AAVB-081

AAVB-081 is an investigational gene therapy designed to treat Usher syndrome type 1B (USH1B), a rare genetic disorder that causes progressive vision loss due to retinitis pigmentosa and hearing impairment. The therapy uses a dual adeno-associated virus serotype 8 (AAV8) vector system, where the large MYO7A gene is split into two parts, each delivered by a separate vector into retinal cells. Inside the cell nucleus, these two halves recombine to form a complete functional MYO7A gene transcript, enabling production of the Myosin 7A protein that is deficient in USH1B patients. This approach aims to address the underlying genetic cause of vision loss in USH1B by restoring functional protein expression in retinal cells[1][4][5][6][8]. The product is administered via subretinal injection and has received FDA Orphan Drug Designation for this indication[2][8]. It is currently being evaluated in phase 1/2 clinical trials.

Other names

Dual AAV8.hMYO7ADual AAV8.5′MYO7A/AAV8.3′MYO7A