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The financing will support the Phase 2 Alpestria-1 study and further profiling of EYP001 (vonafexor), a highly selective NR1H4 agonist, in other kidney diseases, such as Autosomal Dominant Polycystic Kidney Disease (ADPKD).
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).
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.
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.
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.
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.