BLD055

BLD055 is a novel peptide-modified adeno-associated virus (PM-AAV) capsid engineered for high-efficiency gene delivery to the central nervous system, particularly the cerebellum and associated motor structures. Developed by Latus Bio in collaboration with the Children's Hospital of Philadelphia, BLD055 was identified through a round-over-round screening process in non-human primates using unbiased peptide-modified AAV libraries. The capsid demonstrates significantly enhanced tropism for cerebellar tissues compared to conventional vectors like rAAV1, enabling therapeutic transduction at doses up to ten times lower. In preclinical studies for spinocerebellar ataxia type 1 (SCA1), BLD055 has been used to deliver an RNA interference (RNAi) payload that targets and silences the ATXN1 gene, effectively reducing the levels of toxic polyglutamine-expanded ATXN1 protein in both mouse and non-human primate models.