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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":"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":"Tablet","sponsorNew":"Disc medicine \/ Jefferies","highestDevelopmentStatusID":"9","companyTruncated":"Disc medicine \/ Jefferies"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Tividenofusp Alfa","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Disc medicine","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small 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\/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Beacon Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Denali Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Inapplicable","leadProduct":"Tividenofusp Alfa","moa":"IDS","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Denali Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Denali Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"Denali Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Vatiquinone","moa":"15-LOX","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Solution","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Collaboration","leadProduct":"Fazirsiran","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"9","companyTruncated":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical"},{"orgOrder":0,"company":"Arrowhead Pharmaceuticals","sponsor":"Takeda Pharmaceutical","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2020","type":"Licensing Agreement","leadProduct":"Fazirsiran","moa":"Alpha-1 antitrypsin","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Arrowhead Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Subcutaneous Injection","sponsorNew":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical","highestDevelopmentStatusID":"9","companyTruncated":"Arrowhead Pharmaceuticals \/ Takeda Pharmaceutical"}]

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

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Investigational data from long-term, follow-up studies showed that olipudase alfa provided sustained improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes over time in patients with ASMD.

                          Product Name : GZ402665

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          February 09, 2022

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          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 : Small molecule

                          Upfront Cash : Not Applicable

                          June 01, 2021

                          Lead Product(s) : Venglustat

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : Olipudase alfa

                          Therapeutic Area : Genetic Disease

                          Study Phase : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

                          Product Name : Undisclosed

                          Product Type : Large molecule

                          Upfront Cash : Not Applicable

                          January 30, 2020

                          Lead Product(s) : Olipudase alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          04

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          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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                          05

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          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 Alfa

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          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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                          07

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          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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                          08

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          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

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          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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                          10

                          Pittcon 2025
                          Not Confirmed
                          Pittcon 2025
                          Not Confirmed

                          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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