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medicine","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Public Offering","leadProduct":"Bitopertin","moa":"GlyT1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.23000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Jefferies","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Jefferies"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Public Offering","leadProduct":"Bitopertin","moa":"GlyT1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Disc medicine","amount2":0.23000000000000001,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.23000000000000001,"dosageForm":"Oral Tablet","sponsorNew":"Disc medicine \/ Jefferies","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Jefferies"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2020","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Cantor Fitzgerald","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Cantor Fitzgerald","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Cantor Fitzgerald"},{"orgOrder":0,"company":"Agtc","sponsor":"H.C. Wainwright & Co","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Public Offering","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ H.C. Wainwright & Co","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ H.C. Wainwright & Co"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Inapplicable"},{"orgOrder":0,"company":"Agtc","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Acquisition","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Agtc","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Agtc \/ Syncona Limited","highestDevelopmentStatusID":"9","companyTruncated":"Agtc \/ Syncona Limited"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Syncona Limited","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2023","type":"Financing","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ Syncona Limited","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Syncona Limited"},{"orgOrder":0,"company":"Applied Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Govorestat","moa":"Aldose reductase","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Applied Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Applied Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Applied Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"Amyloid-beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Seelos Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Seelos Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Trehalose","moa":"Amyloid-beta","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Seelos Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Seelos Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"TCGX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Series B Financing","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ TCGX","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ TCGX"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2025","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Inapplicable","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Beacon Therapeutics","sponsor":"TCGX","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2024","type":"Series B Financing","leadProduct":"Laruparetigene Zovaparvovec","moa":"RPGR","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Beacon Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Beacon Therapeutics \/ TCGX","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ TCGX"},{"orgOrder":0,"company":"Biogen","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2021","type":"Inapplicable","leadProduct":"Cotoretigene Toliparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subretinal Injection","sponsorNew":"Biogen \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Biogen \/ Inapplicable"}]

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

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                            01

                            Sanofi

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

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Sanofi Halts Venglustat Clinical Programme in ADPKD

                            Details : The STAGED-PKD study was stopped for futility following an independent analysis of the annualized rate of change in total kidney volume (TKV) in patients receiving venglustat compared to placebo.

                            Product Name : GZ402671

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 06, 2021

                            Lead Product(s) : Venglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Regenxbio

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                            German Wound Congress
                            Not Confirmed

                            Regenxbio

                            U.S.A arrow
                            German Wound Congress
                            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) : RGX-202

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            REGENXBIO Reports Positive Biomarker Data from AFFINITY DUCHENNE® Trial

                            Details : RGX-202 is a cell and gene therapy drug that works by transferring the micro-dystrophin gene, It is infused intravenously for the treatment of Duchenne Muscular Dystrophy.

                            Product Name : RGX-202

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 19, 2025

                            Lead Product(s) : RGX-202

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            PTC Therapeutics

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                            German Wound Congress
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                            PTC Therapeutics

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                            German Wound Congress
                            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) : Vatiquinone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            PTC Announces FDA Acceptance and Priority Review for Vatiquinone NDA

                            Details : PTC743 (vatiquinone) is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), which is being developed for the treatment of Friedreich ataxia.

                            Product Name : PTC743

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 19, 2025

                            Lead Product(s) : Vatiquinone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Denali Therapeutics

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                            German Wound Congress
                            Not Confirmed

                            Denali Therapeutics

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                            German Wound Congress
                            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) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Denali Reports Primary & Long-Term Data on Tividenofusp Alfa in MPS II

                            Details : DNL310 (tividenofusp alfa), a fusion protein composed of IDS fused to Denali’s proprietary Enzyme transport vehicle. It is being evaluated in the treatment of Mucopolysaccharidosis Type II.

                            Product Name : DNL310

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            February 06, 2025

                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Beacon Therapeutics

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                            German Wound Congress
                            Not Confirmed

                            Beacon Therapeutics

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                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : Laruparetigene Zovaparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Beacon Therapeutics Receives RMAT Designation for AGTC-501

                            Details : AGTC-501 (laruparetigene zovaparvovec) is an XLRP GTPase regulator cell & gene therapy candidate, which is being evauated for the treatment of patients suffering from X-linked retinitis Pigmentosa.

                            Product Name : AGTC-501

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Inapplicable

                            January 28, 2025

                            Lead Product(s) : Laruparetigene Zovaparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Disc medicine

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                            German Wound Congress
                            Not Confirmed

                            Disc medicine

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                            German Wound Congress
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                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Jefferies

                            Deal Size : $225.5 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Disc Medicine Prices $225.5M Upsized Public Offering Of Common Stock

                            Details : The net proceeds from the offering to fund R&D product candidates, to support the potential commercialization of DISC-1459 (bitopertin) for erythropoietic protoporphyria & X-linked protoporphyria.

                            Product Name : DISC-1459

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            January 22, 2025

                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Jefferies

                            Deal Size : $225.5 million

                            Deal Type : Public Offering

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                            07

                            Disc medicine

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                            German Wound Congress
                            Not Confirmed

                            Disc medicine

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                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Disc Medicine Shares FDA Type C Meeting Success for Bitopertin in EPP, NDA Plans

                            Details : DISC-1459 (bitopertin) is an investigational, orall inhibitor of GlyT1 that is designed to modulate heme biosynthesis. It is being evaluated for the treatment of erythropoietic protoporphyria.

                            Product Name : DISC-1459

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 21, 2025

                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Disc medicine

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                            German Wound Congress
                            Not Confirmed

                            Disc medicine

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                            German Wound Congress
                            Not Confirmed
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                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Jefferies

                            Deal Size : $230.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Disc Medicine Announces Proposed Public Offering of Common Stock and Pre-Funded Warrants

                            Details : The net proceeds from the offering to fund R&D of its current or additional product candidates, to support the potential commercialization of DISC-1459 (bitopertin) for erythropoietic protoporphyria.

                            Product Name : DISC-1459

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            January 21, 2025

                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Jefferies

                            Deal Size : $230.0 million

                            Deal Type : Public Offering

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                            09

                            Regenxbio

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                            German Wound Congress
                            Not Confirmed

                            Regenxbio

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                            German Wound Congress
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : RGX-121

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Nippon Shinyaku

                            Deal Size : $810.0 million

                            Deal Type : Partnership

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                            RegenXBio Sells Hunter Syndrome Gene Therapy Commercial Rights

                            Details : The partnership aims to advance the clinical development of RGX-121, a potential one-time AAV therapeutic for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.

                            Product Name : RGX-121

                            Product Type : Cell and Gene therapy

                            Upfront Cash : $110.0 million

                            January 14, 2025

                            Lead Product(s) : RGX-121

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Nippon Shinyaku

                            Deal Size : $810.0 million

                            Deal Type : Partnership

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                            10

                            Denali Therapeutics

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                            German Wound Congress
                            Not Confirmed

                            Denali Therapeutics

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                            German Wound Congress
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                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Denali Receives FDA Breakthrough Designation for Tividenofusp Alfa in Hunter Syndrome

                            Details : DNL310 (tividenofusp alfa), a fusion protein composed of IDS fused to Denali’s proprietary Enzyme transport vehicle. It is being evaluated in the treatment of Mucopolysaccharidosis Type II.

                            Product Name : DNL310

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            January 08, 2025

                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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