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FrontFundr"},{"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":"Versantis","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"SWITZERLAND","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"VS-01","moa":"Ammonia clearance","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Versantis","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intraperitoneal Injection","sponsorNew":"Versantis \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Versantis \/ Inapplicable"},{"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":"Vivet Therapeutics","sponsor":"French Government","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Funding","leadProduct":"VTX-806","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ French Government","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ French Government"},{"orgOrder":0,"company":"Vivet Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"FRANCE","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"VTX-806","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vivet Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Vivet Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Vivet Therapeutics \/ Inapplicable"},{"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 Drugs for Genetic Disease in Preclinical Development

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                            01

                            Repair Biotechnologies

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Repair Biotechnologies

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                            Discovery Europe
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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

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            IPS Heart

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                            Discovery Europe
                            Not Confirmed
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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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                            Discovery Europe
                            Not Confirmed

                            Airna

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                            Discovery Europe
                            Not Confirmed
                            • fda
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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

                            Augustine Therapeutics

                            Belgium arrow
                            Discovery Europe
                            Not Confirmed

                            Augustine Therapeutics

                            Belgium arrow
                            Discovery Europe
                            Not Confirmed
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                            Lead Product(s) : AGT-100216

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Novo Holdings

                            Deal Size : $84.8 million

                            Deal Type : Series A Financing

                            DEALS_DEV arrow-down

                            Augustine Raises EUR 78M/USD 85M in Oversubscribed Series A Financing

                            Details : The financing will support the company's lead product AGT-100216, which is being evaluated for the treatment of Charcot-Marie-Tooth.

                            Product Name : AGT-100216

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            March 24, 2025

                            Lead Product(s) : AGT-100216

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Novo Holdings

                            Deal Size : $84.8 million

                            Deal Type : Series A Financing

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                            05

                            Prime Medicine

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Prime Medicine

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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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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                            06

                            Peptris Technologies

                            India arrow
                            Discovery Europe
                            Not Confirmed

                            Peptris Technologies

                            India arrow
                            Discovery Europe
                            Not Confirmed
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                            Lead Product(s) : RT001

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Revio Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Peptris Technologies out-licenses AI-discovered drug candidate to Revio Therapeutics

                            Details : Under the licensing agreement, Revio will hold commercialisation rights to PEPR124 (RT001) in all markets except in BRICS countries, which will be retained by Peptris.

                            Product Name : PEPR124

                            Product Type : Undisclosed

                            Upfront Cash : Undisclosed

                            March 17, 2025

                            Lead Product(s) : RT001

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Revio Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            07

                            HBM Alpha Therapeutics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            HBM Alpha Therapeutics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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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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                            08

                            Porosome Therapeutics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            Porosome Therapeutics

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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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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                            09

                            Paradox Immunotherapeutics

                            Canada arrow
                            Discovery Europe
                            Not Confirmed

                            Paradox Immunotherapeutics

                            Canada arrow
                            Discovery Europe
                            Not Confirmed
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                            Lead Product(s) : LX-96

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Symbiosis Pharmaceutical Services

                            Deal Size : $10.0 million

                            Deal Type : Financing

                            DEALS_DEV arrow-down

                            Paradox Gets $10M from SymBiosis to Advance Protein Misfolding Disease Therapies

                            Details : The financing will be used to advance Paradox’s therapeutic pipeline, which includes LX-96, a monoclonal antibody designed for the treatment of late-stage immunoglobulin light chain (AL) amyloidosis.

                            Product Name : LX-96

                            Product Type : Antibody

                            Upfront Cash : Undisclosed

                            February 25, 2025

                            Lead Product(s) : LX-96

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Symbiosis Pharmaceutical Services

                            Deal Size : $10.0 million

                            Deal Type : Financing

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                            10

                            ElevateBio

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed

                            ElevateBio

                            U.S.A arrow
                            Discovery Europe
                            Not Confirmed
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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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