22 Jan Splice Bio
Gerard Caelles, Chief Business Officer
April 10 | 11:00am | Salone dei Cavalieri, Section 2
Barcelona, Spain
(Private)
SpliceBio is a genetic medicines company harnessing its proprietary Protein Splicing platform to develop the next generation of gene therapies. Our platform offers the potential to address diseases that currently cannot be treated with gene therapies because the necessary gene is too large to be delivered by AAV vectors. Our lead program SB007 targets Stargardt disease, a genetic eye disease that causes vision loss in children and adults. SB007 has achieved high-level expression of functional, full-length human ABCA4 in vitro and in vivo after subretinal injection across species including rodents, minipigs and NHP macaques, with dose-dependent pharmacological activity in animal disease models of STGD1. SpliceBio is also developing SB008 for an IRD and SB011 for a monogenic CNS disease. The company raised a $57M series A in 2022 (UCB, Ysios, NEA, Gilde, Novartis and Asabys) and a partnership with Spark (Roche) in October 2023 to develop a novel gene therapy for an undisclosed IRD.
