AAV8-RK-BBS10

AAV8-RK-BBS10 is an investigational gene therapy developed by MeiraGTx for the treatment of Bardet-Biedl syndrome (BBS) resulting from mutations in the BBS10 gene. The therapy utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver a functional copy of the human BBS10 cDNA, driven by a rhodopsin kinase (RK) promoter to target photoreceptor cells. BBS10 is a member of the chaperonin-like family required for the assembly of the BBSome, a protein complex essential for primary cilia function and protein trafficking within the retina. By restoring BBS10 expression via subretinal injection, the therapy aims to preserve retinal structure and function in patients with BBS10-related retinal dystrophy. As of early 2025, the program is in preclinical development and has received FDA Rare Pediatric Disease Designation.

Other names

BBS10 gene therapyBBS-10 gene therapyBBS 10 gene therapyAAV8-BBS10AAV-8-BBS10AAV 8-BBS10