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Disease","graph2":"Preclinical","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Ocugen \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Ocugen \/ Inapplicable"},{"orgOrder":0,"company":"Biogen","sponsor":"Massachusetts Eye And Ear","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Biogen \/ Massachusetts Eye And Ear","highestDevelopmentStatusID":"4","companyTruncated":"Biogen \/ Massachusetts Eye And Ear"},{"orgOrder":0,"company":"IVERIC bio","sponsor":"Opus Genetics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Acquisition","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IVERIC bio","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IVERIC bio \/ Opus Genetics","highestDevelopmentStatusID":"4","companyTruncated":"IVERIC bio \/ Opus Genetics"},{"orgOrder":0,"company":"Fujifilm Cellular Dynamics","sponsor":"BlueRock Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Licensing Agreement","leadProduct":"OpCT-001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Fujifilm Cellular Dynamics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Fujifilm Cellular Dynamics \/ BlueRock Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Fujifilm Cellular Dynamics \/ BlueRock Therapeutics"},{"orgOrder":0,"company":"Opus Genetics","sponsor":"Foundation Fighting Blindness","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"OPGx-RHO","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Opus Genetics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Opus Genetics \/ Foundation Fighting Blindness","highestDevelopmentStatusID":"4","companyTruncated":"Opus Genetics \/ Foundation Fighting Blindness"},{"orgOrder":0,"company":"Emendo Biotherapeutics","sponsor":"AnGes","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Acquisition","leadProduct":"EMD-101","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Emendo Biotherapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Emendo Biotherapeutics \/ AnGes","highestDevelopmentStatusID":"4","companyTruncated":"Emendo Biotherapeutics \/ AnGes"},{"orgOrder":0,"company":"Taysha Gene Therapies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"TSHA-103","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Taysha Gene Therapies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Taysha Gene Therapies \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Taysha Gene Therapies \/ Inapplicable"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Genetic Cures for Kids","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Charles River Laboratories, Inc \/ Genetic Cures for Kids","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ Genetic Cures for Kids"},{"orgOrder":0,"company":"Charles River Laboratories, Inc","sponsor":"Cure AP-4","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Charles River Laboratories, Inc","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Charles River Laboratories, Inc \/ Cure AP-4","highestDevelopmentStatusID":"4","companyTruncated":"Charles River Laboratories, Inc \/ Cure AP-4"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"VMCO-I","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Nanoscope Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Nanoscope Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Intergalactic Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"IG-002","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Intergalactic Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Intergalactic Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Intergalactic Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"reVision Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"REV-0100","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"reVision Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"reVision Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"reVision Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Abeona Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2023","type":"Inapplicable","leadProduct":"ABO-504","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Abeona Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Abeona Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Abeona Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Acadia Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2022","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Stoke Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Stoke Therapeutics \/ Acadia Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Stoke Therapeutics \/ Acadia Pharmaceuticals"},{"orgOrder":0,"company":"4M Therapeutics","sponsor":"SynGAP Research Fund","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Funding","leadProduct":"4MT2001","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"4M Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"4M Therapeutics \/ SynGAP Research Fund","highestDevelopmentStatusID":"4","companyTruncated":"4M Therapeutics \/ SynGAP Research Fund"},{"orgOrder":0,"company":"Diagonal Therapeutics","sponsor":"BVF Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Diagonal Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Diagonal Therapeutics \/ BVF Partners","highestDevelopmentStatusID":"4","companyTruncated":"Diagonal Therapeutics \/ BVF Partners"},{"orgOrder":0,"company":"Rezolute","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2023","type":"Inapplicable","leadProduct":"Ersodetug","moa":"Insulin allosteric site receptor","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rezolute","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Rezolute \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Rezolute \/ Inapplicable"},{"orgOrder":0,"company":"Castle Creek Biosciences","sponsor":"Paragon Biosciences","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"LV-FAH","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Castle Creek Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Castle Creek Biosciences \/ Paragon Biosciences","highestDevelopmentStatusID":"4","companyTruncated":"Castle Creek Biosciences \/ Paragon Biosciences"},{"orgOrder":0,"company":"Denovo Biopharma","sponsor":"Aytu BioScience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Enzastaurin","moa":"PKC-beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Denovo Biopharma","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Denovo Biopharma \/ Aytu BioScience","highestDevelopmentStatusID":"4","companyTruncated":"Denovo Biopharma \/ Aytu BioScience"},{"orgOrder":0,"company":"Rumpus Therapeutics","sponsor":"Aytu BioScience","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Licensing Agreement","leadProduct":"Enzastaurin","moa":"PKC-beta","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Rumpus Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Rumpus Therapeutics \/ Aytu BioScience","highestDevelopmentStatusID":"4","companyTruncated":"Rumpus Therapeutics \/ Aytu BioScience"},{"orgOrder":0,"company":"NephroDI Therapeutics","sponsor":"Xontogeny, LLC","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Financing","leadProduct":"NDI-5033","moa":"AMPK","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"NephroDI Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"NephroDI Therapeutics \/ Xontogeny, LLC","highestDevelopmentStatusID":"4","companyTruncated":"NephroDI Therapeutics \/ Xontogeny, LLC"},{"orgOrder":0,"company":"IPS Heart","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"GIVI-MPC","moa":"Dystrophin","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"IPS Heart","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"IPS Heart \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"IPS Heart \/ Inapplicable"},{"orgOrder":0,"company":"Repair Biotechnologies","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Inapplicable","leadProduct":"REP-0003","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Repair Biotechnologies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Repair Biotechnologies \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Repair Biotechnologies \/ Inapplicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Repair Biotechnologies

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                            Pharmex
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                            Repair Biotechnologies

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                            Lead Product(s) : REP-0003

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Repair Biotech Wins FDA Orphan Drug Tag for Homozygous Familial Hypercholesterolemia

                            Details : REP-0003 is a cutting-edge messenger RNA therapy that clears harmful excess free cholesterol inside cells while preserving essential cholesterol, for homozygous familial hypercholesterolemia.

                            Product Name : REP-0003

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            May 19, 2025

                            Lead Product(s) : REP-0003

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            IPS Heart

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                            Pharmex
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                            IPS Heart

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                            Lead Product(s) : GIVI-MPC

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            IPS HEART Granted Orphan Drug Designation in the EU for Becker Muscular Dystrophy

                            Details : GIVI-MPCs derived from pluripotent stem cells form new skeletal muscle with 100% full-length human dystrophin for DMD and BMD.

                            Product Name : GIVI-MPC

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            May 14, 2025

                            Lead Product(s) : GIVI-MPC

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Airna

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                            Airna

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                            Lead Product(s) : AIR-001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Venrock Healthcare Capital

                            Deal Size : $155.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Airna Raises $155 Million Series B Financing for Alpha-1 Antitrypsin Deficiency

                            Details : The financing aims to advance company's RNA editing product candidate AIR-001, which is being evaluated for the treatment of Alpha-1 antitrypsin deficiency.

                            Product Name : AIR-001

                            Product Type : Oligonucleotide

                            Upfront Cash : Undisclosed

                            April 01, 2025

                            Lead Product(s) : AIR-001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Venrock Healthcare Capital

                            Deal Size : $155.0 million

                            Deal Type : Series B Financing

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                            04

                            Prime Medicine

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                            Pharmex
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                            Prime Medicine

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                            Pharmex
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Prime Medicine Unveils Program for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

                            Details : This program leverages the Company’s universal liver lipid nanoparticle (LNP) to edit the E342K (Pi*Z) mutation in the SERPINA1 gene, the prevalent disease-causing mutation in AATD.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 18, 2025

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            HBM Alpha Therapeutics

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                            Pharmex
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                            HBM Alpha Therapeutics

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                            Pharmex
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                            Lead Product(s) : HAT001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : $395.0 million

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            HBM Alpha Enters Collaboration for Novel Endocrine Therapy Development

                            Details : Under the licensing agreement, the partner gains exclusive global rights, excluding Greater China (mainland China, Taiwan, Hong Kong, and Macau), to develop and commercialize HAT001 for treatting CAH.

                            Product Name : HAT001

                            Product Type : Antibody

                            Upfront Cash : Undisclosed

                            February 25, 2025

                            Lead Product(s) : HAT001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Undisclosed

                            Deal Size : $395.0 million

                            Deal Type : Licensing Agreement

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                            06

                            Porosome Therapeutics

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                            Pharmex
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                            Porosome Therapeutics

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                            Lead Product(s) : CF PT-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            FDA Grants Orphan Drug Designation to Revolutionary Cystic Fibrosis Therapy

                            Details : CF PT-101 is a porosome reconstitution therapy, which is currently being evaluated for the treatment of cystic fibrosis.

                            Product Name : CF PT-101

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 25, 2025

                            Lead Product(s) : CF PT-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            ElevateBio

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                            Pharmex
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                            ElevateBio

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                            Pharmex
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                            Lead Product(s) : LETI-101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            ElevateBio Announces Compelling Preclinical Data of LETI-101 for Huntington's Disease

                            Details : LETI-101 is a selective reducer of disease-causing mutant huntingtin (mHTT) protein, It is being evaluated in patients with huntington's disease.

                            Product Name : LETI-101

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 24, 2025

                            Lead Product(s) : LETI-101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Ensoma

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                            Pharmex
                            Not Confirmed

                            Ensoma

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                            Lead Product(s) : EN-374

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Ensoma's EN-374 Gets Rare Pediatric, Orphan Drug Status for Granulomatous Disease

                            Details : EN-374 is a CD46/CYBB modulator, gene therapy candidate, which is currently being evaluated for the treatment of X-linked chronic granulomatous disease (X-CGD).

                            Product Name : EN-374

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 13, 2025

                            Lead Product(s) : EN-374

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            TikunTX

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                            Pharmex
                            Not Confirmed

                            TikunTX

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                            Pharmex
                            Not Confirmed
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                            • WHO-GMP
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                            Lead Product(s) : BPN-36964

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Tikun Gets FDA Orphan, Rare Pediatric Designation for Dysautonomia

                            Details : BPN-36964 is a gene replacement therapy and a small molecule splicing modulator, being investgated for systemic treatment of familial dysautonomia.

                            Product Name : BPN-36964

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 23, 2025

                            Lead Product(s) : BPN-36964

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            IMMvention Therapeutix

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                            Pharmex
                            Not Confirmed

                            IMMvention Therapeutix

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                            Pharmex
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : IMM–004

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Novo Nordisk

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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                            IMMvention, Novo Nordisk Partner on Oral Sickle Cell, Chronic Disease Therapies

                            Details : The partnership will leverage IMMvention’s investigational small-molecule BACH1 inhibitors, IMM–004. It is being evaluating for the treatment of sickle cell disease.

                            Product Name : IMM–004

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            January 22, 2025

                            Lead Product(s) : IMM–004

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Novo Nordisk

                            Deal Size : Undisclosed

                            Deal Type : Partnership

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