AAVantgarde Bio, a clinical-stage biotech firm headquartered in Italy, revealed that the US Food and Drug Administration (FDA) has awarded Orphan Drug Status to its leading initiative, AAVB-081, aimed at treating Usher Syndrome Type 1B (USH1B) retinitis pigmentosa.
“Individuals with USH1B desperately need a treatment to halt their gradual vision decline, which eventually leads to blindness. The FDA’s Orphan Drug Status underscores this critical unmet requirement and marks a significant advancement for our project,” stated Dr. Natalia Misciattelli, the company’s CEO.
The FDA’s Orphan Drug Designation is granted to drugs and biologics targeting rare conditions meeting specific criteria. This designation offers benefits such as tax incentives for eligible clinical trials, fee exemptions, and the possibility of seven years of market exclusivity upon approval.
AAVB-081 is a retinal-administered, AAV8-based dual hybrid product specifically addressing MYO7A-related Usher syndrome (USH1B). AAVantgarde’s technology employs two separate AAV8 vectors, each holding a segment of an expression cassette required for the Myo7A gene. Within the cell nucleus, these segments recombine into a single entity, facilitating effective protein production seen in animal trials.
Usher syndrome type 1B (USH1B) is a genetic disorder influencing the retina and inner ear, resultant from MYO7A gene mutations. The gene therapy for USH1B measures 6.7 kb, which exceeds the capacity of traditional AAV vectors. Roughly 20,000 individuals across the US and EU are afflicted by Usher1B. Those affected are born with hearing loss, balance issues, and start experiencing progressive vision degradation in childhood. While surgical approaches exist to counteract deafness, treatments for ongoing vision deterioration are currently lacking.
AAVantgarde Bio operates on an international scale at the clinical development stage. The company has pioneered two unique Adeno-Associated Viral (AAV) vector platforms to overcome the gene therapy limitations posed by AAV vector capacity constraints.