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SpliceBio Secures $135 Million Series B Financing to Advance Lead Program SB-007 in Stargardt Disease and Expand Pipeline of Genetic Medicines

SpliceBio, a gene therapy company, secured $135 million in Series B funding to advance its lead gene therapy, SB-007, for Stargardt disease into clinical trials (Phase 1/2 ASTRA and observational POLARIS studies). SB-007 is the first dual AAV gene therapy for Stargardt's to receive FDA and UK regulatory clearance. Stargardt disease, an inherited eye disorder leading to blindness, currently lacks approved treatments. SpliceBio's SB-007 aims to correct the underlying genetic cause by delivering a functional ABCA4 protein using their proprietary Protein Splicing platform, which allows them to deliver large genes (too big for traditional AAVs) by splitting them into two parts that reassemble inside the cell. The funding will also accelerate their pipeline of gene therapies for other eye, neurological, and undisclosed conditions.

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