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SB-011
- Development stage
- Preclinical
- Lead developer
- SpliceBio
- Modality
- Gene Therapies
01
Overview
SB-011 is a preclinical-stage gene therapy candidate developed by SpliceBio for the treatment of monogenic central nervous system (CNS) diseases. The program leverages SpliceBio's proprietary protein splicing platform, which utilizes dual adeno-associated virus (AAV) vectors and engineered inteins to overcome the limited packaging capacity of standard AAV vectors. This technology allows for the delivery of large genes by splitting them into two parts, each carried by a separate vector, and reconstituting the full-length functional protein within the target cells via protein splicing. SB-011 is currently in the preclinical development stage, and its specific biological target and exact CNS indication have not been publicly disclosed.
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