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Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Regenxbio","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2023","type":"Inapplicable","leadProduct":"RGX-202","moa":"Solute carrier family 6 member 8 (SLC6A8)","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Regenxbio","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Regenxbio \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Regenxbio \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2020","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Seelos Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Carbohydrate","year":"2022","type":"Inapplicable","leadProduct":"Trehalose","moa":"TFEB","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Seelos Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Infusion","sponsorNew":"Seelos Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Seelos Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Biogen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2021","type":"Inapplicable","leadProduct":"Cotoretigene Toliparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Biogen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Biogen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Biogen \/ Undisclosed"},{"orgOrder":0,"company":"CSL Behring","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Undisclosed","year":"2015","type":"Inapplicable","leadProduct":"CSL689","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"CSL Behring","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"CSL Behring \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"CSL Behring \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2023","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2025","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Ocugen","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2024","type":"Inapplicable","leadProduct":"OCU410ST","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Ocugen","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Ocugen \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Ocugen \/ Undisclosed"},{"orgOrder":0,"company":"Sarepta Therapeutics","sponsor":"Lysogene","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Cell & Gene Therapy","year":"2022","type":"Termination","leadProduct":"Olenasufligene Relduparvovec","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Sarepta Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Injection","sponsorNew":"Sarepta Therapeutics \/ Lysogene","highestDevelopmentStatusID":"9","companyTruncated":"Sarepta Therapeutics \/ Lysogene"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Skyhawk Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Skyhawk Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Skyhawk Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Undisclosed"},{"orgOrder":0,"company":"Skyhawk Therapeutics","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2024","type":"Inapplicable","leadProduct":"SKY-0515","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Skyhawk Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Skyhawk Therapeutics \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Skyhawk Therapeutics \/ Undisclosed"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            Crinetics Pharmaceuticals

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                            OTS 2025
                            Not Confirmed

                            Crinetics Pharmaceuticals

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                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Atumelnant

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study in Pediatric Participants With Congenital Adrenal Hyperplasia (Balance-CAH)

                            Details : Atumelnant is a Small Molecule drug candidate drug candidate, which is currently being evaluated in Phase II/ Phase III clinical studies for the treatment of Adrenal Hyperplasia, Congenital.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 08, 2025

                            Lead Product(s) : Atumelnant

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            Glycomine

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                            OTS 2025
                            Not Confirmed

                            Glycomine

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                            OTS 2025
                            Not Confirmed
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                            Virtual Booth Boost your online visibility by uploading your products, APIs, FDFs, intermediates, excipients, and services for free on PharmaCompass. Rank higher among suppliers and expand your reach across the internet efficiently and cost-effectively.

                            Lead Product(s) : GLM101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : CTI Life Sciences Fund

                            Deal Size : $115.0 million

                            Deal Type : Series C Financing

                            DEALS_DEV arrow-down

                            Glycomine Raises $115M to Advance GLM101 Into Phase 2b Trial for PMM2-CDG

                            Details : The proceeds will advance GLM101, a first-in-class mannose-1-phosphate replacement therapy, is in development for phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG).

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Undisclosed

                            April 16, 2025

                            Lead Product(s) : GLM101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : CTI Life Sciences Fund

                            Deal Size : $115.0 million

                            Deal Type : Series C Financing

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                            03

                            Denali Therapeutics

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                            OTS 2025
                            Not Confirmed

                            Denali Therapeutics

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                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Denali Starts BLA Filing for Tividenofusp Alfa in Hunter Syndrome Treatment

                            Details : DNL310 (tividenofusp alfa) is composed of the iduronate 2-sulfatase enzyme fused to Denali’s proprietary ETV, designed to deliver IDS, addressing symptoms of Hunter syndrome.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            April 02, 2025

                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Glycomine

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                            OTS 2025
                            Not Confirmed

                            Glycomine

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                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : GLM101

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG

                            Details : GLM101 is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Congenital Disorders of Glycosylation.

                            Product Name : Undisclosed

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            March 24, 2025

                            Lead Product(s) : GLM101

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Regenxbio

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Regenxbio

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                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : RGX-202

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            REGENXBIO Reports Positive Biomarker Data from AFFINITY DUCHENNE® Trial

                            Details : RGX-202 is a cell and gene therapy drug that works by transferring the micro-dystrophin gene, It is infused intravenously for the treatment of Duchenne Muscular Dystrophy.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 19, 2025

                            Lead Product(s) : RGX-202

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            QED Therapeutics

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                            OTS 2025
                            Not Confirmed

                            QED Therapeutics

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                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Infigratinib

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            An Interventional Study of Infigratinib in Children With Hypochondroplasia

                            Details : Infigratinib is a Other Small Molecule drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Hypochondroplasia.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 12, 2025

                            Lead Product(s) : Infigratinib

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Skyhawk Therapeutics

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                            OTS 2025
                            Not Confirmed

                            Skyhawk Therapeutics

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                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : SKY-0515

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Study of SKY-0515 for Safety, Efficacy, and Pharmacodynamics in Participants With Huntington's Disease

                            Details : SKY-0515 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II/ phase III clinical studies for the treatment of Huntington Disease.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            March 12, 2025

                            Lead Product(s) : SKY-0515

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            PTC Therapeutics

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                            OTS 2025
                            Not Confirmed

                            PTC Therapeutics

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                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Vatiquinone

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            PTC Announces FDA Acceptance and Priority Review for Vatiquinone NDA

                            Details : PTC743 (vatiquinone) is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), which is being developed for the treatment of Friedreich ataxia.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            February 19, 2025

                            Lead Product(s) : Vatiquinone

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Denali Therapeutics

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                            OTS 2025
                            Not Confirmed

                            Denali Therapeutics

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                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            Denali Reports Primary & Long-Term Data on Tividenofusp Alfa in MPS II

                            Details : DNL310 (tividenofusp alfa), a fusion protein composed of IDS fused to Denali’s proprietary Enzyme transport vehicle. It is being evaluated in the treatment of Mucopolysaccharidosis Type II.

                            Product Name : Undisclosed

                            Product Type : Protein

                            Upfront Cash : Inapplicable

                            February 06, 2025

                            Lead Product(s) : Tividenofusp Alpha

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Disc medicine

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                            OTS 2025
                            Not Confirmed

                            Disc medicine

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                            OTS 2025
                            Not Confirmed
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                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II/ Phase III

                            Sponsor : Jefferies

                            Deal Size : $225.5 million

                            Deal Type : Public Offering

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                            Disc Medicine Prices $225.5M Upsized Public Offering Of Common Stock

                            Details : The net proceeds from the offering to fund R&D product candidates, to support the potential commercialization of DISC-1459 (bitopertin) for erythropoietic protoporphyria & X-linked protoporphyria.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Undisclosed

                            January 22, 2025

                            Lead Product(s) : Bitopertin

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II/ Phase III

                            Sponsor : Jefferies

                            Deal Size : $225.5 million

                            Deal Type : Public Offering

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