AAV-FAM161A splice-competent vector

AAV-FAM161A splice-competent vector is an adeno-associated virus (AAV) gene therapy candidate designed for the treatment of Retinitis Pigmentosa 28 (RP28), a progressive retinal dystrophy caused by mutations in the FAM161A gene. The vector utilizes a specialized 'splice-competent' architecture that incorporates minimal intronic elements flanking exon 4, enabling the regulated alternative splicing of the FAM161A transcript into its long (L) and short (S) isoforms from a single expression cassette. This approach addresses a critical limitation in retinal gene therapy by reconstituting the physiologic isoform balance necessary for photoreceptor maintenance and function. Preclinical studies presented at ASGCT 2026 demonstrated that this single-vector strategy provides superior molecular rescue and functional improvement in Fam161a knockout mice compared to the delivery of individual isoforms alone.

Other names

AAV-FAM161AAAV-FAM-161AAAV-FAM 161Asplice-competent single-vector AAV platform