AAVantgarde secures $141m Series B funding

AAVantgarde is focused on translating science into transformative therapies for patients with inherited retinal diseases. The funds will support two clinical programmes. AAVB-039 is a gene augmentation therapy for Stargardt disease caused by ABCA4 mutations. It delivers the full-length ABCA4 protein, addressing the root genetic cause of the disease. The programme is in proof-of-concept stage and will complete the CELESTE clinical study and the >100-patient STELLA natural history study.

In Stargardt disease, competition includes Alkeus Pharmaceuticals with ALK‑001 in Phase II, Kubota Vision with a Phase III candidate, VeonGen Therapeutics’ VG801 gene therapy in Phase I/II, and Applied Genetic Technologies Corporation (AGTC) also developing gene therapy programmes. AAVB-039’s full-length ABCA4 approach aims to treat all patients regardless of mutation type, differentiating it from these competitors.

AAVB-081 targets retinitis pigmentosa secondary to Usher 1B, caused by MYO7A mutations. It delivers the full-length MYO7A protein and is in Phase 1/2 clinical development with the LUCE trial. In Usher 1B, Atsena Therapeutics’ ATSN‑301 programme is in preclinical development. AAVB-081’s full-length gene therapy approach may overcome limitations of truncated or early-stage competitors.

Stargardt disease is the most common macular dystrophy in young people. AAVB-039 treats patients regardless of the specific ABCA4 mutation. Usher 1B causes progressive vision loss and congenital deafness. AAVB-081 has the potential to improve outcomes for patients with this dual sensory impairment.

“This investment validates our team, our science, and our clinical programmes,” said Dr Natalia Misciattelli, CEO of AAVantgarde. “Our therapies target the root genetic causes of these devastating diseases.”