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KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Antibiotic","year":"2025","type":"Inapplicable","leadProduct":"Sirolimus","moa":"mTOR","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Biodexa Pharmaceuticals \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Inapplicable"},{"orgOrder":0,"company":"Healx","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"UNITED KINGDOM","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"HLX-1502","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Healx","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Tablet","sponsorNew":"Healx \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Healx \/ Inapplicable"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Braidwell LP","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Other Small Molecule","year":"2025","type":"Public Offering","leadProduct":"Sevasemten","moa":"Myosin","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0.20000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.20000000000000001,"dosageForm":"Oral Tablet","sponsorNew":"Edgewise Therapeutics \/ Braidwell LP","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Braidwell LP"}]

    Find Drugs for Genetic Disease in Phase II Clinical Development

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                            01

                            AbbVie Inc

                            U.S.A
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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

                            Edgewise Therapeutics

                            U.S.A arrow
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                            Edgewise Therapeutics

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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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                            03

                            Healx

                            United Kingdom arrow
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                            Healx

                            United Kingdom arrow
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                            Lead Product(s) : HLX-1502

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Healx Announces First Patient Dosed in Phase 2 Trial Evaluating HLX-1502

                            Details : HLX-1502 is being evaluated through a mid-stage clinical trial study for the treatment of patients suffering from neurofibromatosis Type 1.

                            Product Name : HLX-1502

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 24, 2025

                            Lead Product(s) : HLX-1502

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            AceLink Therapeutics

                            U.S.A arrow
                            AAN
                            Not Confirmed

                            AceLink Therapeutics

                            U.S.A arrow
                            AAN
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                            Lead Product(s) : AL01211

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            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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                            05

                            Shionogi

                            Japan arrow
                            AAN
                            Not Confirmed

                            Shionogi

                            Japan arrow
                            AAN
                            Not Confirmed
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                            Lead Product(s) : Zatolmilast

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Jordan’s Guardian Angels

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

                            DEALS_DEV arrow-down

                            Shionogi, Jordan’s Guardian Angels Begin Jordan’s Syndrome Drug Study

                            Details : The collaboration aims to advance the clinical development of BPN14770 (zatolmilast), which is being evaluated in the mid-stage clinical trial studies for the treatment of Jordan's Syndrome.

                            Product Name : BPN14770

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            February 04, 2025

                            Lead Product(s) : Zatolmilast

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Jordan’s Guardian Angels

                            Deal Size : Undisclosed

                            Deal Type : Collaboration

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                            06

                            Cumberland Pharmaceuticals

                            U.S.A arrow
                            AAN
                            Not Confirmed

                            Cumberland Pharmaceuticals

                            U.S.A arrow
                            AAN
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                            Lead Product(s) : Ifetroban

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Cumberland’s Phase 2 FIGHT DMD Trial Shows Breakthrough Results in DMD Heart Disease

                            Details : Dyscorban (ifetroban) is a once-daily oral medication that works by blocking the thromboxane receptor, being investigated for duchenne muscular dystrophy.

                            Product Name : Dyscorban

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 04, 2025

                            Lead Product(s) : Ifetroban

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            PepGen

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                            Not Confirmed

                            PepGen

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                            AAN
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                            Lead Product(s) : PGN-EDO51

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            PepGen Announces CONNECT Program Updates

                            Details : PGN-EDO51 blocks the inclusion of exon 51, restoring functional, dystrophin production. It is under developement for the treatment of duchenne muscular dystrophy.

                            Product Name : PGN-EDO51

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            January 29, 2025

                            Lead Product(s) : PGN-EDO51

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            NMD PHARMA

                            Denmark arrow
                            AAN
                            Not Confirmed

                            NMD PHARMA

                            Denmark arrow
                            AAN
                            Not Confirmed
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                            Lead Product(s) : NMD670

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            NMD Pharma’s NMD670 Gains FDA Orphan Status for Charcot-Marie-Tooth

                            Details : NMD670 is a first-in-class small molecule inhibitor of ClC-1. It is being developed by NMD Pharma for the treatment of patients with charcot-marie-tooth disease.

                            Product Name : NMD670

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 06, 2025

                            Lead Product(s) : NMD670

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            PepGen

                            U.S.A arrow
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                            Not Confirmed

                            PepGen

                            U.S.A arrow
                            AAN
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                            Lead Product(s) : PGN-EDO51

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            PepGen Announces U.S. Clinical Hold On PGN-EDO51 for Duchenne Muscular Dystrophy

                            Details : PGN-EDO51 blocks the inclusion of exon 51, restoring functional, dystrophin production. It trial for the treatment of duchenne muscular dystrophy has been put on hold.

                            Product Name : PGN-EDO51

                            Product Type : Oligonucleotide

                            Upfront Cash : Inapplicable

                            December 16, 2024

                            Lead Product(s) : PGN-EDO51

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Attralus

                            U.S.A arrow
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                            Attralus

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                            Lead Product(s) : AT-02

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Attralus AT-02 Receives FDA Orphan Drug Designation for ATTR

                            Details : AT-02, a humanized IgG1 monoclonal antibody, company’s lead pan-amyloid removal therapeutic candidate for transthyretin-associated amyloidosis.

                            Product Name : AT-02

                            Product Type : Antibody

                            Upfront Cash : Inapplicable

                            December 11, 2024

                            Lead Product(s) : AT-02

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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