AAVantgarde Bio announced the closing of a $141 million (€122 million) Series B financing round. According to a company press release, the proceeds from the financing will support the completion of clinical PoC of AAVantgarde’s AAVB-039 CELESTE study for Stargardt disease caused by a mutation in the ABCA4 gene and the completion of the > 100 patient STELLA natural history study. The proceeds will also support the completion of clinical PoC of AAVantgarde’s AAVB-081 LUCE phase 1/2 clinical trial for retinitis pigmentosa (RP) secondary to Usher 1B due to a mutation in the MYO7A gene. 

Stargardt disease is the most prevalent macular dystrophy in young people. According to AAVantgarde,  the company's AAVB-039 program addresses the root genetic cause of this disease—mutations in the ABCA4 gene—through gene augmentation therapy that delivers the full-length ABCA4 protein, enabling treatment of any patient, regardless of the specific mutation.

Similarly, RP secondary to Usher syndrome 1B is a rare and devastating IRD caused by mutations in the MYO7A gene. Usher 1B causes progressive vision loss combined with congenital deafness, resulting in a double sensory disability. AAVantgarde said its AAVB-081 program targets the root genetic defect by delivering the full-length MYO7A protein by gene augmentation, offering a treatment potentially able to improve the lives of patients affected by this dual impairment.