AAVantgarde Bio Secures $141M Series B to Advance Eye Disease Gene Therapies

ROME, ITALY — Italian biotech AAVantgarde Bio has successfully raised $141 million in a Series B funding round, with the substantial capital injection set to propel its two lead gene therapies for inherited eye diseases through clinical development. The round was spearheaded by Atlas Venture, Forbion, and new investor Schroders, and notably included participation from Amgen Ventures.

Focus on Inherited Retinal Diseases

AAVantgarde Bio is currently running two separate Phase 1/2 trials targeting conditions that cause progressive vision loss:

• Stargardt Disease: The company’s first asset, AAVB-039, is in a Phase 1/2 trial for Stargardt disease, a major cause of vision loss in children and young adults. The therapy is designed to deliver the full-length $ABCA4 protein, aiming to tackle the root genetic cause of this inherited condition. The company will also use the new funds to support an additional observation study for Stargardt patients.
• Usher Syndrome Type 1B: The second therapy, AAVB-081, is being tested in a Phase 1/2 study for patients with retinitis pigmentosa linked to Usher syndrome Type 1B, a rare genetic disease caused by mutations in the MYO7A gene that affects both hearing and vision. AAVB-081 uses gene augmentation to deliver the MYO7A protein. The biotech recently presented early data at a conference in France, citing initial “signals of clinical benefit” from a handful of patients.

Investor Confidence and Strategy

The $141 million Series B raise reflects strong investor confidence in the biotech’s approach and clinical pipeline. In addition to the lead investors, new backers included Amgen Ventures, Athos Capital, CDP Venture Capital, Columbia IMC, Neva SGR, Sixty Degree Capital, XGen Venture, and Willett Advisors. Previous investors Longwood Fund and Sofinnova Telethon Fund also participated, having supported the company’s €61 million ($71 million) Series A in 2023.

AAVantgarde Bio was spun out of the Naples, Italy-based Telethon Institute for Genetics and Medicine (TIGEM) and co-founded by Alberto Auricchio, M.D., Ph.D., TIGEM’s scientific director, who now serves as the company’s Chief Scientific Officer.

The funding success comes amid a year of significant activity in the eye-related gene therapy space, including Sanofi-backed SpliceBio’s own $135 million Series B for Stargardt disease and Eli Lilly’s acquisition of Adverum Therapeutics. Despite recent clinical setbacks for some candidates, investors remain keen on the potential of gene therapies to treat rare ocular conditions.