Pipeline Watchlist
Company Pipelines
Companies
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- MGC026B7-H3 ADC
- MGC028ADAM9 ADC
- MGC030TOP1i-based ADC
- lorigerlimabPD-1 x CTLA-4 bispecific DART
- MGD024CD123 x CD3 DART
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- MaralixibatLIVMARLI / IBAT / Small molecule / Rare Liver DiseasesAlagille syndrome (ALGS)Progressive familial intrahepatic cholestasis (PFIC)Biliary atresia
- VolixibatSHP626 / IBAT / Small molecule / Rare Liver DiseasesPrimary sclerosing cholangitis (PSC)Primary biliary cholangitis (PBC)
- MRM-3379MRM-3379 / PDE4D / Small molecule / Neurocognitive DisordersFragile X syndrome
- Cholic acidCHOLBAM / Bile acid replacement / Small molecule / Rare Liver DiseasesBile acid synthesis disordersPeroxisomal disorders (Zellweger spectrum disorders)
- ChenodiolCTEXLI / Bile acid replacement / Small molecule / Rare Genetic DisordersCerebrotendinous xanthomatosis (CTX)
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- ALXN1840ALXN1840 / Copper / Small Molecule / Rare DiseasesWilson Disease
- MNPR-101-ZrMNPR-101-Zr / uPAR / Radiopharmaceutical / OncologyAdvanced Solid Cancers (Imaging)
- MNPR-101-LuMNPR-101-Lu / uPAR / Radiopharmaceutical / OncologyAdvanced Solid Cancers
- MNPR-101-AcMNPR-101-Ac / uPAR / Radiopharmaceutical / OncologyAdvanced Solid Cancers
- MNPR-101MNPR-101 / uPAR / Monoclonal Antibody / OncologySolid Tumors
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- PetosemtamabMCLA-158 / EGFR x LGR5 / Bispecific Antibody / Oncology1L PD-L1+ r/m head and neck squamous cell carcinoma (with pembrolizumab)Phase 3OngoingGlobal2/3L r/m head and neck squamous cell carcinomaPhase 3OngoingGlobal1L metastatic colorectal cancer (with standard chemotherapy)Phase 2OngoingGlobal
- ZenocutuzumabMCLA-128 / BIZENGRI / HER2 x HER3 / Bispecific Antibody / OncologyPancreatic adenocarcinoma (NRG1 fusion-positive)ApprovedApprovedUnited StatesNon-small cell lung cancer (NRG1 fusion-positive)ApprovedApprovedUnited StatesOther non-NRG1+ cancerPhase 2OngoingGlobal
- MCLA-129MCLA-129 / EGFR x c-MET / Bispecific Antibody / OncologySolid tumorsPhase 1/Phase 2OngoingGlobal2L+ EGFRm non-small cell lung cancer (with chemotherapy)Phase 2OngoingGlobal
- BesufetamigONO-4685 / PD-1 x CD3 / Bispecific Antibody / Oncology, ImmunologyRelapsed/Refractory T Cell LymphomaPhase 1OngoingGlobalPsoriasisPhase 1OngoingGlobal
- INCA33890INCA33890 / PD-1 x TGFBr2 / Bispecific Antibody / OncologySelect advanced solid tumorsPhase 1OngoingGlobal
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- rezpegaldesleukinNKTR-358 / REZPEG
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- navacaprantNMRA-140 / Kappa opioid receptor (KOR) antagonist / Small molecule / NeuropsychiatryMajor Depressive Disorder
- NMRA-511NMRA-511 / Vasopressin 1a receptor (V1aR) antagonist / Small molecule / Neuropsychiatry / NeurodegenerativeAlzheimer's disease agitation
- NMRA-215NMRA-215 / NLRP3 inhibitor / Small molecule / Endocrinology / MetabolicObesity
- NMRA-861NMRA-861 / M4 muscarinic receptor positive allosteric modulator (M4 PAM) / Small molecule / NeuropsychiatrySchizophreniaNeuropsychiatric disorders
- NMRA-898NMRA-898 / M4 muscarinic receptor positive allosteric modulator (M4 PAM) / Small molecule / NeuropsychiatrySchizophreniaNeuropsychiatric disorders
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- triamcinolone acetonideZILRETTA / Small Molecule Formulation / Musculoskeletal PainShoulder Osteoarthritis (OA)Phase 3Active
- iovera°iovera° / Device / Neurology/PainSpasticityClinicalActive
- bupivacaine liposomeEXPAREL / Small Molecule Formulation / Pain ManagementAcute Post-Surgical Pain (Pediatric 0 to < 6)ClinicalActive
- enekinragene inzadenovecPCRX-201 / IL-1Ra / Gene Therapy / Musculoskeletal PainOsteoarthritis of the Knee (OAK)Phase 2Active
- PCRX-2002PCRX-2002 / Unknown / Pain ManagementPost-Surgical PainClinicalActive
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- VAX-31VAX-31 / Streptococcus pneumoniae (31 Serotypes) / Pneumococcal Conjugate Vaccine (PCV) / Infectious DiseasesInvasive Pneumococcal Disease (IPD) and Pneumonia in AdultsPhase 3OngoingUnited StatesInvasive Pneumococcal Disease (IPD) in InfantsPhase 2OngoingUnited States
- VAX-24VAX-24 / Streptococcus pneumoniae (24 Serotypes) / Pneumococcal Conjugate Vaccine (PCV) / Infectious DiseasesInvasive Pneumococcal Disease (IPD) in InfantsPhase 2OngoingUnited States
- VAX-A1VAX-A1 / Group A Streptococcus / Conjugate Vaccine / Infectious DiseasesGroup A Strep InfectionsPreclinicalOngoing
- VAX-GIVAX-GI / Shigella / Vaccine / Infectious DiseasesShigellosis / Shigella InfectionsPreclinicalOngoing
- VAX-XLVAX-XL / Streptococcus pneumoniae / Pneumococcal Conjugate Vaccine (PCV) / Infectious DiseasesInvasive Pneumococcal Disease (IPD)PreclinicalOngoing
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- PAPZIMEOSzopapogene imadenovec-drba
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- PMN310PMN310 / Amyloid-Beta / Monoclonal Antibody / NeurologyAlzheimer's Disease
- PMN267PMN267 / TDP-43 / Monoclonal Antibody / NeurologyAmyotrophic Lateral Sclerosis (ALS)Frontotemporal Dementia (FTD)Limbic-predominant age-related TDP-43 encephalopathy (LATE)
- PMN442PMN442 / Alpha-Synuclein / Monoclonal Antibody / NeurologyParkinson's Disease (PD)Multiple System Atrophy (MSA)Dementia with Lewy Bodies (DLB)
- PMN311PMN311 / Amyloid-Beta / Vaccine / NeurologyAlzheimer's Disease
- PMN440PMN440 / Alpha-Synuclein / Vaccine / NeurologyParkinson's Disease
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- deupirfenidoneLYT-100 / Small Molecule / Respiratory / Fibrotic DiseasesIdiopathic Pulmonary Fibrosis (IPF)Phase 3ActiveGlobal
- LYT-200LYT-200 / Biologic / OncologyRelapsed/Refractory High-Risk Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)Phase 1ActiveGlobalSolid TumorsPhase 1ActiveGlobal
- GlyphAlloSPT-300 / Allopregnanolone / Small Molecule / NeuropsychiatryAnxiety and Mood DisordersPhase 2ActiveGlobal
- GlyphAgoSPT-320 / Small Molecule / NeuropsychiatryNeurological / Neuropsychiatric IndicationsPhase 1ActiveGlobal
- xanomeline and trospium chlorideKarXT / Cobenfy / Muscarinic Receptors / Small Molecule / NeuropsychiatrySchizophreniaApprovedActiveUS
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- AMT-130AMT-130 / Huntingtin protein (mHTT) / Gene Therapy (AAV5) / Central Nervous SystemHuntington's DiseasePhase 1/2ActiveGlobal
- AMT-191AMT-191 / Alpha-galactosidase A (GLA) / Gene Therapy / Rare DiseasesFabry DiseasePhase 1/2aActiveGlobal
- AMT-260AMT-260 / Undisclosed / Gene Therapy / Central Nervous SystemRefractory Mesial Temporal Lobe EpilepsyPhase 1/2aActiveGlobal
- AMT-162AMT-162 / SOD1 / Gene Therapy / Central Nervous SystemAmyotrophic Lateral Sclerosis (ALS)Preclinical/ClinicalActiveGlobal
- etranacogene dezaparvovecAMT-061 / HEMGENIX / Factor IX / Gene Therapy (AAV5) / HematologyHemophilia BApprovedMarketedUSGlobal
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- BrepocitinibJAK1 / TYK2 / Small Molecule / Autoimmune & InflammatoryDermatomyositisPhase 3ActiveGlobalNon-infectious UveitisPhase 3ActiveGlobalCutaneous SarcoidosisPhase 2ActiveGlobal
- IMVT-1402IMVT-1402 / FcRn / Monoclonal Antibody / AutoimmuneRheumatoid Arthritis (difficult-to-treat)Phase 2ActiveGlobalGraves' DiseasePhase 2/3ActiveGlobal
- BatoclimabFcRn / Monoclonal Antibody / AutoimmuneThyroid Eye Disease (TED)Phase 3ActiveGlobalMyasthenia Gravis (MG)Phase 3ActiveGlobalChronic Inflammatory Demyelinating Polyneuropathy (CIDP)Phase 2bActiveGlobal
- MosliciguatRVT-304 / sGC / Small Molecule / PulmonaryPulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)Phase 2ActiveGlobal
- NamilumabGM-CSF / Monoclonal Antibody / Autoimmune & InflammatorySarcoidosisPhase 2ActiveGlobal
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- RopidoxuridineIPdR / Radiation Sensitizer / Small Molecule / OncologyGlioblastomaPhase 2ActiveUnited StatesSarcomasPhase 1/Phase 2ActiveUnited States
- SP-2-225SP-2-225 / HDAC6 / Small Molecule / OncologyBreast Cancer / Solid TumorsPreclinicalActiveUnited States
- SP-1-303SP-1-303 / Class I HDAC / Small Molecule / OncologyER+ Breast CancerPreclinicalActiveUnited States
- SP-1-161SP-1-161 / HDAC / Small Molecule / OncologySolid TumorsDiscoveryActiveUnited States
- PSMA-B ligandPSMA-B / PSMA / Radiopharmaceutical / OncologyMetastatic Prostate CancerPreclinicalActiveUnited States
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- GlyphAlloSPT-300 / Allopregnanolone prodrug
- GlyphAgoSPT-320 / Agomelatine prodrug
- Glyph2BLSDSPT-348 / Non-hallucinogenic neuroplastogen prodrug
- GlyphCeleCelecoxib prodrug
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- VeligrotugVRDN-001
- ElegrobartVRDN-003
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- MR-146MR-146 / AAV Gene Therapy / OphthalmologyNeurotrophic keratopathy (NK)
- Low Dose Estrogen Weekly PatchEstrogen / Progestin receptors / Transdermal Patch / Women's HealthContraception
- PitolisantHistamine H3 receptor / Small molecule / NeurologyObstructive Sleep Apnea Syndrome
- SelatogrelP2Y12 receptor / Small molecule / CardiovascularAcute Myocardial Infarction
- CenerimodS1P1 receptor / Small molecule / ImmunologySystemic lupus erythematosus (SLE)
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- WVE-006WVE-006 / AAT transcript (Pi*ZZ mutation) / RNA editing oligonucleotide (AIMer) / Genetic/Respiratory/Liver DiseasesAlpha-1 antitrypsin deficiency (AATD)
- WVE-007WVE-007 / INHBE mRNA / siRNA / Metabolic DiseasesObesity
- WVE-008WVE-008 / PNPLA3 I148M / RNA editing oligonucleotide / Liver DiseasesPNPLA3-related liver disease
- WVE-N531WVE-N531 / DMD Exon 53 / Exon skipping oligonucleotide / Neuromuscular DiseasesDuchenne muscular dystrophy (DMD)
- WVE-003WVE-003 / Mutant huntingtin (mHTT) mRNA / Allele-selective silencing oligonucleotide / Neurological DiseasesHuntington's disease (HD)
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- ABCL635ABCL635 / NK3R / antibody medicine / Endocrinology / Women's HealthVasomotor Symptoms (VMS) associated with menopausePhase 2active
- ABCL575ABCL575 / OX40L / monoclonal antibody / Immunology & InflammationAtopic Dermatitis (AD)Phase 1active
- ABCL688ABCL688 / Undisclosed GPCR / Ion Channel / antibody medicine / AutoimmunityUndisclosed autoimmunity indicationIND-Enablingactive
- ABCL386ABCL386 / Undisclosed / antibody medicine / OncologyUndisclosed oncology indicationIND-Enablingactive
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- prademagene zamikeracelpz-cel / ZEVASKYN / COL7A1 / autologous cell-based gene therapy / Dermatology / rare diseaserecessive dystrophic epidermolysis bullosa (RDEB)Approvedapproved
- PSMA SIR-TABO-701 / PSMA / engineered autologous T-cell therapy / OncologyAdvanced prostate cancerPreclinicalpreclinical
- UX111UX111 / SGSH / in vivo AAV9 gene therapy / Rare disease / CNSSanfilippo Syndrome Type A (MPS IIIA)Phase 3clinical
- TSHA-102TSHA-102 / MECP2 / intrathecal AAV9 gene transfer therapy / Rare disease / CNSRett SyndromePhase 1/2clinical
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- MitapivatPYRUKYND®; AQVESME™ / PK / PK activator / Rare diseases; hematologyAdult PK deficiencyApprovedAdult thalassemiaApprovedU.S.KSAUAEAdult sickle cell diseaseRegulatory submissionU.S.
- TebapivatPK / PK activator / Rare diseases; hematologyLower-risk myelodysplastic syndromesClinical developmentSickle cell diseaseClinical development
- AG-181AG-181 / PAH / PAH stabilizer / Rare diseases; metabolic disordersPhenylketonuriaClinical development
- AG-236AG-236 / TMPRSS6 / siRNA / Rare diseases; hematologyPolycythemia veraClinical development
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- PraliciguatsGC / soluble guanylate cyclase stimulator / Kidney diseaseFocal segmental glomerulosclerosisPhase 2first patient enrolled Dec 2025
- Ebribafusp alfaAKB-097 / C3d/factor H / anti-C3d-factor H fusion protein; complement inhibitor / Rare kidney diseasesIgA nephropathyPhase 2 plannedbasket trial planned 2H 2026Lupus nephritisPhase 2 plannedbasket trial planned 2H 2026C3 glomerulopathyPhase 2 plannedbasket trial planned 2H 2026
- AKB-9090AKB-9090 / HIF-PH / HIF-PH inhibitor / Kidney disease; acute careCardiac surgery-associated acute kidney injuryPhase 1first participants dosed Apr 2026Acute respiratory distress syndromePreclinical
- AKB-10108AKB-10108 / HIF-PH / HIF-PH inhibitor / Other disease areasRetinopathy of prematurityPreclinical
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- EnvudeucitinibESK-001 / TYK2 / oral small molecule TYK2 inhibitor / ImmunologyPlaque psoriasisPhase 3ONWARD3 LTE ongoing; NDA planned 2H 2026GlobalJapanSystemic lupus erythematosusPhase 2LUMUS ongoing; topline data expected 2026Global
- A-005A-005 / TYK2 / CNS-penetrant allosteric TYK2 inhibitor / NeuroinflammationMultiple sclerosisPhase 1 completed; Phase 2 plannedPhase 2 initiation planned H1 2026
- LonigutamabIGF-1R / subcutaneous humanized IgG1 monoclonal antibody / Endocrinology/ImmunologyThyroid eye diseaseDevelopment program evaluation ongoing
- IRF5/Additional TargetsIRF5, additional targetsUndisclosed
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- AvexitideGLP-1 receptor / GLP-1 receptor antagonist / Endocrine/metabolic diseasesPost-bariatric hypoglycemia (PBH)Phase 3LUCIDITY completed enrollment; topline data expected Q3 2026USCongenital hyperinsulinism (HI)Clinical development next steps under discussionUS
- AMX0035AMX0035 / ER stress and mitochondrial dysfunction / Oral fixed-dose combination of sodium phenylbutyrate and taurursodiol / Neurodegenerative/rare diseasesWolfram syndromePhase 2HELIOS positive Week 48 data; pivotal Phase 3 planned pending FDA alignmentUSEU
- AMX0114AMX0114 / Calpain-2 (CAPN2) / Antisense oligonucleotide (ASO) / Neurodegenerative diseasesAmyotrophic lateral sclerosis (ALS)Phase 1LUMINA Cohort 2 fully enrolled; biomarker data expected H1 2026US
- AMX0318AMX0318 / GLP-1 receptor / Long-acting GLP-1 receptor antagonist / Endocrine/metabolic diseasesPBH and other rare diseasesIND-enablingIND-enabling studies underway; IND targeted for 2027
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- ZumilokibartAPG777 / IL-13 / subcutaneous half-life extended monoclonal antibody / immunology and inflammationAtopic dermatitisPhase 2in-progress; Part B enrollment completedAsthmaPhase 1complete; positive Phase 1b dataEosinophilic esophagitisPhase 1in-progress
- APG279APG279 / IL-13 + OX40L / coformulation of zumilokibart (APG777) + APG990 monoclonal antibodies / immunology and inflammationAtopic dermatitisPhase 1in-progress; Phase 1b PoC fully enrolled
- APG273APG273 / IL-13 + TSLP / combination of zumilokibart (APG777) + APG333 monoclonal antibodies / respiratory immunology and inflammationAsthma / COPDPreclinicalin-progress
- APG808APG808 / IL-4Rα / antibody / respiratory immunology and inflammationAsthmaPhase 1bpositive interim results announced May 2025
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- BPL-003BPL-003 / Intranasal mebufotenin benzoate / Mental healthTreatment-resistant depressionPhase 2In development
- VLS-01VLS-01 / 5-HT1/2/6/7 receptor agonist / Oral transmucosal DMT buccal film / Mental healthTreatment-resistant depressionPhase 2In development
- EMP-01EMP-01 / Oral R-MDMA / Mental healthSocial anxiety disorderPhase 2Positive topline results announced
- Novel 5-HT2A Receptor Agonists5-HT2A receptor / Small-molecule neuroplastogens / Mental healthOpioid use disorderDiscoveryIn developmentTreatment-resistant depressionDiscoveryIn development
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- DeramiocelCAP-1002 / allogeneic CDCs / Neuromuscular diseaseDuchenne muscular dystrophy (DMD)Phase 3in progressBecker muscular dystrophy (BMD)Preclinicalin progress
- StealthX™STX-S / Exosome-based vaccine / Infectious diseaseSARS-CoV-2Phase 1in progress
- StealthX™Therapeutic ExosomesEvaluatingPreclinicalin progress
- CDC-ExosomesExosome Platform / Neuromuscular diseaseDuchenne muscular dystrophyPreclinicalin progress
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- CLN-978CLN-978 / CD19xCD3 / bispecific T cell engager / ImmunologyOUTRACE: Rheumatoid arthritisPhase 1Initial data in RA in Q2 2026 and repeat dosing data in Q3 2026EuropeOUTRACE: Systemic lupus erythematosusPhase 1bInitial data in SLE in Q2 2026GlobalOUTRACE: Sjögren’s diseasePhase 1Initial data in Sjögren’s disease in Q4 2026Global
- VelinotamigBCMAxCD3 / bispecific T cell engager / ImmunologyAutoimmune diseasesIND-EnablingGenrix Bio initiated a Phase 1 study in China in Q4 2025; initial data expected in Q4 2026; clinical trial status not yet recruitingChina
- ZipalertinibEGFR ex20ins / tyrosine kinase inhibitor (TKI) / OncologyREZILIENT1: Non-small cell lung cancerPhase 2U.S. FDA accepted NDA for relapsed EGFR ex20ins NSCLC; PDUFA target action date February 27, 2027United StatesREZILIENT2: Non-small cell lung cancerPhase 2Parallel cohort study ongoingREZILIENT3: Non-small cell lung cancerPhase 3Phase 3 1L study fully enrolled; Taiho expects topline results by end of 2026Global
- CLN-049CLN-049 / FLT3xCD3 / bispecific T cell engager / OncologyAcute myeloid leukemia and myelodysplastic syndromePhase 1Dose escalation update in H2 2026; dose expansion cohorts to initiate in Q2 2026 and complete enrollment in Q4 2026Measurable residual disease in acute myeloid leukemiaPhase 1Phase 1 study ongoing
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- COM701COM701 / PVRIG / Fc-reduced anti-PVRIG antibody / OncologyRelapsed platinum-sensitive ovarian cancer; maintenance therapyAdaptive platform trialongoing
- COM701 + COM902 + pembrolizumabPVRIG; TIGIT; PD-1 / Antibody combination / OncologyPlatinum-resistant ovarian cancerProof of concept
- rilvegostomigPD-1/TIGIT / Fc-reduced anti-PD-1/TIGIT bispecific antibody / OncologyNSCLCPhase 3; Phase 1/2ongoingGastrointestinal cancerPhase 3; Phase 1/2ongoingEndometrial cancerPhase 3ongoing
- GS-0321GS-0321 / IL18BP / Anti-IL18BP antibody / OncologySolid tumorsPhase 1ongoing
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- CNM-Au8®CNM-Au8 / mitochondrial function / NAD pathway; neuroprotection and remyelination / oral gold nanocrystal suspension / NeurologyAmyotrophic Lateral Sclerosis (ALS)Global Phase 3 plannedPlanned; EAPs ongoing; regulatory agency engagement ongoingUSEUGlobalMultiple Sclerosis (MS)Phase 3 plannedPlanned; EAP ongoing; Repair-MS target engagement and safety confirmedUSGlobalParkinson’s Disease (PD)In development; REPAIR-PD trial listed
- CNM-ZnAgCNM-ZnAg / zinc-silver ionic solution / Infectious diseaseAntiviral and antimicrobial usesDemonstrated antiviral and antimicrobial properties
- CNM-AgZn17CNM-AgZn17 / topical gel polymer suspension of silver and zinc ions / Infectious disease / wound healingInfectious diseasesIn developmentWound healingIn development
- CNM-PtAu7CNM-PtAu7 / gold-platinum nanotherapeutic / OncologyCancerIn vitroInitial research
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- tovecimigCTX-009 / DLL4 x VEGF-A / bispecific antibody / OncologyBiliary tract cancer (2L)Phase 2/3Met primary endpoint; PFS/OS expected late Q1 2026Colorectal cancer (monotherapy 3L/4L)Phase 2CompletedDLL4+ tumors basket studyAdditional indications planned H1 2026
- CTX-471CTX-471 / CD137 / agonistic antibody / OncologyNCAM (CD56) basket studyTrial initiation expected Q1 2026Post-checkpoint basket studyCompleted
- CTX-8371CTX-8371 / PD-1 x PD-L1 / bispecific antibody / OncologySolid tumorsPhase 1Expansion cohorts initiated; Phase 1 data expected H1 2026
- CTX-10726CTX-10726 / PD-1 x VEGF-A / bispecific antibody / OncologySolid tumorsIND-enabling preclinicalPhase 1 initiation expected Q1 2026
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- PIPE-791LPA1R / small molecule LPA1R antagonist / inflammation; immunology; neuroscienceIdiopathic pulmonary fibrosisPhase 2activeProgressive MSPhase 1deferredChronic painPhase 1bactive
- CTX-343LPA1R / peripherally restricted small molecule LPA1R antagonist / peripheral indicationsPeripheralPreclinicaldeferred
- PIPE-307JNJ-5120 / M1R / small molecule M1R antagonist / neuroscienceRelapsing-remitting MSPhase 2licensedMajor depressive disorderPhase 2licensed
- CalpainCalpain / calpain inhibitorUndisclosedPreclinicalactive
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- AficamtenMYQORZO / cardiac myosin inhibitor / small molecule / CardiovascularoHCMApproved & CommercializedApproved in U.S., China, & EUU.S.ChinaEUoHCM (vs. metoprolol)Phase 3MAPLE-HCMPediatric oHCMPhase 2CEDAR-HCM
- Ulacamtencardiac myosin inhibitor / small molecule / CardiovascularHFpEFPhase 2AMBER-HFpEF
- Omecamtiv Mecarbilcardiac myosin activator / small molecule / CardiovascularHFrEFPhase 3COMET-HF
- CK-089CK-089 / fast skeletal muscle troponin activator / small molecule / NeuromuscularMuscular Dystrophy, OtherPhase 1
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- DT-216P2DT-216P2 / FXN GAA repeat expansion / frataxin expression / GeneTAC small molecule / Genetic disease / neurologyFriedreich ataxiaPhase 1/2RESTORE-FA multiple-dose trial opened/ongoing; evaluates IV and subcutaneous administrationex-U.S.
- DT-168DT-168 / TCF4 CTG repeat expansion / GeneTAC small molecule eye drop / Ophthalmology / genetic diseaseFuchs endothelial corneal dystrophyPhase 2Biomarker trial opened/ongoing in FECD patients scheduled for corneal transplant surgery
- DT-818DT-818 / DMPK CTG repeat expansion / GeneTAC small molecule / Neuromuscular / genetic diseaseMyotonic dystrophy type 1Phase 1 plannedPatient dosing in Phase 1 MAD trial anticipated in H1 2026Australia
- Huntington's disease GeneTAC programHTT CAG repeat expansion / mutant huntingtin expression / GeneTAC small molecule / Neurology / genetic diseaseHuntington's diseasePreclinicalProgram disclosed; candidate not named
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- Halneuron® NaV1.7 InjectionHAL / Halneuron® / NaV1.7 / subcutaneous small-molecule/peptide analgesic injection / Pain / neuropathyChemotherapy-induced neuropathic pain (CINP)Phase 2bOngoing; FDA Fast Track designationU.S.General cancer painPhase 2aCompleteAcute surgical painPreclinical
- SP16 Intravenous AdministrationSP16 IV / LRP1 agonist / IV peptide therapeutic / Neuropathy / cancer-related painChemotherapy-induced peripheral neuropathy (CIPN)Phase 1bNCI funded / forthcoming
- IMC-1IMC-1 / Herpesvirus suppression / COX-2 inhibition / Fixed-dose combination of anti-herpesvirus medication and COX-2 inhibitor / Pain / fatigue illnessFibromyalgiaPhase 2aPreviously demonstrated efficacy; disclosed as a novel therapeutic under antiviral combinations
- IMC-2IMC-2 / Herpesvirus suppression / COX-2 inhibition / Fixed-dose combination of anti-herpesvirus medication and COX-2 inhibitor / Pain / fatigue illnessLong COVIDOpen-label investigator-initiated study completed/reported; disclosed as a novel therapeutic under antiviral combinations
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- SevasemtenMyosin ATPase / Oral fast skeletal myosin inhibitor / Muscular dystrophyBecker muscular dystrophyPivotal / Phase 3Duchenne muscular dystrophyPhase 2
- EDG-7500EDG-7500 / Undisclosed cardiac sarcomeric target / Oral selective cardiac sarcomere modulator / CardiovascularHypertrophic cardiomyopathyPhase 2
- EDG-15400EDG-15400 / Undisclosed cardiac sarcomeric target / Oral selective cardiac sarcomere modulator / CardiovascularHFpEFPhase 1
- EDG-003EDG-003 / Undisclosed target / CardiometabolicCardiometabolicPreclinical
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- 4D-1504D-150 / VEGF family members / Intravitreal AAV genetic medicine / RetinaWet AMDPivotal / Phase 34FRONT-1 and 4FRONT-2 Phase 3; PRISM Phase 1/2 ongoingDiabetic macular edemaPivotal readySPECTRA clinical trial Part 1
- 4D-7104D-710 / CFTR / Aerosol-delivered AAV genetic medicine / PulmonologyCystic fibrosis lung disease; not modulator-amenable / ineligible or discontinued CFTR modulator therapyPhase 2AEROW Phase 1/2 study; pipeline lists Phase 2
- 4D-1754D-175 / Complement factor H / complement pathway / Intravitreal AAV genetic medicine / RetinaGeographic atrophySeeking partnerships to advance
- 4D-7254D-725 / Aerosol-delivered AAV genetic medicine / PulmonologyAlpha-1 antitrypsin lung diseasePreclinicalSeeking partnerships to advance
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- MRT-6160MRT-6160 / VAV1 / Oral molecular glue degrader / Immunology & InflammationImmune-mediated diseasesClinical
- MRT-8102MRT-8102 / NEK7 / Oral molecular glue degrader / Immunology & InflammationIL-1β/NLRP3-driven inflammatory diseasesPhase 1
- MRT-2359MRT-2359 / GSPT1 / Oral molecular glue degrader / OncologyCastration-resistant prostate cancerPhase 1/2Ongoing
- Cyclin E1 degrader programCCNE1 / CDK2 / Molecular glue degrader / OncologyCCNE1-amplified tumorsPreclinicalLead optimizationER+ breast cancerPreclinicalLead optimization
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- HCW9302HCW9302 / IL-2Rα / IL-2 fusion protein / AutoimmuneAlopecia areataPhase 1OngoingUS
- HCW11-006HCW11-006 / Multiple cytokines (IL-15/IL-15Rα, IL-7, IL-21, IL-2) / Multi-functional fusion immunotherapeutic / OncologyCancerPhase 1Planned by licenseeChina
- HCW11-018bHCW11-018b / Tissue Factor/CD3 / Tetra-valent bispecific T-cell engager (Big BiTE) / OncologySolid tumorsPreclinicalIND-enablingGlobal
- HCW11-040HCW11-040 / PD-1 (pembrolizumab-based) + TGF-β / Tetra-valent immune checkpoint inhibitor fusion / OncologySolid tumorsPreclinicalIND-enablingGlobal
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- nogapendekin alfa inbakiceptN-803 / ANKTIVA® / IL-15 receptor / Cytokine fusion protein / Interleukin-15 superagonist / Oncology, Infectious DiseasesBCG Unresponsive NMIBC Carcinoma In-Situ & Papillary (QUILT-3.032)ApprovedMarketedUSBCG Naïve NMIBC (QUILT-2.005)Phase 2/Phase 3ActiveUSNon-Small Cell Lung Cancer (ResQ201A)Phase 3ActiveGlobal
- PD-L1 t-haNKPD-L1 / Cell Therapy / Engineered NK cells / OncologyAdvanced Solid TumorsPhase 2ActiveUS
- AldoxAlbumin-bound doxorubicin complex / OncologyAdvanced Solid TumorsPhase 2ActiveUS
- Recombinant BCGVaccine / Immunotherapy / OncologyBladder CancerExpanded AccessActiveUS
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- anzu-cel (IMA203)IMA203 / anzutresgene autoleucel / PRAME / Autologous TCR-T cell therapy / Solid Tumors2L+ cutaneous melanomaPhase 3ongoingUSEU5Uveal melanomaPhase 1bongoingUSEU5PRAME+ solid tumorsPhase 1ongoingUSEU5
- IMA203CD8 (GEN2)IMA203CD8 / PRAME / Autologous TCR-T cell therapy (next-gen with CD8) / Solid TumorsPRAME+ advanced solid tumorsPhase 1dose escalation ongoingUSEU5Ovarian and endometrial cancersPhase 1ongoingUSEU5
- IMA402IMA402 / PRAME / Half-life extended TCR bispecific (TCER) / Solid TumorsRecurrent/refractory solid tumors (melanoma, gynecologic, sqNSCLC)Phase 1/2Phase 1b expansion ongoingUSEU
- IMA401IMA401 / MAGEA4/8 / TCR bispecific (TCER) / Solid TumorsRecurrent/refractory solid tumors (HNSCC, sqNSCLC, melanoma)Phase 1dose escalation completedUSEU
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- INB-100INB-100 / DeltEx Allo allogeneic γδ T cell therapy / OncologyAcute leukemia post-HSCTPhase 1Expansion cohort enrollingUS
- INB-200INB-200 / DeltEx DRI autologous genetically modified γδ T cell therapy / OncologyNewly diagnosed glioblastomaPhase 1Treatment completed; long-term follow-upUS
- INB-400INB-400 / DeltEx DRI autologous and allogeneic γδ T cell therapy / OncologyNewly diagnosed glioblastomaPhase 2Enrollment suspended; treatment completed; data collection ongoingUS
- INB-619INB-619 / CD19 / γδ T cell engager (TCE) / Oncology and autoimmune diseasesCD19+ B-cell malignanciesPreclinicalIND-enabling studiesB-cell mediated autoimmune diseasesPreclinicalIND-enabling studies