SB-008

SB-008 is an investigational gene therapy being developed by SpliceBio for the treatment of monogenic inherited retinal diseases (IRDs). The program utilizes SpliceBio's proprietary Protein Splicing platform, which is designed to overcome the cargo capacity limitations of standard adeno-associated virus (AAV) vectors. This platform employs dual AAV vectors to deliver fragments of a large gene, which are then reassembled into a full-length, functional protein within the target retinal cells using engineered inteins. SB-008 is currently in the IND-enabling stage of development. While the specific target gene for SB-008 has not been publicly disclosed, it follows the same technological approach as the company's lead program, SB-007.