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Pipeline Watchlist

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Companies

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  • MaralixibatLIVMARLI / IBAT / Small molecule / Rare Liver Diseases
    Alagille syndrome (ALGS)Progressive familial intrahepatic cholestasis (PFIC)Biliary atresia
  • VolixibatSHP626 / IBAT / Small molecule / Rare Liver Diseases
    Primary sclerosing cholangitis (PSC)Primary biliary cholangitis (PBC)
  • MRM-3379MRM-3379 / PDE4D / Small molecule / Neurocognitive Disorders
    Fragile X syndrome
  • Cholic acidCHOLBAM / Bile acid replacement / Small molecule / Rare Liver Diseases
    Bile acid synthesis disordersPeroxisomal disorders (Zellweger spectrum disorders)
  • ChenodiolCTEXLI / Bile acid replacement / Small molecule / Rare Genetic Disorders
    Cerebrotendinous xanthomatosis (CTX)
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  • ALXN1840ALXN1840 / Copper / Small Molecule / Rare Diseases
    Wilson Disease
  • MNPR-101-ZrMNPR-101-Zr / uPAR / Radiopharmaceutical / Oncology
    Advanced Solid Cancers (Imaging)
  • MNPR-101-LuMNPR-101-Lu / uPAR / Radiopharmaceutical / Oncology
    Advanced Solid Cancers
  • MNPR-101-AcMNPR-101-Ac / uPAR / Radiopharmaceutical / Oncology
    Advanced Solid Cancers
  • MNPR-101MNPR-101 / uPAR / Monoclonal Antibody / Oncology
    Solid Tumors
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  • PetosemtamabMCLA-158 / EGFR x LGR5 / Bispecific Antibody / Oncology
    1L 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 / Oncology
    Pancreatic 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 / Oncology
    Solid tumorsPhase 1/Phase 2OngoingGlobal2L+ EGFRm non-small cell lung cancer (with chemotherapy)Phase 2OngoingGlobal
  • BesufetamigONO-4685 / PD-1 x CD3 / Bispecific Antibody / Oncology, Immunology
    Relapsed/Refractory T Cell LymphomaPhase 1OngoingGlobalPsoriasisPhase 1OngoingGlobal
  • INCA33890INCA33890 / PD-1 x TGFBr2 / Bispecific Antibody / Oncology
    Select advanced solid tumorsPhase 1OngoingGlobal
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  • navacaprantNMRA-140 / Kappa opioid receptor (KOR) antagonist / Small molecule / Neuropsychiatry
    Major Depressive Disorder
  • NMRA-511NMRA-511 / Vasopressin 1a receptor (V1aR) antagonist / Small molecule / Neuropsychiatry / Neurodegenerative
    Alzheimer's disease agitation
  • NMRA-215NMRA-215 / NLRP3 inhibitor / Small molecule / Endocrinology / Metabolic
    Obesity
  • NMRA-861NMRA-861 / M4 muscarinic receptor positive allosteric modulator (M4 PAM) / Small molecule / Neuropsychiatry
    SchizophreniaNeuropsychiatric disorders
  • NMRA-898NMRA-898 / M4 muscarinic receptor positive allosteric modulator (M4 PAM) / Small molecule / Neuropsychiatry
    SchizophreniaNeuropsychiatric disorders
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  • triamcinolone acetonideZILRETTA / Small Molecule Formulation / Musculoskeletal Pain
    Shoulder Osteoarthritis (OA)Phase 3Active
  • iovera°iovera° / Device / Neurology/Pain
    SpasticityClinicalActive
  • bupivacaine liposomeEXPAREL / Small Molecule Formulation / Pain Management
    Acute Post-Surgical Pain (Pediatric 0 to < 6)ClinicalActive
  • enekinragene inzadenovecPCRX-201 / IL-1Ra / Gene Therapy / Musculoskeletal Pain
    Osteoarthritis of the Knee (OAK)Phase 2Active
  • PCRX-2002PCRX-2002 / Unknown / Pain Management
    Post-Surgical PainClinicalActive
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  • VAX-31VAX-31 / Streptococcus pneumoniae (31 Serotypes) / Pneumococcal Conjugate Vaccine (PCV) / Infectious Diseases
    Invasive 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 Diseases
    Invasive Pneumococcal Disease (IPD) in InfantsPhase 2OngoingUnited States
  • VAX-A1VAX-A1 / Group A Streptococcus / Conjugate Vaccine / Infectious Diseases
    Group A Strep InfectionsPreclinicalOngoing
  • VAX-GIVAX-GI / Shigella / Vaccine / Infectious Diseases
    Shigellosis / Shigella InfectionsPreclinicalOngoing
  • VAX-XLVAX-XL / Streptococcus pneumoniae / Pneumococcal Conjugate Vaccine (PCV) / Infectious Diseases
    Invasive Pneumococcal Disease (IPD)PreclinicalOngoing
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  • PMN310PMN310 / Amyloid-Beta / Monoclonal Antibody / Neurology
    Alzheimer's Disease
  • PMN267PMN267 / TDP-43 / Monoclonal Antibody / Neurology
    Amyotrophic Lateral Sclerosis (ALS)Frontotemporal Dementia (FTD)Limbic-predominant age-related TDP-43 encephalopathy (LATE)
  • PMN442PMN442 / Alpha-Synuclein / Monoclonal Antibody / Neurology
    Parkinson's Disease (PD)Multiple System Atrophy (MSA)Dementia with Lewy Bodies (DLB)
  • PMN311PMN311 / Amyloid-Beta / Vaccine / Neurology
    Alzheimer's Disease
  • PMN440PMN440 / Alpha-Synuclein / Vaccine / Neurology
    Parkinson's Disease
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  • deupirfenidoneLYT-100 / Small Molecule / Respiratory / Fibrotic Diseases
    Idiopathic Pulmonary Fibrosis (IPF)Phase 3ActiveGlobal
  • LYT-200LYT-200 / Biologic / Oncology
    Relapsed/Refractory High-Risk Myelodysplastic Syndrome (MDS) and Acute Myeloid Leukemia (AML)Phase 1ActiveGlobalSolid TumorsPhase 1ActiveGlobal
  • GlyphAlloSPT-300 / Allopregnanolone / Small Molecule / Neuropsychiatry
    Anxiety and Mood DisordersPhase 2ActiveGlobal
  • GlyphAgoSPT-320 / Small Molecule / Neuropsychiatry
    Neurological / Neuropsychiatric IndicationsPhase 1ActiveGlobal
  • xanomeline and trospium chlorideKarXT / Cobenfy / Muscarinic Receptors / Small Molecule / Neuropsychiatry
    SchizophreniaApprovedActiveUS
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  • AMT-130AMT-130 / Huntingtin protein (mHTT) / Gene Therapy (AAV5) / Central Nervous System
    Huntington's DiseasePhase 1/2ActiveGlobal
  • AMT-191AMT-191 / Alpha-galactosidase A (GLA) / Gene Therapy / Rare Diseases
    Fabry DiseasePhase 1/2aActiveGlobal
  • AMT-260AMT-260 / Undisclosed / Gene Therapy / Central Nervous System
    Refractory Mesial Temporal Lobe EpilepsyPhase 1/2aActiveGlobal
  • AMT-162AMT-162 / SOD1 / Gene Therapy / Central Nervous System
    Amyotrophic Lateral Sclerosis (ALS)Preclinical/ClinicalActiveGlobal
  • etranacogene dezaparvovecAMT-061 / HEMGENIX / Factor IX / Gene Therapy (AAV5) / Hematology
    Hemophilia BApprovedMarketedUSGlobal
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  • BrepocitinibJAK1 / TYK2 / Small Molecule / Autoimmune & Inflammatory
    DermatomyositisPhase 3ActiveGlobalNon-infectious UveitisPhase 3ActiveGlobalCutaneous SarcoidosisPhase 2ActiveGlobal
  • IMVT-1402IMVT-1402 / FcRn / Monoclonal Antibody / Autoimmune
    Rheumatoid Arthritis (difficult-to-treat)Phase 2ActiveGlobalGraves' DiseasePhase 2/3ActiveGlobal
  • BatoclimabFcRn / Monoclonal Antibody / Autoimmune
    Thyroid Eye Disease (TED)Phase 3ActiveGlobalMyasthenia Gravis (MG)Phase 3ActiveGlobalChronic Inflammatory Demyelinating Polyneuropathy (CIDP)Phase 2bActiveGlobal
  • MosliciguatRVT-304 / sGC / Small Molecule / Pulmonary
    Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)Phase 2ActiveGlobal
  • NamilumabGM-CSF / Monoclonal Antibody / Autoimmune & Inflammatory
    SarcoidosisPhase 2ActiveGlobal
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  • RopidoxuridineIPdR / Radiation Sensitizer / Small Molecule / Oncology
    GlioblastomaPhase 2ActiveUnited StatesSarcomasPhase 1/Phase 2ActiveUnited States
  • SP-2-225SP-2-225 / HDAC6 / Small Molecule / Oncology
    Breast Cancer / Solid TumorsPreclinicalActiveUnited States
  • SP-1-303SP-1-303 / Class I HDAC / Small Molecule / Oncology
    ER+ Breast CancerPreclinicalActiveUnited States
  • SP-1-161SP-1-161 / HDAC / Small Molecule / Oncology
    Solid TumorsDiscoveryActiveUnited States
  • PSMA-B ligandPSMA-B / PSMA / Radiopharmaceutical / Oncology
    Metastatic Prostate CancerPreclinicalActiveUnited States
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  • MR-146MR-146 / AAV Gene Therapy / Ophthalmology
    Neurotrophic keratopathy (NK)
  • Low Dose Estrogen Weekly PatchEstrogen / Progestin receptors / Transdermal Patch / Women's Health
    Contraception
  • PitolisantHistamine H3 receptor / Small molecule / Neurology
    Obstructive Sleep Apnea Syndrome
  • SelatogrelP2Y12 receptor / Small molecule / Cardiovascular
    Acute Myocardial Infarction
  • CenerimodS1P1 receptor / Small molecule / Immunology
    Systemic lupus erythematosus (SLE)
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  • WVE-006WVE-006 / AAT transcript (Pi*ZZ mutation) / RNA editing oligonucleotide (AIMer) / Genetic/Respiratory/Liver Diseases
    Alpha-1 antitrypsin deficiency (AATD)
  • WVE-007WVE-007 / INHBE mRNA / siRNA / Metabolic Diseases
    Obesity
  • WVE-008WVE-008 / PNPLA3 I148M / RNA editing oligonucleotide / Liver Diseases
    PNPLA3-related liver disease
  • WVE-N531WVE-N531 / DMD Exon 53 / Exon skipping oligonucleotide / Neuromuscular Diseases
    Duchenne muscular dystrophy (DMD)
  • WVE-003WVE-003 / Mutant huntingtin (mHTT) mRNA / Allele-selective silencing oligonucleotide / Neurological Diseases
    Huntington's disease (HD)
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  • ABCL635ABCL635 / NK3R / antibody medicine / Endocrinology / Women's Health
    Vasomotor Symptoms (VMS) associated with menopausePhase 2active
  • ABCL575ABCL575 / OX40L / monoclonal antibody / Immunology & Inflammation
    Atopic Dermatitis (AD)Phase 1active
  • ABCL688ABCL688 / Undisclosed GPCR / Ion Channel / antibody medicine / Autoimmunity
    Undisclosed autoimmunity indicationIND-Enablingactive
  • ABCL386ABCL386 / Undisclosed / antibody medicine / Oncology
    Undisclosed oncology indicationIND-Enablingactive
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  • prademagene zamikeracelpz-cel / ZEVASKYN / COL7A1 / autologous cell-based gene therapy / Dermatology / rare disease
    recessive dystrophic epidermolysis bullosa (RDEB)Approvedapproved
  • PSMA SIR-TABO-701 / PSMA / engineered autologous T-cell therapy / Oncology
    Advanced prostate cancerPreclinicalpreclinical
  • UX111UX111 / SGSH / in vivo AAV9 gene therapy / Rare disease / CNS
    Sanfilippo Syndrome Type A (MPS IIIA)Phase 3clinical
  • TSHA-102TSHA-102 / MECP2 / intrathecal AAV9 gene transfer therapy / Rare disease / CNS
    Rett SyndromePhase 1/2clinical
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  • MitapivatPYRUKYND®; AQVESME™ / PK / PK activator / Rare diseases; hematology
    Adult PK deficiencyApprovedAdult thalassemiaApprovedU.S.KSAUAEAdult sickle cell diseaseRegulatory submissionU.S.
  • TebapivatPK / PK activator / Rare diseases; hematology
    Lower-risk myelodysplastic syndromesClinical developmentSickle cell diseaseClinical development
  • AG-181AG-181 / PAH / PAH stabilizer / Rare diseases; metabolic disorders
    PhenylketonuriaClinical development
  • AG-236AG-236 / TMPRSS6 / siRNA / Rare diseases; hematology
    Polycythemia veraClinical development
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  • PraliciguatsGC / soluble guanylate cyclase stimulator / Kidney disease
    Focal segmental glomerulosclerosisPhase 2first patient enrolled Dec 2025
  • Ebribafusp alfaAKB-097 / C3d/factor H / anti-C3d-factor H fusion protein; complement inhibitor / Rare kidney diseases
    IgA 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 care
    Cardiac surgery-associated acute kidney injuryPhase 1first participants dosed Apr 2026Acute respiratory distress syndromePreclinical
  • AKB-10108AKB-10108 / HIF-PH / HIF-PH inhibitor / Other disease areas
    Retinopathy of prematurityPreclinical
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  • EnvudeucitinibESK-001 / TYK2 / oral small molecule TYK2 inhibitor / Immunology
    Plaque 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 / Neuroinflammation
    Multiple sclerosisPhase 1 completed; Phase 2 plannedPhase 2 initiation planned H1 2026
  • LonigutamabIGF-1R / subcutaneous humanized IgG1 monoclonal antibody / Endocrinology/Immunology
    Thyroid eye diseaseDevelopment program evaluation ongoing
  • IRF5/Additional TargetsIRF5, additional targets
    Undisclosed
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  • AvexitideGLP-1 receptor / GLP-1 receptor antagonist / Endocrine/metabolic diseases
    Post-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 diseases
    Wolfram syndromePhase 2HELIOS positive Week 48 data; pivotal Phase 3 planned pending FDA alignmentUSEU
  • AMX0114AMX0114 / Calpain-2 (CAPN2) / Antisense oligonucleotide (ASO) / Neurodegenerative diseases
    Amyotrophic 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 diseases
    PBH 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 inflammation
    Atopic 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 inflammation
    Atopic dermatitisPhase 1in-progress; Phase 1b PoC fully enrolled
  • APG273APG273 / IL-13 + TSLP / combination of zumilokibart (APG777) + APG333 monoclonal antibodies / respiratory immunology and inflammation
    Asthma / COPDPreclinicalin-progress
  • APG808APG808 / IL-4Rα / antibody / respiratory immunology and inflammation
    AsthmaPhase 1bpositive interim results announced May 2025
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  • BPL-003BPL-003 / Intranasal mebufotenin benzoate / Mental health
    Treatment-resistant depressionPhase 2In development
  • VLS-01VLS-01 / 5-HT1/2/6/7 receptor agonist / Oral transmucosal DMT buccal film / Mental health
    Treatment-resistant depressionPhase 2In development
  • EMP-01EMP-01 / Oral R-MDMA / Mental health
    Social anxiety disorderPhase 2Positive topline results announced
  • Novel 5-HT2A Receptor Agonists5-HT2A receptor / Small-molecule neuroplastogens / Mental health
    Opioid use disorderDiscoveryIn developmentTreatment-resistant depressionDiscoveryIn development
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  • DeramiocelCAP-1002 / allogeneic CDCs / Neuromuscular disease
    Duchenne muscular dystrophy (DMD)Phase 3in progressBecker muscular dystrophy (BMD)Preclinicalin progress
  • StealthX™STX-S / Exosome-based vaccine / Infectious disease
    SARS-CoV-2Phase 1in progress
  • StealthX™Therapeutic Exosomes
    EvaluatingPreclinicalin progress
  • CDC-ExosomesExosome Platform / Neuromuscular disease
    Duchenne muscular dystrophyPreclinicalin progress
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  • CLN-978CLN-978 / CD19xCD3 / bispecific T cell engager / Immunology
    OUTRACE: 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 / Immunology
    Autoimmune 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) / Oncology
    REZILIENT1: 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 / Oncology
    Acute 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 / Oncology
    Relapsed platinum-sensitive ovarian cancer; maintenance therapyAdaptive platform trialongoing
  • COM701 + COM902 + pembrolizumabPVRIG; TIGIT; PD-1 / Antibody combination / Oncology
    Platinum-resistant ovarian cancerProof of concept
  • rilvegostomigPD-1/TIGIT / Fc-reduced anti-PD-1/TIGIT bispecific antibody / Oncology
    NSCLCPhase 3; Phase 1/2ongoingGastrointestinal cancerPhase 3; Phase 1/2ongoingEndometrial cancerPhase 3ongoing
  • GS-0321GS-0321 / IL18BP / Anti-IL18BP antibody / Oncology
    Solid tumorsPhase 1ongoing
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  • CNM-Au8®CNM-Au8 / mitochondrial function / NAD pathway; neuroprotection and remyelination / oral gold nanocrystal suspension / Neurology
    Amyotrophic 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 disease
    Antiviral and antimicrobial usesDemonstrated antiviral and antimicrobial properties
  • CNM-AgZn17CNM-AgZn17 / topical gel polymer suspension of silver and zinc ions / Infectious disease / wound healing
    Infectious diseasesIn developmentWound healingIn development
  • CNM-PtAu7CNM-PtAu7 / gold-platinum nanotherapeutic / Oncology
    CancerIn vitroInitial research
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  • tovecimigCTX-009 / DLL4 x VEGF-A / bispecific antibody / Oncology
    Biliary 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 / Oncology
    NCAM (CD56) basket studyTrial initiation expected Q1 2026Post-checkpoint basket studyCompleted
  • CTX-8371CTX-8371 / PD-1 x PD-L1 / bispecific antibody / Oncology
    Solid tumorsPhase 1Expansion cohorts initiated; Phase 1 data expected H1 2026
  • CTX-10726CTX-10726 / PD-1 x VEGF-A / bispecific antibody / Oncology
    Solid tumorsIND-enabling preclinicalPhase 1 initiation expected Q1 2026
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  • PIPE-791LPA1R / small molecule LPA1R antagonist / inflammation; immunology; neuroscience
    Idiopathic pulmonary fibrosisPhase 2activeProgressive MSPhase 1deferredChronic painPhase 1bactive
  • CTX-343LPA1R / peripherally restricted small molecule LPA1R antagonist / peripheral indications
    PeripheralPreclinicaldeferred
  • PIPE-307JNJ-5120 / M1R / small molecule M1R antagonist / neuroscience
    Relapsing-remitting MSPhase 2licensedMajor depressive disorderPhase 2licensed
  • CalpainCalpain / calpain inhibitor
    UndisclosedPreclinicalactive
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  • AficamtenMYQORZO / cardiac myosin inhibitor / small molecule / Cardiovascular
    oHCMApproved & CommercializedApproved in U.S., China, & EUU.S.ChinaEUoHCM (vs. metoprolol)Phase 3MAPLE-HCMPediatric oHCMPhase 2CEDAR-HCM
  • Ulacamtencardiac myosin inhibitor / small molecule / Cardiovascular
    HFpEFPhase 2AMBER-HFpEF
  • Omecamtiv Mecarbilcardiac myosin activator / small molecule / Cardiovascular
    HFrEFPhase 3COMET-HF
  • CK-089CK-089 / fast skeletal muscle troponin activator / small molecule / Neuromuscular
    Muscular Dystrophy, OtherPhase 1
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  • DT-216P2DT-216P2 / FXN GAA repeat expansion / frataxin expression / GeneTAC small molecule / Genetic disease / neurology
    Friedreich 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 disease
    Fuchs 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 disease
    Myotonic 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 disease
    Huntington's diseasePreclinicalProgram disclosed; candidate not named
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  • Halneuron® NaV1.7 InjectionHAL / Halneuron® / NaV1.7 / subcutaneous small-molecule/peptide analgesic injection / Pain / neuropathy
    Chemotherapy-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 pain
    Chemotherapy-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 illness
    FibromyalgiaPhase 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 illness
    Long 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 dystrophy
    Becker muscular dystrophyPivotal / Phase 3Duchenne muscular dystrophyPhase 2
  • EDG-7500EDG-7500 / Undisclosed cardiac sarcomeric target / Oral selective cardiac sarcomere modulator / Cardiovascular
    Hypertrophic cardiomyopathyPhase 2
  • EDG-15400EDG-15400 / Undisclosed cardiac sarcomeric target / Oral selective cardiac sarcomere modulator / Cardiovascular
    HFpEFPhase 1
  • EDG-003EDG-003 / Undisclosed target / Cardiometabolic
    CardiometabolicPreclinical
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  • 4D-1504D-150 / VEGF family members / Intravitreal AAV genetic medicine / Retina
    Wet 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 / Pulmonology
    Cystic 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 / Retina
    Geographic atrophySeeking partnerships to advance
  • 4D-7254D-725 / Aerosol-delivered AAV genetic medicine / Pulmonology
    Alpha-1 antitrypsin lung diseasePreclinicalSeeking partnerships to advance
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  • MRT-6160MRT-6160 / VAV1 / Oral molecular glue degrader / Immunology & Inflammation
    Immune-mediated diseasesClinical
  • MRT-8102MRT-8102 / NEK7 / Oral molecular glue degrader / Immunology & Inflammation
    IL-1β/NLRP3-driven inflammatory diseasesPhase 1
  • MRT-2359MRT-2359 / GSPT1 / Oral molecular glue degrader / Oncology
    Castration-resistant prostate cancerPhase 1/2Ongoing
  • Cyclin E1 degrader programCCNE1 / CDK2 / Molecular glue degrader / Oncology
    CCNE1-amplified tumorsPreclinicalLead optimizationER+ breast cancerPreclinicalLead optimization
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  • HCW9302HCW9302 / IL-2Rα / IL-2 fusion protein / Autoimmune
    Alopecia areataPhase 1OngoingUS
  • HCW11-006HCW11-006 / Multiple cytokines (IL-15/IL-15Rα, IL-7, IL-21, IL-2) / Multi-functional fusion immunotherapeutic / Oncology
    CancerPhase 1Planned by licenseeChina
  • HCW11-018bHCW11-018b / Tissue Factor/CD3 / Tetra-valent bispecific T-cell engager (Big BiTE) / Oncology
    Solid tumorsPreclinicalIND-enablingGlobal
  • HCW11-040HCW11-040 / PD-1 (pembrolizumab-based) + TGF-β / Tetra-valent immune checkpoint inhibitor fusion / Oncology
    Solid tumorsPreclinicalIND-enablingGlobal
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  • nogapendekin alfa inbakiceptN-803 / ANKTIVA® / IL-15 receptor / Cytokine fusion protein / Interleukin-15 superagonist / Oncology, Infectious Diseases
    BCG 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 / Oncology
    Advanced Solid TumorsPhase 2ActiveUS
  • AldoxAlbumin-bound doxorubicin complex / Oncology
    Advanced Solid TumorsPhase 2ActiveUS
  • Recombinant BCGVaccine / Immunotherapy / Oncology
    Bladder CancerExpanded AccessActiveUS
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  • anzu-cel (IMA203)IMA203 / anzutresgene autoleucel / PRAME / Autologous TCR-T cell therapy / Solid Tumors
    2L+ cutaneous melanomaPhase 3ongoingUSEU5Uveal melanomaPhase 1bongoingUSEU5PRAME+ solid tumorsPhase 1ongoingUSEU5
  • IMA203CD8 (GEN2)IMA203CD8 / PRAME / Autologous TCR-T cell therapy (next-gen with CD8) / Solid Tumors
    PRAME+ advanced solid tumorsPhase 1dose escalation ongoingUSEU5Ovarian and endometrial cancersPhase 1ongoingUSEU5
  • IMA402IMA402 / PRAME / Half-life extended TCR bispecific (TCER) / Solid Tumors
    Recurrent/refractory solid tumors (melanoma, gynecologic, sqNSCLC)Phase 1/2Phase 1b expansion ongoingUSEU
  • IMA401IMA401 / MAGEA4/8 / TCR bispecific (TCER) / Solid Tumors
    Recurrent/refractory solid tumors (HNSCC, sqNSCLC, melanoma)Phase 1dose escalation completedUSEU
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  • INB-100INB-100 / DeltEx Allo allogeneic γδ T cell therapy / Oncology
    Acute leukemia post-HSCTPhase 1Expansion cohort enrollingUS
  • INB-200INB-200 / DeltEx DRI autologous genetically modified γδ T cell therapy / Oncology
    Newly diagnosed glioblastomaPhase 1Treatment completed; long-term follow-upUS
  • INB-400INB-400 / DeltEx DRI autologous and allogeneic γδ T cell therapy / Oncology
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