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                            Lead Product(s) : ABBV-CLS-628

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Calico Life Sciences

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                            Lead Product(s) : ABBV-CLS-628

                            Therapeutic Area : Genetic Disease

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                            Sponsor : Calico Life Sciences

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            02

                            AbbVie Inc

                            U.S.A
                            ESCRS
                            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 : Other Small Molecule

                            Upfront Cash : Undisclosed

                            July 16, 2024

                            Lead Product(s) : Galicaftor,Navocaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Sionna Therapeutics

                            Deal Size : Undisclosed

                            Deal Type : Licensing Agreement

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                            03

                            AbbVie Inc

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

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            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 : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 21, 2021

                            Lead Product(s) : Galicaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            AbbVie Inc

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

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            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 : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 31, 2019

                            Lead Product(s) : Navocaftor

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            BioMarin Pharmaceutical

                            U.S.A arrow
                            ESCRS
                            Not Confirmed

                            BioMarin Pharmaceutical

                            U.S.A arrow
                            ESCRS
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Vosoritide

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Icon Plc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Vosoritide vs Placebo in Children with Hypochondroplasia Aged 0 to 36 Months

                            Details : Vosoritide is a Peptide drug candidate, which is currently being evaluated in Phase II clinical studies for the treatment of Hypochondroplasia.

                            Product Name : Undisclosed

                            Product Type : Peptide

                            Upfront Cash : Inapplicable

                            August 17, 2025

                            Lead Product(s) : Vosoritide

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Icon Plc

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Sionna Therapeutics

                            U.S.A arrow
                            ESCRS
                            Not Confirmed

                            Sionna Therapeutics

                            U.S.A arrow
                            ESCRS
                            Not Confirmed
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                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : SION-719

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Undisclosed

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            SION-719 Safety, Tolerability, PD/PK Added to Trikafta

                            Details : SION-719 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 : Other Small Molecule

                            Upfront Cash : Inapplicable

                            August 06, 2025

                            Lead Product(s) : SION-719

                            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
                            ESCRS
                            Not Confirmed

                            Tisento Therapeutics

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

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            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 : Undisclosed

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 07, 2025

                            Lead Product(s) : Zagociguat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            PTC Therapeutics

                            U.S.A arrow
                            ESCRS
                            Not Confirmed

                            PTC Therapeutics

                            U.S.A arrow
                            ESCRS
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : Votoplam

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            PTC518 PIVOT-HD Study Achieves Primary Endpoint

                            Details : PTC518 (votoplam) is a small molecule splicing modifier. It is being evaluated for the treatment of Stage 2 and Stage 3 Huntington's disease.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 05, 2025

                            Lead Product(s) : Votoplam

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Tempest Therapeutics

                            U.S.A arrow
                            ESCRS
                            Not Confirmed

                            Tempest Therapeutics

                            U.S.A arrow
                            ESCRS
                            Not Confirmed
                            • fda
                            • EDQM
                            • WHO-GMP
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                            Lead Product(s) : TPST-1495

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Tempest gets FDA Orphan Drug Tag for TPST-1495 in Treating FAP

                            Details : TPST-1495, the company’s novel dual receptor inhibitor of prostaglandin (PGE2) signaling, in a Phase 2 clinical trial for the treatment of patients with familial adenomatous polyposis.

                            Product Name : Undisclosed

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            April 21, 2025

                            Lead Product(s) : TPST-1495

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Syros Pharmaceuticals

                            U.S.A arrow
                            ESCRS
                            Not Confirmed

                            Syros Pharmaceuticals

                            U.S.A arrow
                            ESCRS
                            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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