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Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"vMCO-010","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Nanoscope Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene 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Therapeutics \/ Verana Health","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Verana Health"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2021","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2020","type":"Inapplicable","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ Inapplicable"},{"orgOrder":0,"company":"Scholar Rock","sponsor":"J.P. Morgan","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2020","type":"Public Offering","leadProduct":"Apitegromab","moa":"MSTN activation","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Scholar Rock","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Scholar Rock \/ J.P. Morgan","highestDevelopmentStatusID":"8","companyTruncated":"Scholar Rock \/ J.P. Morgan"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell and Gene therapy","year":"2022","type":"Inapplicable","leadProduct":"vMCO-010","moa":"MCO","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravitreal Injection","sponsorNew":"Nanoscope Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Bain Capital Life Sciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2023","type":"Series B Financing","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Bain Capital Life Sciences","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Bain Capital Life Sciences"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Gildeuretinol Acetate","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Stargazer Pharmaceuticals","sponsor":"Organovo Holdings","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2020","type":"Series A Financing","leadProduct":"STG-001","moa":"RBP4","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Stargazer Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Stargazer Pharmaceuticals \/ Organovo Holdings","highestDevelopmentStatusID":"8","companyTruncated":"Stargazer Pharmaceuticals \/ Organovo Holdings"},{"orgOrder":0,"company":"Attralus","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Antibody","year":"2024","type":"Inapplicable","leadProduct":"AT-02","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Attralus","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Attralus \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Inapplicable"},{"orgOrder":0,"company":"Syros Pharmaceuticals","sponsor":"Rege Nephro","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Vitamins\/Minerals\/Inorganic Salts","year":"2025","type":"Acquisition","leadProduct":"Tamibarotene","moa":"RAR-alpha","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Syros Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Syros Pharmaceuticals \/ Rege Nephro","highestDevelopmentStatusID":"8","companyTruncated":"Syros Pharmaceuticals \/ Rege Nephro"},{"orgOrder":0,"company":"BioMarin Pharmaceutical","sponsor":"Spruce Biosciences","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Protein","year":"2025","type":"Acquisition","leadProduct":"Tralesinidase Alfa","moa":"Heparan sulfate","graph1":"Genetic Disease","graph2":"Phase II","graph3":"BioMarin Pharmaceutical","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intracerebroventricular Infusion","sponsorNew":"BioMarin Pharmaceutical \/ Spruce Biosciences","highestDevelopmentStatusID":"8","companyTruncated":"BioMarin Pharmaceutical \/ Spruce Biosciences"},{"orgOrder":0,"company":"Solaxa","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Fampridine","moa":"Potassium channel","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Solaxa","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet, Orally Disintegrating","sponsorNew":"Solaxa \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Solaxa \/ Inapplicable"},{"orgOrder":0,"company":"Tempest Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"TPST-1495","moa":"PGE2","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Tempest Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Tempest Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Tempest 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":"Votoplam","moa":"HTT mRNA","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"PTC Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Inapplicable"},{"orgOrder":0,"company":"Esperion Therapeutics","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Bempedoic Acid","moa":"||ACL\/NPC1L1","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Esperion Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet, Film Coated","sponsorNew":"Esperion Therapeutics \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Esperion Therapeutics \/ Inapplicable"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            AbbVie Inc

                            U.S.A
                            Specialty & Agro Chemical
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                            Lead Product(s) : Galicaftor,Navocaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Sionna Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Sionna Expands CF Pipeline with AbbVie License Agreement

                            Details : Under the terms of the agreement, Sionna will assume all development for ABBV-2222 (galicaftor), which is being evaluated in combination with navocaftor for the treatment of Cystic Fibrosis.

                            Product Name : ABBV-2222

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            July 16, 2024

                            Lead Product(s) : Galicaftor,Navocaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Sionna Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            02

                            PTC Therapeutics

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                            Specialty & Agro Chemical
                            Not Confirmed

                            PTC Therapeutics

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                            Specialty & Agro Chemical
                            Not Confirmed
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                            Lead Product(s) : Votoplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            PTC518 PIVOT-HD Study Achieves Primary Endpoint

                            Details : PTC518 (votoplam) is a small molecule splicing modifier. It is being evaluated for the treatment of Stage 2 and Stage 3 Huntington's disease.

                            Product Name : PTC518

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 05, 2025

                            Lead Product(s) : Votoplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Syros Pharmaceuticals

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                            Specialty & Agro Chemical
                            Not Confirmed

                            Syros Pharmaceuticals

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                            Specialty & Agro Chemical
                            Not Confirmed
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                            Lead Product(s) : Tamibarotene

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Rege Nephro

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            DEALS_DEV arrow-down

                            Rege Nephro Acquires Tamibarotene Assets for U.S. Trials

                            Details : Through the acquisition, Rege Nephro will leverage RN-014 (tamibarotene) related clinical and non-clinical assets from Syros. It is being evaluated for autosomal dominant polycystic kidney disease.

                            Product Name : RN-014

                            Product Type : Vitamins/Minerals/Inorganic Salts

                            Upfront Cash : Undisclosed

                            April 15, 2025

                            Lead Product(s) : Tamibarotene

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Rege Nephro

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

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                            04

                            BioMarin Pharmaceutical

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                            Specialty & Agro Chemical
                            Not Confirmed

                            BioMarin Pharmaceutical

                            U.S.A arrow
                            Specialty & Agro Chemical
                            Not Confirmed
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                            Lead Product(s) : Tralesinidase Alfa

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Spruce Biosciences

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            DEALS_DEV arrow-down

                            Spruce Unveils New Strategy and Acquires Drug for Sanfilippo Syndrome Type B

                            Details : Through the acquisition, Spruce will focus on the clinical development of AX 250 (tralesinidase alfa) enzyme replacement therapy (TA-ERT) for the treatment of Sanfilippo Syndrome Type B.

                            Product Name : AX 250

                            Product Type : Protein

                            Upfront Cash : Undisclosed

                            April 15, 2025

                            Lead Product(s) : Tralesinidase Alfa

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Spruce Biosciences

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

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                            05

                            Edgewise Therapeutics

                            U.S.A arrow
                            Specialty & Agro Chemical
                            Not Confirmed

                            Edgewise Therapeutics

                            U.S.A arrow
                            Specialty & Agro Chemical
                            Not Confirmed
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                            Lead Product(s) : Sevasemten

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Braidwell LP

                            Deal Size : $200.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Edgewise Announces $200M Underwritten Offering of Common Stock

                            Details : Edgewise intends to use the net proceeds from the offering to support the potential U.S. commercial launch of EDG-5506 (sevasemten) in patients with Becker muscular dystrophy.

                            Product Name : EDG-5506

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            April 02, 2025

                            Lead Product(s) : Sevasemten

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Braidwell LP

                            Deal Size : $200.0 million

                            Deal Type : Public Offering

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                            06

                            Tempest Therapeutics

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                            Specialty & Agro Chemical
                            Not Confirmed

                            Tempest Therapeutics

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                            Specialty & Agro Chemical
                            Not Confirmed
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                            Lead Product(s) : TPST-1495

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            FDA Clears Phase 2 TPST-1495 Trial for Familial Adenomatous Polyposis

                            Details : TPST-1495, the company’s novel dual receptor inhibitor of prostaglandin (PGE2) signaling, in a Phase 2 clinical trial for the treatment of patients with familial adenomatous polyposis.

                            Product Name : TPST-1495

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 13, 2025

                            Lead Product(s) : TPST-1495

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Nanoscope Therapeutics

                            U.S.A arrow
                            Specialty & Agro Chemical
                            Not Confirmed

                            Nanoscope Therapeutics

                            U.S.A arrow
                            Specialty & Agro Chemical
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Sonpiretigene Isteparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Verana Health

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            Verana & Nanoscope Collaborate on Retinitis Pigmentosa Research

                            Details : The collaboration aims fot the development and commercialization of MCO-010 (sonpiretigene isteparvovec), an optogenetic gene therapy designed to restore vision and potentially slow neurodegeneration.

                            Product Name : MCO-010

                            Product Type : Cell and Gene therapy

                            Upfront Cash : Undisclosed

                            February 25, 2025

                            Lead Product(s) : Sonpiretigene Isteparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Verana Health

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            08

                            Spinogenix

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                            Specialty & Agro Chemical
                            Not Confirmed

                            Spinogenix

                            U.S.A arrow
                            Specialty & Agro Chemical
                            Not Confirmed
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                            Lead Product(s) : SPG601

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Spinogenix Shows Strong Phase 2 Efficacy for SPG601 in Fragile X Syndrome

                            Details : SPG601 is a novel small molecule BK channel activator that works by binding to BK channels and increasing their activation to restore synaptic function to address core symptoms of Fragile X Syndrome.

                            Product Name : SPG601

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 11, 2025

                            Lead Product(s) : SPG601

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            AceLink Therapeutics

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                            Specialty & Agro Chemical
                            Not Confirmed

                            AceLink Therapeutics

                            U.S.A arrow
                            Specialty & Agro Chemical
                            Not Confirmed
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                            Lead Product(s) : AL01211

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            AceLink Shows Phase 2 Data on AL01211 for Fabry Disease at WORLD 2025

                            Details : AL01211 is a novel, oral, non-brain penetrant GCS inhibitor which is under development to treat glycosphingolipid storage diseases, including Fabry Disease and Type 1 Gaucher Disease.

                            Product Name : AL01211

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 07, 2025

                            Lead Product(s) : AL01211

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Spinogenix

                            U.S.A arrow
                            Specialty & Agro Chemical
                            Not Confirmed

                            Spinogenix

                            U.S.A arrow
                            Specialty & Agro Chemical
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : SPG601

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            FDA Grants Fast Track Designation to Spinogenix's SPG601 for Fragile X Syndrome

                            Details : SPG601 is a novel small molecule BK channel activator that works by binding to BK channels and increasing their activation to restore synaptic function to address core symptoms of Fragile X Syndrome.

                            Product Name : SPG601

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 13, 2025

                            Lead Product(s) : SPG601

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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