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Disease","amount2New":0.68999999999999995,"dosageForm":"Infusion","sponsorNew":"Avidity Biosciences \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"Avidity Biosciences \/ Leerink Partners"},{"orgOrder":0,"company":"Vertex Pharmaceuticals","sponsor":"Undisclosed","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Miscellaneous","year":"2025","type":"Inapplicable","leadProduct":"VX-407","moa":"Undisclosed","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Vertex Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Tablet","sponsorNew":"Vertex Pharmaceuticals \/ Undisclosed","highestDevelopmentStatusID":"8","companyTruncated":"Vertex Pharmaceuticals \/ Undisclosed"},{"orgOrder":0,"company":"PepGen","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Public Offering","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0.10000000000000001,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Leerink Partners"},{"orgOrder":0,"company":"PepGen","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Public Offering","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Leerink Partners"},{"orgOrder":0,"company":"PepGen","sponsor":"Leerink Partners","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Oligonucleotide","year":"2025","type":"Public Offering","leadProduct":"PGN-EDODM1","moa":"Exon51 skipping","graph1":"Genetic Disease","graph2":"Phase II","graph3":"PepGen","amount2":0.12,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0.12,"dosageForm":"Infusion","sponsorNew":"PepGen \/ Leerink Partners","highestDevelopmentStatusID":"8","companyTruncated":"PepGen \/ Leerink Partners"}]

    Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                            01

                            AbbVie Inc

                            U.S.A
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : ABBV-CLS-628

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Study to Assess Adverse Events and Effectiveness of IntraVenous Infusions of ABBV-CLS-628 in Adult Participa...

                            Details : ABBV-CLS-628 is a drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.

                            Product Name : Undisclosed

                            Product Type : Undisclosed

                            Upfront Cash : Inapplicable

                            March 30, 2025

                            Lead Product(s) : ABBV-CLS-628

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            AbbVie Inc

                            U.S.A
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Galicaftor,Navocaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Sionna Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

                            DEALS_DEV arrow-down

                            Sionna Expands CF Pipeline with AbbVie License Agreement

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

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            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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                            03

                            AbbVie Inc

                            U.S.A
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Galicaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Study to Evaluate Adverse Events and Change in Disease Activity With Oral Capsules of Galicaftor/Navocaftor/AB...

                            Details : Galicaftor is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            April 21, 2021

                            Lead Product(s) : Galicaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            AbbVie Inc

                            U.S.A
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Navocaftor

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            A Phase 2 Study of ABBV-3067 Alone and in Combination With ABBV-2222

                            Details : ABBV-3067 is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Cystic Fibrosis.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 31, 2019

                            Lead Product(s) : Navocaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Avidity Biosciences

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Avidity Biosciences

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Delpacibart Zotadirsen

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Leerink Partners

                            Deal Size : $690.0 million

                            Deal Type : Public Offering

                            DEALS_DEV arrow-down

                            Avidity Biosciences Announces Closing of Upsized Public Offering of Common Stock

                            Details : Avidity intends to use the net proceeds to advance the its three late-stage clinical programs, including AOC 1044 (delpacibart zotadirsen), designed to deliver phosphorodiamidate morpholino oligomers.

                            Product Name : AOC 1044

                            Product Type : Antibody-oligonucleotide Conjugate

                            Upfront Cash : Undisclosed

                            September 16, 2025

                            Lead Product(s) : Delpacibart Zotadirsen

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Leerink Partners

                            Deal Size : $690.0 million

                            Deal Type : Public Offering

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                            06

                            Vertex Pharmaceuticals

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Vertex Pharmaceuticals

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : VX-407

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants

                            Details : VX-407 is a Small Molecule drug candidate drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Polycystic Kidney, Autosomal Dominant.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            September 08, 2025

                            Lead Product(s) : VX-407

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Tisento Therapeutics

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Tisento Therapeutics

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Zagociguat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)

                            Details : Zagociguat is a Other Small Molecule drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of MELAS Syndrome.

                            Product Name : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            May 07, 2025

                            Lead Product(s) : Zagociguat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Syros Pharmaceuticals

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            Syros Pharmaceuticals

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Tamibarotene

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Rege Nephro

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

                            DEALS_DEV arrow-down

                            Rege Nephro Acquires Tamibarotene Assets for U.S. Trials

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

                            Product Name : Undisclosed

                            Product Type : Vitamins/Minerals/Inorganic Salts

                            Upfront Cash : Undisclosed

                            April 15, 2025

                            Lead Product(s) : Tamibarotene

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Rege Nephro

                            Deal Size : Undisclosed

                            Deal Type : Acquisition

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                            09

                            jCyte

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            jCyte

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Human Retinal Progenitor Cell

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Safety of a Single, Intravitreal Injection of 8.8M jCell (Famzeretcel) in Retinitis Pigmentosa (RP)

                            Details : JCell (Human Retinal Progenitor Cell) is a Cell and Gene Therapy drug candidate, which is currently being evaluated in phase II clinical studies for the treatment of Retinitis Pigmentosa.

                            Product Name : jCell

                            Product Type : Cell & Gene Therapy

                            Upfront Cash : Inapplicable

                            April 06, 2025

                            Lead Product(s) : Human Retinal Progenitor Cell

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            AceLink Therapeutics

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed

                            AceLink Therapeutics

                            U.S.A arrow
                            OTS 2025
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : AL01211

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            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 : Undisclosed

                            Product Type : Miscellaneous

                            Upfront Cash : Inapplicable

                            February 07, 2025

                            Lead Product(s) : AL01211

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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