SB009

SB009 is a preclinical gene therapy candidate being developed by Splice Therapeutics (also known as Splice Bio) for the treatment of monogenic inherited retinal dystrophy. The program utilizes the company's proprietary Protein Splicing platform, which is designed to overcome the packaging capacity limitations of standard adeno-associated virus (AAV) vectors. By employing engineered inteins, the technology allows for the delivery of large therapeutic genes that exceed the typical 4.7 kb limit of AAV. The large gene is split into two halves, each packaged into a separate AAV vector; upon co-infection of the target retinal cells, the inteins facilitate the reconstitution of the full-length, functional protein. SB009 is specifically aimed at addressing genetic eye disorders caused by mutations in large genes that were previously inaccessible to conventional AAV-based gene replacement therapies.