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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":"Oxford Biomedica","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2020","type":"Licensing Agreement","leadProduct":"SAR422459","moa":"ABCR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Oxford Biomedica","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Oxford Biomedica \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"Oxford Biomedica \/ Sanofi"},{"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":"Cell and Gene Therapy Catapult","sponsor":"INmune Bio","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Cell and Gene therapy","year":"2025","type":"Partnership","leadProduct":"Umbilical Cord Derived Mesenchymal Stromal Cell","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Cell and Gene Therapy Catapult","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Cell and Gene Therapy Catapult \/ INmune Bio","highestDevelopmentStatusID":"8","companyTruncated":"Cell and Gene Therapy Catapult \/ INmune Bio"},{"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"}]

    Find Drugs for Genetic Disease in Phase II Clinical Development

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                            01

                            AbbVie Inc

                            U.S.A
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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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

                            Syros Pharmaceuticals

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed

                            Syros Pharmaceuticals

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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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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                            03

                            Edgewise Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed

                            Edgewise Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            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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                            04

                            Ascendis Pharma

                            Denmark arrow
                            AACR Annual meeting
                            Not Confirmed

                            Ascendis Pharma

                            Denmark arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
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                            Lead Product(s) : Navepegritide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Ascendis Submits NDA for TransCon CNP for Achondroplasia in Kids

                            Details : TransCon CNP (c-type natriuretic peptide prodrug) is a FGFR 3 inhibitor. It is being evaluated for the treatment of children with achondroplasia (ACH) aged upto 3 years.

                            Product Name : TransCon CNP

                            Product Type : Peptide

                            Upfront Cash : Inapplicable

                            March 31, 2025

                            Lead Product(s) : Navepegritide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Tempest Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed

                            Tempest Therapeutics

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

                            DEALS_DEV arrow-down

                            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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                            06

                            Biodexa Pharmaceuticals

                            United Kingdom arrow
                            AACR Annual meeting
                            Not Confirmed

                            Biodexa Pharmaceuticals

                            United Kingdom arrow
                            AACR Annual meeting
                            Not Confirmed
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                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Biodexa Succeeds in FDA Type C Meeting for eRapa Phase 3 in FAP

                            Details : eRapa, a proprietary encapsulated form of rapamycin, also known as sirolimus, is being developed for the treatment of familial adenomatous polyposis (FAP).

                            Product Name : eRapa

                            Product Type : Antibiotic

                            Upfront Cash : Inapplicable

                            March 10, 2025

                            Lead Product(s) : Sirolimus

                            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
                            AACR Annual meeting
                            Not Confirmed

                            Nanoscope Therapeutics

                            U.S.A arrow
                            AACR Annual meeting
                            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

                            DEALS_DEV arrow-down

                            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

                            Biodexa Pharmaceuticals

                            United Kingdom arrow
                            AACR Annual meeting
                            Not Confirmed

                            Biodexa Pharmaceuticals

                            United Kingdom arrow
                            AACR Annual meeting
                            Not Confirmed
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                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Biodexa's FAP Drug Gains FDA Fast Track After Positive Phase 2 Results

                            Details : eRapa, a proprietary encapsulated form of rapamycin, also known as sirolimus, is being developed for the treatment of familial adenomatous polyposis (FAP).

                            Product Name : eRapa

                            Product Type : Antibiotic

                            Upfront Cash : Inapplicable

                            February 12, 2025

                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Spinogenix

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed

                            Spinogenix

                            U.S.A arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • 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

                            DEALS_DEV arrow-down

                            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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                            10

                            Biodexa Pharmaceuticals

                            United Kingdom arrow
                            AACR Annual meeting
                            Not Confirmed

                            Biodexa Pharmaceuticals

                            United Kingdom arrow
                            AACR Annual meeting
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Biodexa Receives US FDA Fast Track Designation for eRapa in Familial Adenomatous Polyposis

                            Details : eRapa, a proprietary encapsulated form of rapamycin, also known as sirolimus, is being developed for the treatment of familial adenomatous polyposis (FAP).

                            Product Name : eRapa

                            Product Type : Antibiotic

                            Upfront Cash : Inapplicable

                            February 10, 2025

                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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