Pipeline Watchlist
Company Pipelines
Companies
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- INO-3107INO-3107 / HPV-6 and HPV-11 / DNA immunotherapy / HPV-related rare diseaseRecurrent Respiratory Papillomatosis (RRP) in adultsBLA accepted under accelerated approval, PDUFA Oct 30 2026United States
- VGX-3100VGX-3100 / HPV-16/18 / DNA immunotherapy / HPV-related diseasesCervical dysplasia (HSIL)3Ongoing (partnered)ChinaAnal dysplasia
- INO-3112INO-3112 / HPV-16/18 / DNA immunotherapy / OncologyHPV16/18-positive oropharyngeal squamous cell carcinoma (OPSCC)3Phase 3 planningUnited StatesEurope
- INO-5401INO-5401 / Tumor-associated antigens / DNA immunotherapy / OncologyGlioblastoma (GBM)1Ongoing Phase 1; Phase 2 planning (as INO-5412 w/ Akeso)United StatesPrevention in BRCA1/2 mutation carriers1Ongoing Phase 1United States
- INO-4201INO-4201 / Ebola virus / DNA medicine / Infectious diseasesEbola vaccine booster (to ERVEBO)2Phase 2 planning; Phase 1b completed
- DMAb SARS-CoV-2SARS-CoV-2 / DNA-encoded monoclonal antibody / Infectious diseasesSARS-CoV-21Ongoing Phase 1; data expected/reportedUnited States
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- PemivibartPEMGARDA / SARS-CoV-2 / Monoclonal Antibody / Infectious DiseasesPre-exposure prophylaxis (PrEP) of COVID-19
- VYD2311VYD2311 / SARS-CoV-2 / Monoclonal Antibody / Infectious DiseasesCOVID-19 prevention
- AdintrevimabSARS-CoV-2 / Monoclonal Antibody / Infectious DiseasesCOVID-19 prevention and treatment
- Measles mAbMeasles (rubeola) virus / Monoclonal Antibody / Infectious DiseasesMeasles treatment and post-exposure prophylaxis
- RSV mAbRespiratory Syncytial Virus (RSV) / Monoclonal Antibody / Infectious DiseasesRSV infection
- Influenza mAbInfluenza virus / Monoclonal Antibody / Infectious DiseasesInfluenza infection
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- MP0712MP0712 / DLL3 / Radio-DARPin Therapy / OncologySmall Cell Lung Cancer (SCLC)Neuroendocrine Tumors
- MP0726MP0726 / MSLN / Radio-DARPin Therapy / OncologyOvarian Cancer
- MP0533MP0533 / CD33 x CD123 x CD70 x CD3 / Multispecific T Cell Engager / OncologyRelapsed/Refractory AMLAML/MDS
- MP0317MP0317 / FAP x CD40 / Tumor-localized CD40 Agonist / OncologyAdvanced Solid TumorsCholangiocarcinoma
- MP0632MP0632 / CD2 x CD3 x MSLN x EpCAM / Switch-DARPin T Cell Engager / OncologyMSLN/EpCAM Expressing Solid TumorsOvarian Cancer
- MP0621MP0621 / cKIT x CD16a x CD47 / Switch-DARPin / HematologyConditioning for Hematopoietic Stem Cell Transplantation
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- DA-1726DA-1726 / GLP1R / GCGR (Glucagon-like peptide-1 receptor and glucagon receptor) / Oxyntomodulin (OXM) analogue / Cardiometabolic DiseasesObesityPhase 1ActiveGlobalMetabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 1ActiveGlobal
- VanoglipelDA-1241 / GPR119 (G-protein-coupled receptor 119) / Small Molecule / Cardiometabolic DiseasesMetabolic Dysfunction-Associated Steatohepatitis (MASH)Phase 2aActiveGlobalType 2 Diabetes (T2D)Phase 1CompletedGlobal
- ANA001ANA001 / Unknown / Oral Niclosamide Formulation / Infectious DiseasesModerate COVID-19Phase 2LegacyUS
- NB-01NB-01 / Unknown / Unknown / NeurologyPainful Diabetic NeuropathyUnknownLegacyGlobal
- NB-02NB-02 / Unknown / Unknown / NeurologyCognitive Impairment / Neurodegenerative DiseasesUnknownLegacyGlobal
- GemcabeneUnknown / Small Molecule / Cardiovascular / Infectious DiseasesDyslipidemiaUnknownLegacyGlobalAcute COVID-19UnknownLegacyGlobal
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- ebopiprantPreterm labor
- OG-6219Endometriosis
- OG-7191PCOS
- Cirqle Biomedical candidateNon-hormonal contraceptive
- Xaciatoclindamycin phosphate vaginal gel
- Jada systemPostpartum hemorrhage device
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- OST-HER2OST31-164 / HER2 / Bioengineered Listeria monocytogenes (Immunotherapy) / OncologyOsteosarcoma (Pulmonary Metastatic)Phase 2bActiveGlobalBreast CancerPhase 1ActiveGlobalCanine OsteosarcomaApprovedMarketedUS
- OST-AXALAXAL / ADXS-HPV / HPV / Listeria-based Immunotherapy / OncologyHPV-associated cancersPhase 3ActiveGlobal
- OST-503ADXS-503 / KRAS G12 position-related antigens / Listeria-based Immunotherapy / OncologyNon-Small Cell Lung Cancer (NSCLC)Phase 2ActiveGlobalGlioblastoma (GBM)Phase 2ActiveGlobalPancreatic CancerPreclinicalActiveGlobal
- OST-504OST-PSA / ADXS-504 / ADXS31142 / PSA / Listeria-based Immunotherapy / OncologyProstate Cancer (Castration-Resistant)Phase 1bActiveGlobal
- OST-tADCVarious / Tunable Antibody-Drug Conjugate (ADC) / OncologyOvarian CancerPreclinicalActiveGlobalBreast CancerPreclinicalActiveGlobal
- OST-HOT constructsShared hotspot mutations and cancer-testes antigens / Listeria-based Immunotherapy / OncologySolid TumorsPreclinicalActiveGlobal
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- AX-0810AX-0810 / NTCP (SLC10A1) / RNA Editing Oligonucleotide (Axiomer EON) / LiverBiliary AtresiaPrimary Sclerosing Cholangitis
- AX-0811AX-0811 / NTCP / RNA Editing Oligonucleotide (Axiomer EON) / LiverCholestatic Diseases
- AX-0422AX-0422 / IDUA / RNA Editing Oligonucleotide (Axiomer EON) / Metabolic / Rare DiseaseHurler Syndrome
- AX-2402AX-2402 / MECP2 (R270X) / RNA Editing Oligonucleotide (Axiomer EON) / CNS / Rare DiseaseRett Syndrome
- AX-2911AX-2911 / PNPLA3 / RNA Editing Oligonucleotide (Axiomer EON) / Liver / MetabolicMASH
- AX-1412AX-1412 / B4GALT1 / RNA Editing Oligonucleotide (Axiomer EON) / CardiovascularCardiovascular Disease
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- rivunatpagene miziparvovecUX701 / ATP7B / AAV9 gene therapy / Inborn Errors of MetabolismWilson disease
- rebisufligene etisparvovecUX111 / AAV gene therapy / NeurogeneticSanfilippo syndrome type A (MPS IIIA)
- avalotcagene ontaparvovecDTX301 / OTC / AAV8 gene therapy / Inborn Errors of MetabolismOrnithine transcarbamylase (OTC) deficiency
- DTX401DTX401 / AAV gene therapy / Inborn Errors of MetabolismGlycogen storage disease type Ia (GSDIa)
- GTX-102GTX-102 / UBE3A / Antisense oligonucleotide (ASO) / NeurogeneticAngelman Syndrome
- setrusumabUX143 / Biologic / Bone-EndocrineOsteogenesis imperfecta
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- DaraxonrasibRMC-6236 / RAS(ON) multi-selective / Small Molecule / OncologyPancreatic Ductal Adenocarcinoma (PDAC)Non-Small Cell Lung Cancer (NSCLC)Colorectal Cancer (CRC)
- ZoldonrasibRMC-9805 / RAS(ON) G12D / Small Molecule / OncologyNon-Small Cell Lung Cancer (NSCLC)Solid Tumors
- ElironrasibRMC-6291 / RAS(ON) G12C / Small Molecule / OncologyKRAS-G12C mutant cancersNon-Small Cell Lung Cancer (NSCLC)Solid Tumors
- RMC-5127RMC-5127 / RAS(ON) G12V / Small Molecule / OncologyRAS-G12V mutant cancers
- RMC-0708RMC-0708 / RAS(ON) Q61H / Small Molecule / OncologyRAS-Q61H mutant cancers
- RMC-8839RMC-8839 / RAS(ON) G13C / Small Molecule / OncologyRAS-G13C mutant cancers
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- SGR-1505SGR-1505 / MALT1 / Small Molecule / OncologyRelapsed/Refractory B-cell MalignanciesPhase 1Active
- SGR-3515SGR-3515 / PKMYT1 / Wee1 / Small Molecule / OncologyAdvanced Solid TumorsPhase 1Active
- SGR-4174SGR-4174 / SOS1 / Small Molecule / OncologyKRAS-driven CancersPreclinicalActive
- SGR-2921SGR-2921 / CDC7 / Small Molecule / OncologyAcute Myeloid Leukemia (AML) / Myelodysplastic Syndrome (MDS)Phase 1Discontinued
- ACCG-2671ACCG-2671 / Calcitonin Receptor (DACRA) / Small Molecule / MetabolicObesityPhase 1Active
- AJ1-11095AJ1-11095 / JAK2 / Small Molecule / HematologyMyelofibrosisPreclinicalActiveUS
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- DivesiranSLN124 / TMPRSS6 / siRNA / Hematologic ConditionsPolycythemia VeraOther Blood Disorders
- ZerlasiranSLN360 / LPA / siRNA / Cardiovascular DiseaseElevated Lp(a)Atherosclerotic Cardiovascular Disease
- SLN312SLN312 / ANGPTL3 / siRNA / CardiometabolicDyslipidemia
- SLN548SLN548 / CFB / siRNA / Complement
- SLN365SLN365 / GPR146 / siRNA / CardiometabolicHomozygous familial hypercholesterolemia (HoFH)
- SLN098SLN098 / INHBE / siRNA / MetabolicObesity
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- AmezalpatTPST-1120 / PPARα antagonist / Small Molecule / OncologyHepatocellular Carcinoma (HCC)CholangiocarcinomaRenal Cell Carcinoma (RCC)
- TPST-1495TPST-1495 / Dual EP2/EP4 Antagonist / Small Molecule / Oncology / GastrointestinalFamilial Adenomatous Polyposis (FAP)Solid Tumors
- TREX-1 InhibitorTREX-1 / Small Molecule / OncologySolid Tumors
- TPST-2003TPST-2003 / CD19/BCMA / Autologous CAR-T Cell Therapy / OncologyRelapsed/Refractory Multiple Myeloma (rrMM)
- TPST-3003TPST-3003 / CD19/BCMA / Allogeneic Cell Therapy / OncologyRelapsed/Refractory Multiple Myeloma (rrMM)
- TPST-4003TPST-4003 / CD19/BCMA / In Vivo Cell Therapy / Immunology / AutoimmuneSystemic Lupus Erythematosus (SLE)Immunology Disorders
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- TreprostinilTyvaso / Prostacyclin receptor / Small Molecule / RespiratoryIdiopathic Pulmonary Fibrosis (IPF)
- RalinepagProstacyclin receptor / Small Molecule / CardiopulmonaryPulmonary Arterial Hypertension (PAH)
- UKidneyUKidney / Xenotransplantation / Organ TransplantationEnd Stage Renal Disease
- UThymoKidneyUThymoKidney / Xenotransplantation / Organ TransplantationEnd Stage Renal Disease
- miroliverELAPmiroliverELAP / Regenerative Medicine / Organ TransplantationAcute Liver Failure
- Centralized Lung Evaluation System (CLES)Ex-vivo Perfusion System / Organ TransplantationLung Transplant
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- Chikungunya vaccineVLA1553 / IXCHIQ
- Lyme Disease vaccineVLA15
- Shigella vaccineS4V2
- Zika vaccineVLA1601
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- ElebsiranVIR-2218 / HBV / siRNA / Infectious DiseasesChronic Hepatitis Delta (CHD)Phase 3ActiveGlobalChronic Hepatitis B (CHB)Phase 2ActiveGlobal
- TobevibartVIR-3434 / HBV / Monoclonal Antibody / Infectious DiseasesChronic Hepatitis Delta (CHD)Phase 3ActiveGlobalChronic Hepatitis B (CHB)Phase 2ActiveGlobal
- VIR-1388VIR-1388 / HIV / T Cell Vaccine / Infectious DiseasesHIV PreventionPhase 1ActiveUnited StatesSouth Africa
- VIR-5500VIR-5500 / PSMA / Masked T-Cell Engager / OncologySolid TumorsPhase 1ActiveGlobal
- VIR-5525VIR-5525 / EGFR / Masked T-Cell Engager / OncologySolid TumorsPhase 1ActiveGlobal
- VIR-5818VIR-5818 / HER2 / Masked T-Cell Engager / OncologySolid TumorsPhase 1ActiveGlobal
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- VK2735 (Subcutaneous)VK2735 / Dual GLP-1/GIP receptor agonist / Peptide / Metabolic DisordersObesity
- VK2735 (Oral)VK2735 / Dual GLP-1/GIP receptor agonist / Peptide / Metabolic DisordersObesity
- VK2809VK2809 / Thyroid hormone receptor beta (TRβ) agonist / Small molecule / Metabolic DisordersNASHFibrosisNAFLD
- VK0214VK0214 / Thyroid hormone receptor beta (TRβ) agonist / Small molecule / Rare DiseasesX-linked adrenoleukodystrophy (X-ALD)
- Amylin Agonist (DACRAs)Dual amylin and calcitonin receptor agonist (DACRA) / Agonist / Metabolic DisordersObesityMetabolic disorders
- VK5211VK5211 / Selective androgen receptor modulator (SARM) / Small molecule / Musculoskeletal DisordersHip fracture recoveryMuscle and bone disorders
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- fasedienolPH94B / peripheral nasal chemosensory receptors / Intranasal pherine / PsychiatrySocial Anxiety Disorder (SAD)
- itruvonePH10 / peripheral nasal chemosensory receptors / Intranasal pherine / PsychiatryMajor Depressive Disorder (MDD)
- refisolonePH80 / peripheral nasal chemosensory receptors / Intranasal pherine / Women's HealthMenopausal Vasomotor SymptomsPremenstrual Dysphoric Disorder (PMDD)
- PH15PH15 / peripheral nasal chemosensory receptors / Intranasal pherine / Neurology/PsychiatryPsychomotor and cognitive impairment due to mental fatigue
- PH284PH284 / peripheral nasal chemosensory receptors / Intranasal pherine / Oncology Supportive CareCancer Cachexia
- AV-101AV-101 / NMDA receptor glycine site / Small molecule / Neurology/PsychiatryNeurological disorders
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- VilastobartXTX101 / CTLA-4 / Monoclonal Antibody / OncologyAdvanced solid tumors / MSS mCRCPhase 2ActiveGlobal
- Efarindodekin alfaXTX301 / IL-12 / Cytokine / OncologyAdvanced solid tumorsPhase 2ActiveGlobal
- XTX501XTX501 / PD-1 / IL-2 / Bispecific Antibody / Cytokine / OncologyNon-small cell lung cancer (NSCLC) and other solid tumorsPreclinicalActiveGlobal
- PSMA+STEAP1 Masked T Cell EngagerPSMA / STEAP1 / T Cell Engager / OncologyProstate cancerPreclinicalActiveGlobal
- CLDN18.2 Masked T Cell EngagerCLDN18.2 / T Cell Engager / OncologyGastric, pancreatic, esophageal, and lung cancersPreclinicalActiveGlobal
- Undisclosed Masked T Cell EngagerUndisclosed / T Cell Engager / OncologyCancerPreclinicalActiveGlobal
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- BosakitugATI-045 / TSLP / humanized anti-TSLP monoclonal antibody / immuno-inflammatory; respiratory; dermatologyatopic dermatitisPhase 2ongoing
- ATI-052ATI-052 / TSLP; IL-4Rα / humanized bispecific antibody / immuno-inflammatory; respiratory; dermatologyatopic dermatitisPhase 1bongoingasthmaPhase 1bongoingasthmaPhase 2bplanned
- ATI-2138ATI-2138 / ITK; JAK3 / oral covalent small-molecule inhibitor / T cell-mediated autoimmune diseases; dermatologylichen planusPhase 2bplannedatopic dermatitisPhase 2acompleted
- ATI-9494ATI-9494 / ITK; TXK/RLK / oral covalent small-molecule inhibitor / Th1, Th2 and Th17-driven diseasesundisclosed inflammatory diseasespreclinicalIND planned
- LepzacitinibATI-1777 / JAK1; JAK3 / topical soft small-molecule inhibitor / dermatologyatopic dermatitisPhase 2bseeking partner
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- TanruprubartANX005 / C1q / classical complement pathway / IV mAb / Autoimmune / neurologyGuillain-Barré SyndromeMAA filed; FDA discussions ongoingactiveUSEU
- VonaprumentANX007 / C1q / classical complement pathway / IVT Fab / OphthalmologyGeographic AtrophyPhase 3ARCHER II enrollment completed; data expected 2H26
- ANX1502ANX1502 / Classical complement pathway / Oral small molecule / AutoimmuneCold Agglutinin DiseaseProof-of-conceptactive; update upon CAD study completion in 2026
- ANX005ANX005 / C1q / classical complement pathway / IV mAb / NeurodegenerationHuntington’s DiseasePoised for late-stage Phase 2b/3activeAmyotrophic Lateral SclerosisPoised for late-stage Phase 2b/3active
- ANX009ANX009 / C1q / classical complement pathway / Subcutaneous Fab / AutoimmuneLupus NephritisEvaluating options for future development
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- TransCon hGHSKYTROFA / growth hormone / TransCon prodrug therapy / Endocrinology Rare DiseasesTurner syndrome (children aged 1–10)Phase 2United StatesMulti-Indication: Turner syndrome, ISS, SHOX Deficiency, SGA (children aged 2–17)Phase 3MultinationalPediatric GHDPhase 3Japan
- TransCon CNPC-type natriuretic peptide / TransCon prodrug therapy / Endocrinology Rare DiseasesAchondroplasia (infants aged 0–<2)Pivotal Phase 2MultinationalAchondroplasia (children aged 2–11)Pivotal Phase 2MultinationalAchondroplasia (adolescents aged 12–18)Pivotal Phase 2bMultinational
- TransCon CNP + TransCon hGHCNP; growth hormone / TransCon prodrug therapy combination / Endocrinology Rare DiseasesAchondroplasia (children aged 2–11)Phase 2Multinational
- TransCon PTHYORVIPATH / parathyroid hormone / TransCon prodrug therapy / Endocrinology Rare DiseasesAdult Hypoparathyroidism (dosing expansion)Phase 3United StatesAdult HypoparathyroidismPhase 3China
- TransCon IL-2 β/γ (Systemic)IL-2 β/γ / TransCon prodrug therapy / OncologyVarious tumor typesPhase 2Multinational
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- FirmonertinibEGFR TKI / oral small molecule / Oncology1L NSCLC EGFR exon 20 insertion mutationsPhase 3Monotherapyglobal1L+ NSCLC EGFR PACC mutationsPhase 3MonotherapyglobalAdjuvant EGFR uncommon mutationsPhase 3Monotherapy
- ARR-217ARR-217 / CDH17 ADC / antibody-drug conjugate / OncologyGI tumorsPreclinical
- ARR-002ARR-002 / antibody-drug conjugate / OncologySolid tumorsDiscovery
- ARR-421ARR-421 / antibody-drug conjugate / OncologySolid tumorsDiscovery
- ARR-173ARR-173 / antibody-drug conjugate / OncologySolid tumorsDiscovery
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- AcoramidisAttruby / TTR (Transthyretin) / Small Molecule / Genetic diseaseEarly-Stage Variant Transthyretin Amyloidosis (ATTRv)Phase 3Under investigation
- BBP-418BBP-418 / FKRP / Small Molecule / Genetic disease; neuromuscularLimb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)Phase 3Investigational; FORTIFY interim analysis met endpoints
- EncaleretCASR / Small Molecule / Endocrine; genetic diseaseAutosomal Dominant Hypocalcemia Type 1 (ADH1)Phase 3NDA planned/submission stated for H1 2026; pediatric registrational trial initiatedUSEUChronic HypoparathyroidismPhase 2NIH investigator-initiated study; Phase 3 planned in 2026
- InfigratinibFGFR3 / Small Molecule / Skeletal dysplasia; genetic diseaseAchondroplasiaPhase 3PROPEL 3 registrational study; infant/toddler study enrollingUSEUHypochondroplasiaPhase 2ACCEL 2/3 Phase 2 portion dosed; data expected H2 2026
- BBP-812BBP-812 / ASPA / Gene Therapy / Genetic disease; neurologyCanavan DiseaseInvestigational AAV gene therapy
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- ORLADEYO (berotralstat)ORLADEYO / plasma kallikrein / oral small-molecule inhibitor / Rare diseasesHereditary angioedema (HAE) prophylaxisApproved/Commercialmarketed
- ORLADEYO (berotralstat) in PediatricsORLADEYO / plasma kallikrein / oral small-molecule inhibitor / Rare diseasesHereditary angioedema (HAE) prophylaxis, ages 2 to <12Approved/Commercialmarketed
- NavenibartSTAR-0215 / plasma kallikrein / monoclonal antibody inhibitor / Rare diseasesHereditary angioedema (HAE) prophylaxisPhase 3/Pivotalinvestigational
- BCX17725BCX17725 / KLK5 / protein therapeutic / Rare diseasesNetherton syndromePhase 1investigational
- STAR-0310STAR-0310 / OX40 / monoclonal antibody antagonist / Immunology/DermatologyAtopic dermatitisPreclinicalinvestigational; strategic alternatives planned
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- nuzefatide pevedotinBT5528 / EphA2 / Bicycle Drug Conjugate / OncologyHigh EphA2-expressing tumorsPhase I/IIOngoingMetastatic pancreatic ductal adenocarcinomaPhase IIOngoing
- zelenectide pevedotinBT8009 / Nectin-4 / Bicycle Drug Conjugate / OncologyNectin-4-expressing tumorsPhase I/IIOngoing Duravelo-1Metastatic urothelial cancerPhase IIOngoing Duravelo-2
- BT7480BT7480 / Nectin-4/CD137 / Bicycle TICA / OncologyAdvanced solid tumors associated with Nectin-4 expressionPhase I/IIOngoing; seeking partner for future development
- BT1702BT1702 / MT1-MMP / Bicycle Radioligand / OncologyMT1-MMP-expressing tumorsIND-enabling / human imagingCompany-sponsored BRC clinical trial planned in 2027
- ION286/AZD4063ION286/AZD4063 / TfR1 / TfR1-targeted Bicycle conjugate / CardiomyopathyCardiomyopathyPhase 1Ongoing
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- BMN 333BMN 333 / C-type natriuretic peptide pathway / Long-acting CNP peptide / Skeletal ConditionsAchondroplasiaPhase 2/3Registration-enabling study enrolling
- BMN 351BMN 351 / Dystrophin / Oligonucleotide / InnovationDuchenne muscular dystrophyPhase 1/2Clinical data update expected year-end 2026
- BMN 401BMN 401 / ENPP1 / Subcutaneous enzyme replacement therapy / Enzyme TherapiesENPP1 deficiencyPhase 3ENERGY 3 met 1 of 2 co-primary endpoints; next steps under evaluation
- vosoritideVOXZOGO / CNP receptor/NPR-B pathway / Peptide / Skeletal ConditionsHypochondroplasiaPhase 3Positive pivotal data; sNDA planned Q3 2026USEUAchondroplasiasNDAFull approval sNDA submitted April 2026USHypochondroplasia under 3 years oldPhase 2Enrollment progressing
- pegvaliase-pqpzPALYNZIQ / Phenylalanine ammonia lyase / Enzyme therapy / Enzyme TherapiesPhenylketonuria adolescents 12+RegulatoryFDA approved February 2026; EU approval expected 2026USEU
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- BDC-4182BDC-4182 / Claudin 18.2 / Boltbody ISAC / OncologyGastric cancerPhase 1Ongoing dose-escalationGastroesophageal cancerPhase 1Ongoing dose-escalation
- BDC-3042BDC-3042 / Dectin-2 / Agonist monoclonal antibody / OncologyNon-small cell lung cancer, melanoma and other solid tumorsPhase 1Dose-escalation complete; seeking partner
- CEA ISACCEA/CEACAM5 / Boltbody ISAC / OncologyColorectal, non-small cell lung, pancreatic and gastric cancersPreclinicalPre-IND; seeking partner
- PD-L1 ISACPD-L1 / Boltbody ISAC / OncologySolid tumors resistant to checkpoint inhibitorsPreclinicalPre-IND; seeking partner
- Caprin-1 ISACCaprin-1 / Boltbody ISAC / Oncology
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- Lasme-celCD22 / allogeneic CAR-T / Oncologyrelapsed/refractory B-cell acute lymphoblastic leukemiaPhase 2BALLI-01; pivotal Phase 2 first interim analysis expected Q4 2026
- Eti-celCD20, CD22 / dual allogeneic CAR-T / Oncologyrelapsed/refractory Non-Hodgkin lymphomaPhase 1NatHaLi-01; full Phase 1 dataset expected Q4 2026
- Cemacabtagene ansegedleucelCema-cel / CD19 / allogeneic CAR-T / Oncologylarge B-cell lymphomaPivotal Phase 2ALPHA3
- ALLO-316ALLO-316 / CD70 / allogeneic CAR-T / Oncologyadvanced or metastatic clear cell renal cell carcinomaPhase 1TRAVERSE
- IOV-4001IOV-4001 / PD-1 / TALEN-modified tumor-infiltrating lymphocyte therapy / Oncologyunresectable or metastatic melanomaIOV-GM1-201stage III or IV NSCLCIOV-GM1-201
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- CLS-AX (axitinib)CLS-AX / VEGF receptors-1, -2 and -3 / Small molecule tyrosine kinase inhibitor / OphthalmologyWet AMDPhase 3 readyFDA end-of-Phase 2 alignment
- XIPERE (triamcinolone acetonide injectable suspension)XIPERE; ARCATUS / Corticosteroid injectable suspension / OphthalmologyUveitic macular edemaApproved/commercialUnited StatesAustraliaSingaporeUveitic macular edemaClinical development/commercialization partnerChinaDiabetic macular edemaClinical development/commercialization partnerAsia Pacific ex-Japan
- Bel-SarViral-like drug conjugate / Ophthalmology/oncologyChoroidal melanomaPartner clinical development program
- ABBV-RGX-314ABBV-RGX-314 / anti-VEGF / AAV gene therapy / OphthalmologyDiabetic retinopathyPartner clinical development programWet AMDPartner clinical development program
- AvoralstatPlasma kallikrein / Small molecule plasma kallikrein inhibitor / OphthalmologyDiabetic macular edemaPartner clinical development program
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- bezuclastinibCGT9486 / KIT exon 17/KIT D816V / small molecule tyrosine kinase inhibitor / Oncology; hematologyNonAdvanced Systemic MastocytosisNDA acceptedFDA review; PDUFA December 30, 2026USGastrointestinal Stromal Tumors after prior imatinibNDA submittedFDA reviewUSAdvanced Systemic Mastocytosisregistration-directed trial completedNDA planned 1H 2026US
- CGT4255CGT4255 / mutant ErbB2/HER2 / CNS-penetrant small molecule inhibitor / OncologyAdvanced solid tumors with HER2 mutations or HER2 overexpressionPhase 1dose escalation ongoing
- CGT6297CGT6297 / PI3Kα / small molecule inhibitor / OncologyPI3Kα-mutant cancersPhase 1initiated
- CGT1815CGT1815 / pan-KRAS(ON) / small molecule inhibitor / Oncology
- CGT1145CGT1145 / JAK2 V617F / small molecule inhibitor / Hematology
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- COYA 302COYA 302 / Treg enhancement; T effector and macrophage depletion / Treg-enhancing/T effector and macrophage-depleting biologic combination; low-dose IL-2 + CTLA4-Ig / Neurodegenerative diseasesAmyotrophic lateral sclerosisPhase 2Fast Track; ALSTARS Phase 2 enrollingUSCanadaFrontotemporal dementiaIND acceptedactiveUSAlzheimer's diseasePreclinical/POCactive
- COYA 301COYA 301 / IL-2; Treg function and expansion / Proprietary low-dose IL-2 biologic / Neurodegenerative diseasesAlzheimer's diseaseIND-enablingactive
- COYA 303COYA 303 / IL-2; GLP-1 receptor / COYA 301 + GLP-1 receptor agonist combination / Inflammatory diseasesUndisclosedDiscoveryactive
- COYA 201COYA 201 / Allogeneic Treg-derived exosomes / UndisclosedUndisclosedPreclinicalactive
- COYA 206COYA 206 / Antigen-directed allogeneic Treg-derived exosomes / UndisclosedUndisclosedPreclinicalactive
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- EB613EB613 / PTH 1-34, teriparatide / oral peptide tablet / OsteoporosisPostmenopausal women with low BMD and high-risk osteoporosisPhase 3Phase 3 protocol submitted to FDA; registrational program plannedU.S.
- EB612EB612 / LA-PTH 1-34 / oral long-acting PTH analog peptide tablet / HypoparathyroidismHypoparathyroidismPreclinicalDevelopment ongoing; pre-IND initiatives underway
- OXMOPK-88006 / GLP-1 & glucagon agonist / oral oxyntomodulin peptide tablet / Obesity / metabolic diseasesObesity and metabolic diseasesPreclinicalIND filing expected late H1 2026U.S.
- GLP-2Long-acting GLP-2 / oral GLP-2 peptide tablet / Short bowel syndromeShort bowel syndromePreclinicalProof-of-concept single-dose pharmacokinetic study completed in rodents
- EB613EB613 / PTH 1-34, teriparatide / oral peptide tablet / Stress fracturesStress fracturesInvestigator Sponsored Trial
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- HydronidoneF351 / TGFβRI/p38γ (anti-fibrotic) / Small molecule / FibrosisCHB-associated liver fibrosisNDA (priority review)NDA submission for conditional approval expected 1H 2026; Breakthrough Therapy designation (NMPA, Mar 2021)ChinaMASH-associated liver fibrosisIND-enablingU.S. IND application anticipated in 2026United States
- PirfenidoneETUARY / Anti-fibrotic / Small molecule / Fibrosis/RespiratoryIdiopathic pulmonary fibrosis (IPF)Approved/MarketedApproved in China (2011); commercializedChinaPneumoconiosis (PD)Phase 352-week Phase 3 trial enrollment completed (272 patients, 18 sites)ChinaRadiation-induced lung injury (RILI) with or without immune-related pneumonitisClinicalIn developmentChina
- F573F573 / Caspase inhibitor / Small molecule / HepatologyAcute/acute-on-chronic liver failure (ALF/ACLF)ClinicalIn developmentChina
- F528F528 / Small molecule / RespiratoryChronic obstructive pulmonary disease (COPD)PreclinicalIn developmentChina
- F230F230 / Small molecule / CardiopulmonaryPulmonary arterial hypertension (PAH)PreclinicalIn developmentChina
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- CrofelemerMytesi / CFTR and CaCC channels / Botanical Drug / GastrointestinalHIV-Related DiarrheaApprovedMarketedUnited StatesCancer Therapy-related Diarrhea (CTD) / Chemotherapy-induced overactive bowelPhase 3ActiveUnited StatesIrritable Bowel SyndromeIn DevelopmentActiveUnited States
- Crofelemer Powder for Oral SolutionCFTR and CaCC channels / Botanical Drug / Gastrointestinal / Rare DiseaseMicrovillus Inclusion Disease (MVID)Late StageActiveUnited StatesEuropeShort Bowel Syndrome with Intestinal Failure (SBS-IF)Late StageActiveUnited StatesEurope
- NP-300NP-300 / Unknown / Botanical Drug / Gastrointestinal / Infectious DiseaseCholera Diarrhea / Infectious DiarrheaIn DevelopmentActiveGlobal
- GelclairGelclair / Unknown / Bioadherent Oral Gel / Supportive Care / OncologyOral MucositisApprovedMarketedUnited States
- Crofelemer (Animal Health)Canalevia-CA1 / CFTR and CaCC channels / Botanical Drug / Animal HealthChemotherapy-induced diarrhea in dogsApprovedMarketedUnited States
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- KRRO-121KRRO-121 / Glutamine Synthetase (GS) / GalNAc-conjugated oligonucleotide (RNA Editing) / Metabolic / LiverUrea Cycle Disorders (UCD) / Hepatic Encephalopathy (HE)PreclinicalActiveGlobal
- AATD GalNAc ProgramSERPINA1 mRNA / GalNAc-conjugated oligonucleotide (RNA Editing) / Rare Diseases / Respiratory / LiverAlpha-1 Antitrypsin Deficiency (AATD)PreclinicalActiveGlobal
- AMPKγ1 ProgramAMPKγ1 / GalNAc-conjugated oligonucleotide (RNA Editing) / Metabolic / LiverLiver Function / LongevityPreclinicalActiveGlobal
- ALS ProgramTDP-43 mRNA / Oligonucleotide (RNA Editing) / NeurologyAmyotrophic Lateral Sclerosis (ALS)PreclinicalActiveGlobal
- Cardiometabolic ProgramUndisclosed / Oligonucleotide (RNA Editing) / Metabolic / CardiovascularCardiometabolic DiseasePreclinicalActiveGlobal
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- ziftomenibKOMZIFTI / Menin / Small Molecule / OncologyAcute Myeloid Leukemia (AML) (Combination)Phase 3ActiveGlobalRelapsed or refractory (R/R) NPM1-mutant AMLPre-RegistrationNDA Submitted / Priority ReviewUS
- rocatinlimabKHK4083 / AMG 451 / OX40 / Monoclonal Antibody / Immunology/AllergyModerate to Severe Atopic DermatitisPhase 3ActiveGlobalUSEUJPPrurigo NodularisPhase 3ActiveGlobalAsthmaPhase 2ActiveGlobal
- atidarsagene autotemcelOTL-200 / Lenmeldy / Libmeldy / ARSA / Hematopoietic Stem Cell (HSC) Gene Therapy / Rare DiseasesEarly-onset Metachromatic Leukodystrophy (MLD)Pre-RegistrationPreparation underway for clinical trial / Regulatory ReviewJP
- tivozanibKHK4951 / VEGF Receptor Tyrosine Kinase / Small Molecule / OphthalmicOphthalmic DiseasesPhase 2/3ActiveGlobal
- mogamulizumabKW-0761 / POTELIGEO / CCR4 / Monoclonal Antibody / OncologyMycosis fungoides and Sézary syndromePre-RegistrationFiledCanada
5assets
5assets
- sparsentansparsentan / Filspari / ETA/AT1 receptors / Small molecule / NephrologyIgA Nephropathy
- ensifentrineensifentrine / Ohtuvayre / PDE3/PDE4 / Small molecule / RespiratoryCOPD
- pneumococcal 15-valent conjugate vaccineV114 / Vaxneuvance / Pneumococcal serotypes / Vaccine / Infectious DiseasePneumococcal disease
- carfilzomibcarfilzomib / Kyprolis / Proteasome / Small molecule / OncologyMultiple Myeloma
- berdazimerberdazimer / Zelsuvmi / Nitric oxide / Small molecule / DermatologyMolluscum contagiosum
5assets
- CertepetideLSTA1 / alpha-v integrins / Cyclic Peptide / OncologyAdvanced Solid TumorsPancreatic Ductal Adenocarcinoma (PDAC)Cholangiocarcinoma (CCA)
- LSTA12LSTA12 / HONEDRA / CD34+ / Cell Therapy / CardiovascularCritical Limb Ischemia (CLI)
- LSTA14LSTA14 / OLOGO / CD34+ / Cell Therapy / CardiovascularNo-Option Refractory Disabling Angina (NORDA)
- XOWNAXOWNA / CD34+ / Cell Therapy / CardiovascularCoronary Microvascular Dysfunction (CMD)
- LSTA201LSTA201 / CD34+ / Cell Therapy / NephrologyDiabetic Kidney Disease (DKD)
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- LP-300LP-300 / PTPN11 / Small molecule / OncologyNon-small cell lung cancer (NSCLC) in never-smokers
- LP-184LP-184 / PTGR1-activated alkylating agent / Small molecule / OncologySolid tumorsCNS cancersGlioblastoma multiforme (GBM)
- LP-284LP-284 / PKC-beta / Small molecule / OncologyHematologic malignanciesAggressive B-cell lymphomas
- STAR-001STAR-001 / Unknown / Small molecule / OncologyCNS cancersBrain metastases
- ADC ProgramUnknown / Antibody-drug conjugate (ADC) / OncologyCancer
5assets
5assets
- CanvuparatideMBX 2109
- ImapextideMBX 1416