Drugs in Dev. Genetic Disease
IND Enabling
France 
Lead Product(s) : Vonafexor
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : OrbiMed Advisors
Deal Size : $42.5 million
Deal Type : Series C Financing
ENYO Pharma Secures €39M in Series C and FDA Clearance for Vonafexor
Details : Financing will support the Phase 2 Alpestria-1 study and profiling of EYP001 (vonafexor), a selective NR1H4 agonist, in Autosomal Dominant Polycystic Kidney Disease (ADPKD).
Product Name : EYP001
Product Type : Other Small Molecule
Upfront Cash : Undisclosed
March 01, 2024
Lead Product(s) : Vonafexor
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : OrbiMed Advisors
Deal Size : $42.5 million
Deal Type : Series C Financing
Lead Product(s) : ATA-100
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : ATA-100 (GNT0006), a single-administration gene therapy candidate for LGMD2I/R9, delivers a normal copy of the gene for production of FKRP proteins, which is investigated for the treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9).
Product Name : ATA-100
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
June 09, 2023
Lead Product(s) : ATA-100
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : SPVN06
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Jeito Capital
Deal Size : $75.0 million
Deal Type : Series B Financing
Details : Proceeds from the financing will be used to fund the first-in-human trials of the Company’s two lead gene-independent assets, SPVN06 and SPVN20, as well as the development of genome editing assets through its collaboration with Intellia Therapeutics.
Product Name : SPVN06
Product Type : Cell and Gene therapy
Upfront Cash : Undisclosed
September 14, 2022
Lead Product(s) : SPVN06
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Jeito Capital
Deal Size : $75.0 million
Deal Type : Series B Financing
Lead Product(s) : SPVN06
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress which, acting synergistically, aim at slowing or stopping the degeneration of cone photoreceptors.
Product Name : SPVN06
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
January 12, 2022
Lead Product(s) : SPVN06
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : LYS-GM101
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Details : First regulatory authorization to initiate this global, multi-center, single-arm, two-stage, adaptive-design clinical trial of LYS-GM101 in patients with a diagnosis of early or late infantile GM1 gangliosidosis.
Product Name : LYS-GM101
Product Type : Cell and Gene therapy
Upfront Cash : Inapplicable
November 01, 2021
Lead Product(s) : LYS-GM101
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
Lead Product(s) : SPVN06
Therapeutic Area : Genetic Disease
Study Phase : IND Enabling
Sponsor : 4BIO Capital
Deal Size : $51.9 million
Deal Type : Financing
SparingVision Raises €44.5 Million and Appoints Stéphane Boissel as Chief Executive Officer
Details : Proceeds from the financing will be primarily used to advance the development of SparingVision’s breakthrough treatment SPVN06 for the mutation-agnostic treatment of retinitis pigmentosa.
Product Name : SPVN06
Product Type : Cell and Gene therapy
Upfront Cash : Undisclosed
October 21, 2020
Lead Product(s) : SPVN06
Therapeutic Area : Genetic Disease
Highest Development Status : IND Enabling
Sponsor : 4BIO Capital
Deal Size : $51.9 million
Deal Type : Financing
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