarian cancer (CCNE1+)Phase 3Initiation planned 2H 2026
- povorcitinibJAK1 / small molecule / Inflammation and AutoimmunityModerate-to-severe hidradenitis suppurativa (HS)Phase 3NDA accepted; approval anticipated 1Q 2027 (US), late 2026 (EU)USEUNonsegmental vitiligoPhase 3Positive data; sNDA submission 1H 2027Moderate-to-severe prurigo nodularis (PN)Phase 3Ongoing; topline data 4Q 2026
- ruxolitinib creamOpzelura / JAK1/JAK2 / topical / Inflammation and AutoimmunityModerate atopic dermatitisApprovedApproval and launch (ex-US)EUMild-to-moderate hidradenitis suppurativa (HS)Phase 3Ongoing; topline data 4Q 2026
- axatilimabNiktimvo / CSF-1R / Hematology1L chronic GVHD (+ ruxolitinib)PivotalData 2H 20261L chronic GVHD (+ steroids)Data early 20283L+ chronic GVHDApprovedApprovedUS
- INCB160058INCB160058 / JAK2V617F / HematologyJAK2 V617F-mutated myeloproliferative neoplasms (MPNs)Phase 1Data 2H 2026 (ASD formulation)
- tafasitamabMonjuvi / CD19 / Hematology1L diffuse large B-cell lymphoma (DLBCL)Phase 3sBLA submission 1H 2026USRelapsed/refractory DLBCLApprovedApprovedUSEUJapan
- ruxolitinib XRJakafi XR / JAK1/JAK2 / HematologyMyelofibrosis, polycythemia vera, chronic GVHDRegistrationFDA resubmissionUS
- retifanlimabZynyz / PD-1 / OncologySquamous cell carcinoma of the anal canal (SCAC)ApprovedApproved (EC approval noted)USEUMerkel cell carcinomaApprovedApprovedUSEUJapan
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- B-VECB-VEC / VYJUVEK / Type VII collagen (COL7A1) / Gene Therapy (HSV-1 vector) / DermatologyDystrophic Epidermolysis Bullosa (DEB)CommercialApprovedGlobal
- KB105KB105 / Transglutaminase 1 (TGM1) / Gene Therapy / DermatologyTGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)Phase 1/2ActiveGlobal
- KB104KB104 / Serine Peptidase Inhibitor Kazal Type 5 (SPINK5) / Gene Therapy / DermatologyNetherton SyndromePreclinicalActiveGlobal
- KB407KB407 / Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) / Gene Therapy / RespiratoryCystic FibrosisPhase 1ActiveGlobal
- KB408KB408 / Alpha-1 antitrypsin / Gene Therapy / RespiratoryAlpha-1 Antitrypsin Deficiency (AATD)PreclinicalActiveGlobal
- KB707KB707 / IL-12 and IL-2 / Gene Therapy (HSV-1 vector) / OncologyLocally advanced or metastatic solid tumor malignanciesPhase 1ActiveGlobal
- KB301KB301 / Type III collagen / Gene Therapy / AestheticsAesthetic Skin ConditionsPhase 1/2ActiveGlobal
- KB302KB302 / Type I collagen / Gene Therapy / AestheticsAesthetic Skin ConditionsPreclinicalActiveGlobal
- KB303KB303 / Elastin / Gene Therapy / AestheticsAesthetic Skin ConditionsPreclinicalActiveGlobal
- KB304KB304 / Type III collagen and Elastin / Gene Therapy / AestheticsAesthetic Skin ConditionsPreclinicalActiveGlobal
- KB305KB305 / Type IV collagen / Gene Therapy / AestheticsAesthetic Skin ConditionsPreclinicalActiveGlobal
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- amlitelimabOX40L / Monoclonal antibody / ImmunologyAtopic dermatitisPhase 3ActiveGlobal
- tolebrutinibBTK / Small molecule / NeurologyMultiple sclerosisRegistrationActiveGlobalUS
- lunsekimigBiologic / Immunology and InflammationAsthma / Respiratory diseasesPhase 2ActiveGlobal
- rilzabrutinibBTK / Small molecule / Rare Blood Disorders / ImmunologySickle cell diseasePhase 2/3ActiveGlobalIgG4-related diseasePhase 3ActiveGlobalAsthmaPhase 2ActiveGlobal
- venglustatSmall molecule / Rare DiseasesGaucher disease type 3 (GD3)Phase 3ActiveGlobal
- frexalimabCD40L / Monoclonal antibody / ImmunologyMultiple sclerosisPhase 3ActiveGlobal
- SAR441566SAR441566 / TNFR1si / Small molecule / ImmunologyImmunological diseasesPhase 2ActiveGlobal
- duvakitugTL1A / Monoclonal antibody / ImmunologyUlcerative colitisPhase 2ActiveGlobalCrohn's diseasePhase 2ActiveGlobal
- itepekimabIL-33 / Monoclonal antibody / Immunology and InflammationCOPDPhase 3ActiveGlobal
- isatuximabSarclisa / CD38 / Monoclonal antibody / OncologyMultiple myeloma (subcutaneous)RegistrationActiveEU
- acoziboroleSmall molecule / Infectious DiseasesSleeping sicknessRegistrationActiveAfricaEU
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- Ozuriftamab vedotinBA3021 / ROR2 / CAB-ROR2-ADC / OncologyOPSCCPhase 3
- Mecbotamab vedotinBA3011 / AXL / CAB-AXL-ADC / OncologySarcoma (soft tissue and bone)Phase 2mKRAS NSCLCPhase 2
- EvalstotugBA3071 / CTLA-4 / CAB-CTLA-4 antibody / OncologyUnresectable and/or metastatic cutaneous melanomaPhase 2
- BA3182BA3182 / EpCAM x CD3 / Dual CAB T cell engager / OncologyAdenocarcinomaPhase 1ongoing
- BA3361BA3361 / Nectin-4 / CAB-Nectin4-ADC / OncologyMultiple tumor typesPreclinicalIND-approved
- BA3151BA3151 / B7-H4 / CAB-B7H4-ADC / OncologyBreast cancerPreclinical
- BA3142BA3142 / B7-H3 x CD3 / Dual CAB TCE / OncologyMultiple tumor typesPreclinicalIND-ready
- BA3311BA3311 / EGFR x CD3 / TCE / OncologyMultiple tumor typesPreclinical
- BA3241BA3241 / Trop2 x CD3 / Dual CAB TCE / OncologyEpidermoid cancerPreclinical
- BA3362BA3362 / Nectin4 x CD3 / Dual CAB TCE / Oncology
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- Relacorilantglucocorticoid receptor / selective cortisol modulator / EndocrinologyHypercortisolism (GRACE)Phase IIIFiledHypercortisolism associated with adrenal adenoma or hyperplasia (GRADIENT)Phase IIIFiledLong-term safety extensionPhase IIFiled
- KorlymKorlym / glucocorticoid receptor / cortisol modulator / EndocrinologyPrevalence and treatment of hypercortisolism in difficult-to-control type 2 diabetes (CATALYST)Phase IV
- LifyorliLifyorli / glucocorticoid receptor / selective cortisol modulator / OncologyPlatinum-resistant ovarian cancer (ROSELLA)Phase IIIApproved
- Relacorilant + Nab-paclitaxel + Bevacizumabglucocorticoid receptor / selective cortisol modulator combination / OncologyPlatinum-resistant ovarian cancer (BELLA Arm A)Phase IIPlatinum-sensitive ovarian cancer (BELLA Arm B)Phase III
- Relacorilant + Nab-paclitaxelglucocorticoid receptor / selective cortisol modulator combination / OncologyEndometrial cancer (BELLA Arm C)Phase IICervical cancer (STELLA)Phase II
- Relacorilant + Nab-paclitaxel + Gemcitabineglucocorticoid receptor / selective cortisol modulator combination / OncologyPancreatic cancer (TRIDENT)Phase II
- Nenocorilant + Nivolumabglucocorticoid receptor / selective cortisol modulator combination / OncologySolid tumors (SYNERGY)Phase II
- Relacorilant + Enzalutamideglucocorticoid receptor / selective cortisol modulator combination / OncologyProstate cancerPhase II
- Dazucorilantglucocorticoid receptor / selective cortisol modulator / NeurologyALS (DAZALS)Phase II
- Miricorilantglucocorticoid receptor / selective cortisol modulator / MetabolismMASH (MONARCH)Phase II
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- FruquintinibHMPL-013 / ELUNATE / FRUZAQLA / VEGFR 1/2/3 / Small molecule / OncologyColorectal cancerApproved/MarketedApprovedChinaUSEuropeJapanGastric cancerPhase IIIActiveGlobalOther solid tumors (combination studies)ClinicalActiveGlobal
- SavolitinibHMPL-504 / AZD6094 / ORPATHYS / c-MET / Small molecule / OncologyNon-small cell lung cancer (MET exon 14)Approved/MarketedApprovedChinaNSCLC combinations (with immunotherapy/targeted therapy)Phase IIIActiveGlobal
- SurufatinibHMPL-012 / SULANDA / VEGFR/FGFR/CSF-1R / Small molecule / OncologyNeuroendocrine tumorsApproved/MarketedApprovedChina
- SovleplenibHMPL-523 / Syk / Small molecule / Hematology/ImmunologyImmune thrombocytopenia (ITP)NDA/Regulatory reviewUnder regulatory reviewChina
- FanregratinibHMPL-453 / FGFR / Small molecule / OncologyCholangiocarcinoma (FGFR2 fusion)NDA/Regulatory reviewUnder regulatory reviewChina
- HMPL-760HMPL-760 / BTK (non-covalent) / Small molecule / Oncology/HematologyRelapsed/refractory diffuse large B-cell lymphoma (DLBCL)Phase IIIInitiatedChina
- HMPL-A251HMPL-A251 / HER2 (PI3K/PIKK payload) / Antibody-Targeted Therapy Conjugate (ATTC) / OncologyHER2-positive/HER2-low solid tumorsPhase IActiveChinaUS
- HMPL-A580HMPL-A580 / EGFR (PI3K/PIKK payload) / Antibody-Targeted Therapy Conjugate (ATTC) / OncologySolid tumorsPhase IInitiatedGlobal
- HMPL-A830HMPL-A830 / ATTC (undisclosed) / Antibody-Targeted Therapy Conjugate (ATTC) / OncologySolid tumorsPreclinical/Phase I plannedPlannedGlobal
- TazemetostatTAZVERIK / EZH2 / Small molecule / OncologyEpithelioid sarcoma / follicular lymphomaLicensed/CommercialLicensed product updateChina
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- tirzepatideMounjaro / Zepbound
- donanemabKisunla
- abemaciclibVerzenio
- pirtobrutinibJaypirca
- mirikizumabOmvoh
- lebrikizumabEbglyss
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- BerobenatidePF-06954522 / GLP-1/GIP / Small Molecule / Internal MedicineChronic Weight ManagementPhase 2bOngoingGlobal
- elranatamabPF-06863135 / ELREXFIO / BCMA x CD3 / Bispecific Antibody / OncologyDouble-class Exposed Relapsed / Refractory Multiple MyelomaPhase 3OngoingGlobal
- ritlecitinibPF-06651600 / LITFULO / JAK3/TEC / Small Molecule / Inflammation and ImmunologyVitiligoPhase 3OngoingGlobalModerate Alopecia AreataPhase 3OngoingGlobal
- marstacimabPF-06741086 / HYMPAVZI / TFPI / Monoclonal Antibody / Rare DiseaseHemophilia A/B with InhibitorsRegistrationOngoingGlobal
- MevrometostatPF-06821497 / EZH2 / Small Molecule / Oncology1-2L Metastatic Castration-resistant Prostate Cancer Post-abirateronePhase 3OngoingGlobal
- Sigvotatug vedotinPF-08046047 / Integrin beta-6 / Antibody-Drug Conjugate (ADC) / OncologyNon-Small Cell Lung Cancer (NSCLC)Phase 3OngoingGlobal
- VepdegestrantARV-471 / Estrogen Receptor (ER) / PROTAC Protein Degrader / OncologyER+/HER2- Metastatic Breast CancerPhase 3OngoingGlobal
- PCV25PF-07872412 / Pneumococcus / Vaccine / VaccinesPneumococcal InfectionPhase 3OngoingGlobal
- enfortumab vedotinPADCEV / Nectin-4 / Antibody-Drug Conjugate (ADC) / OncologyMuscle-invasive Bladder Cancer (Bladder Sparing)Phase 3OngoingGlobal
- Lyme Disease Vaccine CandidatePF-07307405 / Borrelia burgdorferi OspA / Vaccine / VaccinesLyme Disease InfectionPhase 3OngoingGlobal
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- ADG126ADG126 / muzastotug / CTLA-4 / fully human anti-CTLA-4 SAFEbody / Oncologyadvanced/metastatic solid tumorsPhase 1b/2dose expansion ongoing in combination with anti-PD-1 therapies
- ADG116ADG116 / CTLA-4 / fully human anti-CTLA-4 NEObody / Oncologyadvanced/metastatic solid tumorsPhase 1b/2ongoing
- ADG206ADG206 / CD137 / masked IgG1 Fc-engineered anti-CD137 POWERbody / Oncologyadvanced/metastatic tumorsPhase 1ongoing
- ADG106ADG106 / CD137 / fully human ligand-blocking agonistic anti-CD137 IgG4 mAb NEObody / Oncologyadvanced/metastatic solid tumors and/or relapsed/refractory non-Hodgkin's lymphomaPhase 1b/2ongoing
- ADG153ADG153 / CD47 / masked anti-CD47 IgG1 SAFEbody / Oncologyhematologic and solid tumorsIND-enablingpreclinical
- ADG138ADG138 / HER2 x CD3 / HER2xCD3 POWERbody T-cell engager / OncologyHER2-expressing solid tumorsIND-enablingpreclinical
- ADG152ADG152 / CD20 x CD3 / bispecific CD20xCD3 T-cell engager POWERbody / OncologyB-cell malignanciesIND-enablingpreclinical
- ADG104ADG104 / PD-L1 / antibody / OncologycancerPhase 2in development by Sanjin
- ADG125ADG125 / CSF-1R / antibody / OncologycancerPhase 1in development by Dragon Boat Biopharmaceutical
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- CASGEVYexa-cel / CASGEVY / BCL11A erythroid-specific enhancer / Non-viral ex vivo CRISPR/Cas9 gene-edited autologous cell therapy / HemoglobinopathiesSickle cell disease, ages 5-11Regulatory submissions expected H1 2026Active pediatric expansionGlobalTransfusion-dependent beta thalassemia, ages 5-11Regulatory submissions expected H1 2026Active pediatric expansionGlobal
- CTX310CTX310 / ANGPTL3 / In vivo CRISPR/Cas9 gene editing; LNP delivery / Cardiovascular/metabolicSevere hypertriglyceridemiaPhase 1bActive; update expected H2 2026Refractory hypercholesterolemiaPhase 1bActive; update expected H2 2026Heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias or severe hypertriglyceridemiaPhase 1Developed/being evaluated
- CTX320CTX320 / LPA / In vivo CRISPR/Cas9 gene editing; LNP delivery / CardiovascularElevated lipoprotein(a)Clinical trial / dose escalationActive; Lp(a) program update expected in 2026
- CTX321CTX321 / LPA / Next-generation in vivo CRISPR/Cas9 gene editing; LNP delivery / CardiovascularElevated lipoprotein(a)IND/CTA-enablingActive
- CTX460CTX460 / SERPINA1 / In vivo SyNTase gene correction; LNP delivery / Rare diseaseAlpha-1 antitrypsin deficiencyPreclinical; clinical trial expected mid-2026Active
- CTX340CTX340 / AGT / In vivo CRISPR/Cas9 gene editing; LNP delivery / CardiovascularRefractory hypertensionIND/CTA-enabling; clinical trial expected H1 2026Active
- CTX611SRSD107 / Factor XI / FXI mRNA / Long-acting double-stranded siRNA / Thromboembolic/cardiovascular diseasesVenous thromboembolism prevention after total knee arthroplastyPhase 2Ongoing; top-line data expected H2 2026EuropeAtrial fibrillation, venous thromboembolism, ischemic stroke, cancer-associated thrombosis, chronic kidney disease, peripheral vascular disease, chronic coronary artery diseaseTarget indications disclosed
- Zugocabtagene geleucelzugo-cel; formerly CTX112 / CD19 / Allogeneic CAR-T cell therapy; CRISPR Cas9 engineered / Autoimmune disease; oncologySystemic lupus erythematosusPhase 1Ongoing; update expected H2 2026Systemic sclerosisPhase 1Ongoing; update expected H2 2026Inflammatory myositisPhase 1Ongoing; update expected H2 2026
- CTX213CTX213 / iPSC-derived allogeneic gene-edited beta islet cell precursor therapy / Regenerative medicine / diabetesType 1 diabetesPreclinical; progressing toward clinicActive
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- EVT8683BMS-986419 / eIF2b / NeuroscienceNeurodegeneration
- CNTX 6016CB2 / PainPain
- EVT801VEGFR3 / OncologyOncology
- EVT401P2X7 / ImmunologyInflammationAutoimmune
- EVT894Infectious DiseasesChikungunya
- EXS21546A2a / OncologyOncology
- DSP-11815-HT1A / NeuroscienceObsessive Compulsive Disorder (OCD)
- CT7001CDK7 / OncologyOncology
- TPM203ImmunologyPemphigus Vulgaris
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- DarovasertibIDE196 / PKC / Small Molecule / Oncology1L metastatic uveal melanoma (HLA-A*02:01 negative)Phase 2/3Positive topline; NDA initiationGlobalNeoadjuvant primary uveal melanomaPhase 2/3OngoingUS
- IDE397IDE397 / MAT2A / Small Molecule / OncologyMTAP-deleted solid tumorsPhase 1OngoingUS
- IDE849IDE849 / DLL3 / ADC / OncologyDLL3-expressing solid tumors (SCLC, NEC)Phase 1OngoingUS
- IDE161IDE161 / PARG / Small Molecule / OncologyHRD solid tumorsPhase 1RecruitingUS
- IDE034IDE034 / PTK7/B7H3 / Bispecific ADC / OncologyB7H3/PTK7 solid tumors (NSCLC, CRC, breast, ovarian)Phase 1OngoingUS
- IDE892IDE892 / PRMT5 / Small Molecule / OncologyMTAP-deleted solid tumorsPhase 1OngoingUS
- IDE574IDE574 / KAT6/7 / Small Molecule / OncologySolid tumors (breast, prostate, CRC, lung)Phase 1OngoingUS
- IDE275IDE275 / Werner Helicase (WRN) / Small Molecule / OncologyMSI-high solid tumorsPhase 1OngoingUS
- IDE705IDE705 / Pol Theta (POLQ) / Small Molecule / OncologyHRD solid tumorsPhase 1OngoingUS
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- BrensocatibBRINSUPRI / DPP1 / Small Molecule / Respiratory / ImmunologyNon-Cystic Fibrosis Bronchiectasis (NCFB)MarketedActiveUSEUHidradenitis SuppurativaPhase 2ActiveGlobal
- Amikacin Liposome Inhalation SuspensionARIKAYCE / Bacterial 30S ribosomal subunit / Liposomal Inhalation Suspension / Respiratory / Infectious DiseaseRefractory Mycobacterium avium complex (MAC) Lung DiseaseMarketedActiveUSEUJPMAC Lung Disease (Label Expansion)Phase 3ActiveGlobal
- Treprostinil Palmitil Inhalation PowderTPIP / Prostacyclin receptor / Inhalation Powder / Respiratory / PulmonaryPulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)Phase 3ActiveGlobalPulmonary Arterial Hypertension (PAH)Phase 3ActiveGlobalProgressive Pulmonary Fibrosis (PPF)Phase 3 PlannedActiveGlobal
- INS1148INS1148 / Stem Cell Factor SCF248 / Monoclonal Antibody / Respiratory / ImmunologyInterstitial Lung Disease (ILD)Phase 2ActiveGlobalModerate-to-severe asthmaPhase 2ActiveGlobal
- INS1033INS1033 / Unknown / Unknown / Immunology & InflammationInflammatory diseasesPreclinicalActiveGlobal
- Intrathecal gene therapy for DMDDystrophin / Gene Therapy / Neurology / Rare DiseaseDuchenne Muscular Dystrophy (DMD)Phase 1ActiveGlobal
- INS1202INS1202 / Unknown / Unknown / Neurology / Rare DiseaseAmyotrophic Lateral Sclerosis (ALS)Phase 1ActiveGlobal
- Stargardt Disease Gene TherapyUnknown / Gene Therapy / Ophthalmology / Rare DiseaseStargardt DiseasePreclinicalActiveGlobal
- Next-Gen DPP1 InhibitorsDPP1 / Small Molecule / Immunology & InflammationUndisclosedPreclinicalActiveGlobal
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- ZanidatamabHER2 / Bispecific Antibody / OncologyBiliary Tract Cancers (BTC)Gastroesophageal Adenocarcinoma (GEA)Colorectal Cancer (CRC)
- LurbinectedinZepzelca / DNA alkylating / Small Molecule / OncologySmall Cell Lung Cancer (SCLC)Ewing Sarcoma
- DordaviproneModeyso / ClpP agonist / DRD2 antagonist / Small Molecule / OncologyH3 K27M-mutant diffuse glioma
- Daunorubicin and cytarabineJZP351 / Vyxeos / Vyxeos Liposomal / Topoisomerase inhibitor / nucleoside metabolic inhibitor / Liposomal Combination / OncologyMyelodysplastic Syndromes (MDS)Relapsed/Refractory Acute Myeloid Leukemia (R/R AML)
- JZP898JZP898 / IFNα2b cytokine pro-drug / Biologic / OncologySolid Tumors
- JZP3507JZP3507 / ClpP agonist / DRD2 antagonist / Small Molecule / OncologyOncology
- JZP815JZP815 / pan-RAF / Small Molecule / OncologySolid TumorsHematologic Malignancies
- CannabidiolEpidyolex / Cannabinoid Receptors / Small Molecule / NeuroscienceLennox-Gastaut Syndrome (LGS)Dravet Syndrome (DS)Tuberous Sclerosis Complex (TSC)
- JZP441JZP441 / Orexin-2 receptor agonist / Small Molecule / NeuroscienceNarcolepsy Type 1Idiopathic Hypersomnia
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- pembrolizumabMK-3475 / Keytruda
- belzutifanMK-6482 / Welireg
- sotaterceptMK-7962
- sacituzumab tirumotecanMK-2870
- efinopegdutideMK-6024
- gocatamigMK-6070
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- Isaralgagene civaparvovecST-920 / alpha-galactosidase A (GLA) / Gene Therapy (AAV) / Metabolic DiseasesFabry DiseaseBLA/FilingActiveUS
- Giroctogene fitelparvovecSB-525 / Factor VIII / Gene Therapy (AAV) / HematologyHemophilia APhase 3ActiveUS
- ST-503ST-503 / Nav1.7 (SCN9A) / Gene Therapy (Zinc Finger Repressor, AAV9) / NeurologySmall Fiber Neuropathy (Chronic Neuropathic Pain)Phase 1/2ActiveUS
- ST-506ST-506 / Prion protein (PRNP) / Gene Therapy (Zinc Finger Repressor, STAC-BBB AAV) / NeurologyPrion DiseasePreclinicalActiveUS
- TX200TX200 / HLA-A2 / Cell Therapy (Autologous CAR-Treg) / TransplantationPrevention of Immune-Mediated Rejection in HLA-A2 Mismatched Kidney TransplantationPhase 1/2ActiveUSEurope
- IL23R CAR-TregIL23R / Cell Therapy (CAR-Treg) / AutoimmunityInflammatory Bowel Disease (Crohn's Disease)PreclinicalActiveGlobal
- Genentech Tau ProgramTau / Gene Therapy (Zinc Finger Epigenetic Regulator) / NeurologyTauopathies / Alzheimer's DiseasePreclinicalActiveGlobal
- Takeda Huntington's ProgramHTT / Gene Therapy (Zinc Finger Epigenetic Regulator) / NeurologyHuntington's DiseasePreclinicalActiveGlobal
- Alexion C9ORF72 ProgramC9ORF72 / Gene Therapy (Zinc Finger Transcription Factor) / NeurologyAmyotrophic Lateral Sclerosis (ALS)PreclinicalActiveGlobalFrontotemporal Lobar Degeneration (FTLD)PreclinicalActiveGlobal
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- Olanzapine LAITEV-'749
- DuvakitugAnti-TL1A
- DARI (ICS/SABA)TEV-'248
- EmrusolminTEV-'286
- Anti-IL-15TEV-'408
- Anti-PD1-IL2TEV-'278
- Sery433TEV-'2871
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- SuzetrigineVX-548 / Journavx / NaV1.8 / Small Molecule / PainModerate-to-Severe Acute PainPainful Diabetic Peripheral Neuropathy
- VX-993VX-993 / NaV1.8 / Small Molecule / PainPainful Diabetic Peripheral NeuropathyModerate-to-Severe Acute Pain
- Vanzacaftor/tezacaftor/deutivacaftorCFTR / Small Molecule / Cystic FibrosisCystic Fibrosis
- VX-522VX-522 / CFTR / mRNA / Cystic FibrosisCystic Fibrosis (non-responsive to modulators)
- InaxaplinVX-147 / APOL1 / Small Molecule / Kidney DiseaseAPOL1-mediated Kidney Disease (AMKD)
- VX-407VX-407 / PC1 / Small Molecule / Kidney DiseaseAutosomal Dominant Polycystic Kidney Disease (ADPKD)
- VX-880VX-880 / Islet Cell / Cell Therapy / Type 1 DiabetesType 1 Diabetes
- VX-264VX-264 / Islet Cell / Cell Therapy / Type 1 DiabetesType 1 Diabetes
- PovetaciceptALPN-303 / BAFF/APRIL / Biologic / Autoimmune / Kidney DiseaseIgA Nephropathy
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- XmAb819XmAb819 / ENPP3 x CD3 / Bispecific Antibody / OncologyClear Cell Renal Cell Carcinoma (ccRCC)Phase 1ActiveGlobalColorectal Cancer (CRC)Phase 1ActiveGlobalNon-Small Cell Lung Cancer (NSCLC)Phase 1ActiveGlobal
- XmAb541XmAb541 / CLDN6 x CD3 / Bispecific Antibody / OncologyOvarian CancerPhase 1ActiveGlobalGerm Cell Tumors (GCT)Phase 1ActiveGlobalSolid TumorsPhase 1ActiveGlobal
- XmAb808XmAb808 / B7-H3 x CD28 / Bispecific Antibody / OncologyProstate Cancer / Solid TumorsPhase 1ActiveGlobal
- XmAb942XmAb942 / TL1A / Monoclonal Antibody / ImmunologyUlcerative Colitis (UC)Phase 2ActiveGlobalInflammatory Bowel Disease (IBD)Phase 2ActiveGlobal
- XmAb412XmAb412 / TL1A x IL23p19 / Bispecific Antibody / ImmunologyInflammatory Bowel Disease (IBD)Phase 1ActiveGlobal
- PlamotamabCD20 x CD3 / Bispecific Antibody / ImmunologyRheumatoid ArthritisPhase 1ActiveGlobal
- XmAb657XmAb657 / CD19 x CD3 / Bispecific Antibody / ImmunologyIdiopathic Inflammatory MyositisPhase 1ActiveGlobal
- ObexelimabCD19 x FcγRIIb / Monoclonal Antibody / ImmunologyAutoimmune DiseasesPhase 3ActiveGlobal
- TobevibartHepatitis B Virus / Monoclonal Antibody / Infectious DiseaseChronic Hepatitis B / Hepatitis DPhase 3ActiveGlobal