Sepul Bio, an innovative business unit of Théa, dedicated to the advancement of RNA therapies for inherited retinal diseases, has announced the dosing of its first two participants in the Phase 3 HYPERION clinical trial for sepofarsen, in individuals with CEP290-associated Leber Congenital Amaurosis Type 10 (LCA10).
HYPERION
HYPERION, or SB-110-007, is a two-year double-masked, randomized, placebo-controlled, paired-eye study to evaluate the efficacy, safety and tolerability of sepofarsen in 32 participants with LCA10 due to the c.2991+1655A>G (p.Cys998X) variant in the CEP290 gene.
SEPOFARSEN
Sepofarsen is an experimental mRNA therapy designed to improve visual function for patients with LCA10. Sepofarsen targets a specific genetic variant (c.2991+1655A>G) in the CEP290 gene. This variant stops the cell from producing an essential protein needed for the cells in the retina to function. By addressing this variant, sepofarsen aims to restore cell function in the retina.
Sepofarsen is in Phase 3 clinical development.
The Sepul Bio messenger RNA or ‘mRNA’ therapies belong to a class of compounds known as antisense oligonucleotides (AONs) or ASO treatments. These mRNA therapies utilize short, synthetically designed RNA sequences that are chemically modified to enhance their stability and cellular uptake. mRNA is a type of single-stranded RNA involved in protein creation.
Comments from the Community:
“The launch of the HYPERION study is an exciting milestone for the LCA community,” said Courtney Coates, Executive Director, Hope in Focus. “We have been eagerly waiting for the re-initiation of the sepofarsen program, and today’s announcement reflects renewed hope for the community. As a community partner, we look forward to working alongside the Sepul Bio team as they advance RNA therapies to potentially help children, adults, and families affected by CEP290-associated LCA10.”
