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Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Wave Life Sciences \/ Inapplicable"},{"orgOrder":0,"company":"Atsena Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"ATSN-201","moa":"RS1","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Atsena Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Atsena Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Atsena Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Uniqure","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"AMT-130","moa":"mHTT production","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Uniqure","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intrastriatal Infusion","sponsorNew":"Uniqure \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Uniqure \/ Inapplicable"},{"orgOrder":0,"company":"Dyne Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody-oligonucleotide Conjugate","year":"2025","type":"Inapplicable","leadProduct":"DYNE-251","moa":"DMD exon 51","graph1":"Genetic Disease","graph2":"Phase I\/ Phase II","graph3":"Dyne Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph I\/II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Dyne Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"7","companyTruncated":"Dyne Therapeutics \/ Inapplicable"}]

    Find Drugs for Genetic Disease in Phase I/ Phase II Clinical Development

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                            01

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Translate Bio Provides MRT5005 Program Updates

                            Details : Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date.

                            Product Name : MRT5005

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            March 26, 2020

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Sanofi

                            France
                            BIO International Convention
                            Exhibiting
                            • fda
                            • EDQM
                            • WHO-GMP
                            Virtual BoothSanofi is a pioneer in Diabetes Solutions, Human Vaccines, Innovative Drugs, Consumer Healthcare, and the new Genzyme.

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Translate Bio Receives FDA Fast Track Designation for MRT5005 for the Treatment of Cystic Fibrosis

                            Details : First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.

                            Product Name : MRT5005

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            February 26, 2020

                            Lead Product(s) : MRT5005

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Dyne Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed

                            Dyne Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
                            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) : DYNE-251

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Dyne gets EMA Orphan Drug Status for DYNE-251 in Duchenne Dystrophy

                            Details : DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for people living with DMD who are amenable to exon 51 skipping.

                            Product Name : DYNE-251

                            Product Type : Antibody-oligonucleotide Conjugate

                            Upfront Cash : Inapplicable

                            April 24, 2025

                            Lead Product(s) : DYNE-251

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Atsena Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed

                            Atsena Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : ATSN-201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Bain Capital

                            Deal Size : $150.0 million

                            Deal Type : Series C Financing

                            DEALS_DEV arrow-down

                            Atsena Raises $150M Series C to Advance Ocular Gene Therapy

                            Details : The proceeds from the financing will be used to advance Atsena’s lead program, ATSN-201, for the treatment of X-linked retinoschisis (XLRS).

                            Product Name : ATSN-201

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            April 02, 2025

                            Lead Product(s) : ATSN-201

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Bain Capital

                            Deal Size : $150.0 million

                            Deal Type : Series C Financing

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                            05

                            Wave Life Sciences

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed

                            Wave Life Sciences

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : WVE-N531

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Wave Reports Functional Gains in DMD After 48 Weeks of WVE-N531

                            Details : WVE-N531 is being evaluated in the mid-stage clinical trials studies for the treatment of patients suffering from Duchenne muscular dystrophy.

                            Product Name : WVE-N531

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            March 26, 2025

                            Lead Product(s) : WVE-N531

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Atsena Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed

                            Atsena Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : ATSN-201

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Atsena Gets FDA Fast Track for ATSN-201 Gene Therapy in X-Linked Retinoschisis

                            Details : ATSN-201 is an investigational gene therapy that leverages one of the company’s novel spreading capsids, AAV.SPR, it is being developed for the treatment of X-linked retinoschisis (XLRS).

                            Product Name : ATSN-201

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            March 12, 2025

                            Lead Product(s) : ATSN-201

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Hemab Therapeutics

                            Denmark arrow
                            AACR Annual meeting
                            Not Confirmed

                            Hemab Therapeutics

                            Denmark arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : HMB-002

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Hemab Starts Velora Pioneer Trial for Von Willebrand Disease

                            Details : HMB-002 is a monovalent human antibody, which is currently being evaluated for the treatment of von willebrand disease.

                            Product Name : HMB-002

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            February 27, 2025

                            Lead Product(s) : HMB-002

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Solid Biosciences

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed

                            Solid Biosciences

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : SGT-003

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Solid Biosciences Reports Positive Data from Next-Generation Duchenne Gene Therapy

                            Details : SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding microdystrophin. It is being evaluated for duchenne muscular dystrophy.

                            Product Name : SGT-003

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 18, 2025

                            Lead Product(s) : SGT-003

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Sangamo Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed

                            Sangamo Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Isaralgagene Civaparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sangamo Updates Phase 1/2 STAAR Study, Showing Fabry Disease Benefits

                            Details : ST-920 (isaralgagene civaparvovec) is an AAV2/6 human alpha-Gal A gene therapy product candidate in patients for the treatment of Fabry disease.

                            Product Name : ST-920

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 06, 2025

                            Lead Product(s) : Isaralgagene Civaparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Uniqure

                            Netherlands arrow
                            AACR Annual meeting
                            Not Confirmed

                            Uniqure

                            Netherlands arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : AMT-191

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            uniQure Completes AMT-191 Enrollment, Receives Favorable Review

                            Details : AMT-191 is a alpha-GLA transgene Transference cell and gene therapy drug candidate, delivered via intravenous infusion for the treatment of Fabry disease.

                            Product Name : AMT-191

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            February 03, 2025

                            Lead Product(s) : AMT-191

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I/ Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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