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Disease","amount2New":0.40000000000000002,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Adage Capital Partners LP","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Adage Capital Partners LP"},{"orgOrder":0,"company":"Astria Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"STAR-0215","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Astria Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Astria Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Astria Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Nanoscope Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Cell and Gene therapy","year":"2024","type":"Not Applicable","leadProduct":"Sonpiretigene Isteparvovec","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Nanoscope Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Nanoscope Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Nanoscope Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Attralus","sponsor":"Alpha Wave Ventures","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Financing","leadProduct":"AT-02","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Attralus","amount2":0.059999999999999998,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.059999999999999998,"dosageForm":"Infusion","sponsorNew":"Attralus \/ Alpha Wave Ventures","highestDevelopmentStatusID":"8","companyTruncated":"Attralus \/ Alpha Wave Ventures"},{"orgOrder":0,"company":"PepGen","sponsor":"RA Capital Management","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"PGN-EDO51","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Infusion","sponsorNew":"PepGen \/ RA Capital Management","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ RA Capital Management"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"Nomlabofusp","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0.17000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.17000000000000001,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Leerink Partners"},{"orgOrder":0,"company":"Visen Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Peptide","year":"2023","type":"Not Applicable","leadProduct":"C-type Natriuretic Peptide Prodrug","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Visen Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Visen Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Visen Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Nizubaglustat","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Azafaros \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Not Applicable"},{"orgOrder":0,"company":"NS Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2023","type":"Not Applicable","leadProduct":"Brogidirsen","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"NS Pharma","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"NS Pharma \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"NS Pharma \/ Not Applicable"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"PGN-EDO51","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Not Applicable"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"EDG-5506","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"PGN-EDO51","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Not Applicable"},{"orgOrder":0,"company":"Avidity Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Del-desiran","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Avidity Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Nomlabofusp","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Nizubaglustat","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Azafaros \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Not Applicable"},{"orgOrder":0,"company":"PepGen","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"PGN-EDO51","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Not Applicable"},{"orgOrder":0,"company":"Spruce Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Tildacerfont","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spruce Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Spruce Biosciences \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Spruce Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Sevasemten","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Calliditas Therapeutics AB","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Setanaxib","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Calliditas Therapeutics AB","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Calliditas Therapeutics AB \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Calliditas Therapeutics AB \/ Not Applicable"},{"orgOrder":0,"company":"Harmony Biosciences","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Pitolisant Hydrochloride","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Harmony Biosciences","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet, Film Coated","sponsorNew":"Harmony Biosciences \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Harmony Biosciences \/ Not Applicable"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sevasemten","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Edgewise Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Eloxx Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Exaluren Sulfate","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Eloxx Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Eloxx Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Eloxx Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Edgewise Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sevasemten","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Edgewise Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Edgewise Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Edgewise Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Centessa Pharmaceuticals","sponsor":"Goldman Sachs","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"SerpinPC","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Centessa Pharmaceuticals","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.10000000000000001,"dosageForm":"Injection","sponsorNew":"Centessa Pharmaceuticals \/ Goldman Sachs","highestDevelopmentStatusID":"8","companyTruncated":"Centessa Pharmaceuticals \/ Goldman Sachs"},{"orgOrder":0,"company":"Emtora Biosciences","sponsor":"Biodexa Pharmaceuticals","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Sirolimus","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Emtora Biosciences","amount2":0.040000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.040000000000000001,"dosageForm":"Tablet","sponsorNew":"Emtora Biosciences \/ Biodexa Pharmaceuticals","highestDevelopmentStatusID":"8","companyTruncated":"Emtora Biosciences \/ Biodexa Pharmaceuticals"},{"orgOrder":0,"company":"Crinetics Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Atumelnant","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Crinetics Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Crinetics Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Crinetics Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Aardvark Therapeutics","sponsor":"Cantor Fitzgerald","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Series C Financing","leadProduct":"Denatonium Acetate","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Aardvark Therapeutics","amount2":0.089999999999999997,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.089999999999999997,"dosageForm":"","sponsorNew":"Aardvark Therapeutics \/ Cantor Fitzgerald","highestDevelopmentStatusID":"8","companyTruncated":"Aardvark Therapeutics \/ Cantor Fitzgerald"},{"orgOrder":0,"company":"Alkeus Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Gildeuretinol Acetate","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Alkeus Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Alkeus Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Alkeus Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Inhibrx","sponsor":"Sanofi","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Acquisition","leadProduct":"SAR447537","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Inhibrx","amount2":2.2000000000000002,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":2.2000000000000002,"dosageForm":"Infusion","sponsorNew":"Inhibrx \/ Sanofi","highestDevelopmentStatusID":"8","companyTruncated":"Inhibrx \/ Sanofi"},{"orgOrder":0,"company":"Larimar Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Not Applicable","leadProduct":"Nomlabofusp","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Larimar Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Larimar Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Larimar Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Mereo BioPharma Group","sponsor":"Jefferies","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Large molecule","year":"2024","type":"Public Offering","leadProduct":"Setrusumab","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Mereo BioPharma Group","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Mereo BioPharma Group \/ Jefferies","highestDevelopmentStatusID":"8","companyTruncated":"Mereo BioPharma Group \/ Jefferies"},{"orgOrder":0,"company":"Neuren Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Pyrrolo(1,2-a)pyrazine-1,4-dione, hexahydro-8a-(2-propenyl)-, (8aR)-","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Neuren Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Neuren Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Neuren Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"PTC518","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PTC Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"PTC Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"PTC Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Ladenburg Thalmann","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Private Placement","leadProduct":"Sirolimus","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0.01,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.01,"dosageForm":"","sponsorNew":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Ladenburg Thalmann"},{"orgOrder":0,"company":"Spinogenix","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"SPG601","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Spinogenix","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Spinogenix \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Spinogenix \/ Not Applicable"},{"orgOrder":0,"company":"Rege Nephro","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Tamibarotene","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Rege Nephro","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Rege Nephro \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Rege Nephro \/ Not Applicable"},{"orgOrder":0,"company":"Biodexa Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Sirolimus","moa":"","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Biodexa Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Biodexa Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Biodexa Pharmaceuticals \/ Not Applicable"}]

    Find Drugs for Genetic Disease in Phase II Clinical Development

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                            01

                            AbbVie Inc

                            U.S.A
                            Fi Europe 2024
                            Not Confirmed
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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.

                            Brand Name : ABBV-2222

                            Molecule Type : 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

                            Oranda Therapeutics

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                            Fi Europe 2024
                            Not Confirmed

                            Oranda Therapeutics

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                            Fi Europe 2024
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                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Undisclosed

                            Deal Type : Series A Financing

                            DEALS_DEV arrow-down

                            Oranda Therapeutics Secures Series A Funding for Rare Disease Therapies

                            Details : The proceeds will be used for the commercialisation of novel medicine undergoing evaluation for treatment of hyperphagia in patients with Prader-Willi syndrome (PWS).

                            Brand Name : Undisclosed

                            Molecule Type : Undisclosed

                            Upfront Cash : Undisclosed

                            November 12, 2024

                            Lead Product(s) : Undisclosed

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Undisclosed

                            Deal Type : Series A Financing

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                            03

                            Astria Therapeutics

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                            Fi Europe 2024
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                            Astria Therapeutics

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                            Fi Europe 2024
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                            Lead Product(s) : Navenibart

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            EU Grants Orphan Status for Astria’s Navenibart in Hereditary Angioedema

                            Details : STAR-0215 (navenibart) is a monoclonal antibody inhibitor of plasma kallikrein designed to provide long-acting attack prevention for hereditary angioedema.

                            Brand Name : STAR-0215

                            Molecule Type : Large molecule

                            Upfront Cash : Not Applicable

                            October 16, 2024

                            Lead Product(s) : Navenibart

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            04

                            Beacon Therapeutics

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                            Fi Europe 2024
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                            Beacon Therapeutics

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                            Fi Europe 2024
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                            Lead Product(s) : Laruparetigene Zovaparvovec

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Beacon Shows Positive 24-Month Data from AGTC-501 Phase 2 in XLRP

                            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.

                            Brand Name : AGTC-501

                            Molecule Type : Cell and Gene therapy

                            Upfront Cash : Not Applicable

                            October 15, 2024

                            Lead Product(s) : Laruparetigene Zovaparvovec

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            05

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            Fi Europe 2024
                            Not Confirmed

                            F. Hoffmann-La Roche

                            Switzerland arrow
                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Chugai Pharmaceutical

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Evrysdi Two-Year Data in SMA: Most Children Can Sit, Stand, and Walk Independently

                            Details : Evrysdi (risdiplam) is a survival of motor neuron 2 (SMN2) splicing modifier, which is being evaluated for the treatment of infants with pre-symptomatic spinal muscular atrophy.

                            Brand Name : Evrysdi

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            October 14, 2024

                            Lead Product(s) : Risdiplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Chugai Pharmaceutical

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            06

                            OMEICOS Therapeutics GmbH

                            Germany arrow
                            Fi Europe 2024
                            Not Confirmed

                            OMEICOS Therapeutics GmbH

                            Germany arrow
                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : OMT-28

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            OMEICOS Completes Enrollment of PMD-OPTION Study in Primary Mitochondrial Disease

                            Details : OMT-28 is a first-in-class PPARα agonist small molecule drug candidate, which is currently being evaluated for the treatment of primary mitochondrial disease.

                            Brand Name : OMT-28

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            September 26, 2024

                            Lead Product(s) : OMT-28

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            07

                            Biodexa Pharmaceuticals

                            United Kingdom arrow
                            Fi Europe 2024
                            Not Confirmed

                            Biodexa Pharmaceuticals

                            United Kingdom arrow
                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : CPRIT

                            Deal Size : $17.0 million

                            Deal Type : Funding

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                            Company Pays $3M Final Match For $17M CPRIT Grant Access

                            Details : The proceeds will be used to fund company's upcoming Phase 3 registrational study of eRapa, a proprietary oral tablet formulation of rapamycin, in Familial Adenomatous Polyposis.

                            Brand Name : eRapa

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            September 16, 2024

                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : CPRIT

                            Deal Size : $17.0 million

                            Deal Type : Funding

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                            08

                            Biodexa Pharmaceuticals

                            United Kingdom arrow
                            Fi Europe 2024
                            Not Confirmed

                            Biodexa Pharmaceuticals

                            United Kingdom arrow
                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Ladenburg Thalmann & Co

                            Deal Size : $5.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Announces Pricing of $5M Registered Direct Offering and Concurrent Private Placement

                            Details : The net proceeds will be used to advance the clinical development of eRapa (rapamycin), also known as sirolimus. It is being evaluated for treating Familial Adenomatous Polyposis.

                            Brand Name : eRapa

                            Molecule Type : Small molecule

                            Upfront Cash : Undisclosed

                            July 19, 2024

                            Lead Product(s) : Sirolimus

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Ladenburg Thalmann & Co

                            Deal Size : $5.0 million

                            Deal Type : Public Offering

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                            09

                            Abliva

                            Sweden arrow
                            Fi Europe 2024
                            Not Confirmed

                            Abliva

                            Sweden arrow
                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : KL1333

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            Abliva Report Positive Interim Analysis of FALCON Study of KL1333 in Mitochondrial Disease

                            Details : Abliva lead product candidate KL1333 is being evaluated in the mid-stage clinical trial studies with patients for the treatment of Primary Mitochondrial Disease.

                            Brand Name : KL1333

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            July 18, 2024

                            Lead Product(s) : KL1333

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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                            10

                            Alkeus Pharmaceuticals

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed

                            Alkeus Pharmaceuticals

                            U.S.A arrow
                            Fi Europe 2024
                            Not Confirmed
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                            Lead Product(s) : Gildeuretinol Acetate

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

                            DEALS_DEV arrow-down

                            Alkeus Pharmaceuticals Shows Progress in Stargardt Disease with TEASE-3 Study Update

                            Details : ALK-001 (gildeuretinol acetate) is a novel molecule created as a specialized form of deuterated vitamin A. It is under phase 2 clinical development for the treatment of Stargardt Disease.

                            Brand Name : ALK-001

                            Molecule Type : Small molecule

                            Upfront Cash : Not Applicable

                            July 18, 2024

                            Lead Product(s) : Gildeuretinol Acetate

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Not Applicable

                            Deal Size : Not Applicable

                            Deal Type : Not Applicable

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