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Inapplicable"},{"orgOrder":0,"company":"Stoke Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Inapplicable","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 \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Stoke Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Jnana Therapeutics","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2021","type":"Series B Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Jnana Therapeutics","amount2":0.050000000000000003,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.050000000000000003,"dosageForm":"Undisclosed","sponsorNew":"Jnana Therapeutics \/ RA Capital Management","highestDevelopmentStatusID":"4","companyTruncated":"Jnana Therapeutics \/ RA Capital Management"},{"orgOrder":0,"company":"GondolaBio","sponsor":"BridgeBio Pharma","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2024","type":"Demerger","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"GondolaBio","amount2":0.29999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.29999999999999999,"dosageForm":"Undisclosed","sponsorNew":"GondolaBio \/ BridgeBio Pharma","highestDevelopmentStatusID":"4","companyTruncated":"GondolaBio \/ BridgeBio Pharma"},{"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":"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":"Vico Therapeutics","sponsor":"Life Science Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Undisclosed","year":"2020","type":"Series A Financing","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Vico Therapeutics","amount2":0.029999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.029999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Vico Therapeutics \/ Life Science Partners","highestDevelopmentStatusID":"4","companyTruncated":"Vico Therapeutics \/ Life Science Partners"},{"orgOrder":0,"company":"Oak Bay Biosciences","sponsor":"FrontFundr","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"CANADA","productType":"Undisclosed","year":"2021","type":"Funding","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Oak Bay Biosciences","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oak Bay Biosciences \/ FrontFundr","highestDevelopmentStatusID":"4","companyTruncated":"Oak Bay Biosciences \/ FrontFundr"},{"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":0.96999999999999997,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.96999999999999997,"dosageForm":"Undisclosed","sponsorNew":"Stoke Therapeutics \/ Acadia Pharmaceuticals","highestDevelopmentStatusID":"4","companyTruncated":"Stoke Therapeutics \/ Acadia Pharmaceuticals"},{"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":0.13,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.13,"dosageForm":"Undisclosed","sponsorNew":"Diagonal Therapeutics \/ BVF Partners","highestDevelopmentStatusID":"4","companyTruncated":"Diagonal Therapeutics \/ BVF Partners"},{"orgOrder":0,"company":"Orchard Therapeutics","sponsor":"Pharming","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Undisclosed","year":"2021","type":"Collaboration","leadProduct":"OTL-105","moa":"C1 esterase","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Orchard Therapeutics","amount2":0.20999999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20999999999999999,"dosageForm":"Undisclosed","sponsorNew":"Orchard Therapeutics \/ Pharming","highestDevelopmentStatusID":"4","companyTruncated":"Orchard Therapeutics \/ Pharming"},{"orgOrder":0,"company":"Peptris Technologies","sponsor":"Revio Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"INDIA","productType":"Undisclosed","year":"2025","type":"Licensing Agreement","leadProduct":"RT001","moa":"Lipid peroxidation","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Peptris Technologies","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Peptris Technologies \/ Revio Therapeutics","highestDevelopmentStatusID":"4","companyTruncated":"Peptris Technologies \/ Revio Therapeutics"},{"orgOrder":0,"company":"Amplo Biotechnology","sponsor":"NIH-NIAMS","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Funding","leadProduct":"AMP-201","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Amplo Biotechnology","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Amplo Biotechnology \/ NIH-NIAMS","highestDevelopmentStatusID":"4","companyTruncated":"Amplo Biotechnology \/ NIH-NIAMS"},{"orgOrder":0,"company":"Moderna Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vaccine","year":"2022","type":"Inapplicable","leadProduct":"mRNA-3139","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Moderna Therapeutics","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Moderna Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"4","companyTruncated":"Moderna Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Wave Life Sciences","sponsor":"GSK","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":"Wave Life Sciences","amount2":3.3399999999999999,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":3.3399999999999999,"dosageForm":"Undisclosed","sponsorNew":"Wave Life Sciences \/ GSK","highestDevelopmentStatusID":"4","companyTruncated":"Wave Life Sciences \/ GSK"},{"orgOrder":0,"company":"MaxCyte","sponsor":"Prime Medicine","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2023","type":"Licensing Agreement","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"MaxCyte","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"MaxCyte \/ Prime Medicine","highestDevelopmentStatusID":"4","companyTruncated":"MaxCyte \/ Prime Medicine"},{"orgOrder":0,"company":"Scribe Therapeutics","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2025","type":"Expanded Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Scribe Therapeutics","amount2":1.24,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":1.24,"dosageForm":"Undisclosed","sponsorNew":"Scribe Therapeutics \/ Sanofi","highestDevelopmentStatusID":"4","companyTruncated":"Scribe Therapeutics \/ Sanofi"},{"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":"2Seventy Bio","sponsor":"Novo Nordisk","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":"2Seventy Bio","amount2":0.040000000000000001,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Undisclosed","sponsorNew":"2Seventy Bio \/ Novo Nordisk","highestDevelopmentStatusID":"4","companyTruncated":"2Seventy Bio \/ Novo Nordisk"},{"orgOrder":0,"company":"Precision BioSciences","sponsor":"Eli Lilly","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2020","type":"Collaboration","leadProduct":"Undisclosed","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Preclinical","graph3":"Precision BioSciences","amount2":0.56000000000000005,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Genetic Disease","amount2New":0.56000000000000005,"dosageForm":"Undisclosed","sponsorNew":"Precision BioSciences \/ Eli Lilly","highestDevelopmentStatusID":"4","companyTruncated":"Precision BioSciences \/ Eli Lilly"}]

    Find Drugs for Genetic Disease in Preclinical Development

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                            01

                            Repair Biotechnologies

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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 : Undisclosed

                            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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                            ChemOutsourcing
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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 : Undisclosed

                            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

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                            ChemOutsourcing
                            Not Confirmed

                            Augustine Therapeutics

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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 : Undisclosed

                            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

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                            ChemOutsourcing
                            Not Confirmed

                            Prime Medicine

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                            ChemOutsourcing
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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 : Undisclosed

                            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

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                            Peptris Technologies

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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 : Undisclosed

                            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

                            Paradox Immunotherapeutics

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                            ChemOutsourcing
                            Not Confirmed

                            Paradox Immunotherapeutics

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                            ChemOutsourcing
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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 : Undisclosed

                            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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                            08

                            ElevateBio

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                            ChemOutsourcing
                            Not Confirmed

                            ElevateBio

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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 : Undisclosed

                            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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                            09

                            Axovia Therapeutics

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                            Axovia Therapeutics

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                            ChemOutsourcing
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                            Lead Product(s) : AXV101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : A Race Against Blindness

                            Deal Size : $3.0 million

                            Deal Type : Funding

                            DEALS_DEV arrow-down

                            A Race Against Blindness Announces $2 Million Grant to Support Sight-Saving Clinical Trial

                            Details : The funding aims to advance the clinical development of company's AXV101, which is aimed at combating childhood blindness due to retinitis pigmentosa (RP) caused by Bardet-Biedl Syndrome 1.

                            Product Name : Undisclosed

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            February 05, 2025

                            Lead Product(s) : AXV101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : A Race Against Blindness

                            Deal Size : $3.0 million

                            Deal Type : Funding

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                            10

                            E-therapeutics

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                            ChemOutsourcing
                            Not Confirmed

                            E-therapeutics

                            United Kingdom arrow
                            ChemOutsourcing
                            Not Confirmed
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : ETX-148

                            Therapeutic Area : Genetic Disease

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            e-therapeutics to Present Preclinical Data on ETX-148 at EAHAD 2025

                            Details : ETX-148 is a potential pan-hemophilia rebalancing agent. It utilizes e-therapeutics' GalOmic GalNAc-siRNA technology to silence novel gene targets in hepatocytes.

                            Product Name : Undisclosed

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 05, 2025

                            Lead Product(s) : ETX-148

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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