AAVantgarde Bio Raises $141M Series B to Advance Gene Therapies for Inherited Retinal Diseases

AAVantgarde Bio, the clinical-stage biotechnology company developing next-generation gene therapies for inherited retinal diseases (IRDs), has completed a $141 million Series B financing round to accelerate its most advanced clinical programs targeting Stargardt disease and Usher syndrome type 1B.

The Series B round was co-led by Schroders Capital, a global investment firm with expertise in life sciences, together with existing venture partners Atlas Venture and Forbion. New investors in the round included Amgen Ventures, Athos KG, CDP Venture Capital through its Large Ventures Fund, Columbia IMC, Neva SGR, Sixty Degree Capital, XGen Venture, and Willett Advisors. AAVantgarde also received continued backing from its previous supporters, Longwood Fund and Sofinnova Partners.

Founded in 2021 as a spin-out of the Telethon Institute for Genetics and Medicine (TIGEM) in Naples, Italy, AAVantgarde Bio is advancing a suite of proprietary adeno-associated virus (AAV) vector platforms designed to deliver large therapeutic genes — a technical challenge that has limited the development of gene therapies for many serious inherited conditions. The company’s technology aims to overcome these size limitations, unlocking treatments for disorders that were previously difficult to target with conventional AAV approaches.

AAVantgarde’s lead clinical programs, AAVB‑039 and AAVB‑081, target diseases that currently lack approved therapies. AAVB‑039 is engineered to address Stargardt disease, a hereditary retinal dystrophy caused by mutations in the ABCA4 gene, while AAVB‑081 targets retinitis pigmentosa (RP) secondary to Usher syndrome type 1B, which involves both progressive vision loss and congenital deafness. Clinical development of these programs has advanced into proof-of-concept stages, with the company aiming to generate robust data that could support regulatory filings and future approvals.

The Series B proceeds will be directed primarily toward completing these clinical proof-of-concept trials, including the CELESTE study for Stargardt disease and the LUCE phase 1/2 trial for Usher 1B. In addition, AAVantgarde plans to support an extensive natural history study involving more than 100 patients to better understand disease progression and optimize clinical development plans.

AAVantgarde’s mission is to offer transformative therapies for patients affected by debilitating genetic conditions that currently have few or no treatment options. Its scientific strategy leverages dual proprietary platforms — one based on DNA recombination and another using protein trans-splicing — to expand the range of genetic payloads that can be delivered effectively to target tissues, including the retina.

The company’s ability to secure a large Series B round reflects investor confidence in its scientific approach, clinical strategy, and leadership. AAVantgarde’s team includes experienced gene therapy researchers and executives who are steering the company through advanced clinical development and toward potential commercial milestones.

Investors in the round emphasized the urgent need for new therapeutic options for rare genetic diseases such as Stargardt and Usher 1B, noting that AAVantgarde’s platforms have the potential to deliver meaningful clinical benefits to patients who currently have limited choices. Strategic participation by life science-focused funds like Schroders Capital and Amgen Ventures is expected to bring not only capital but also industry expertise and networks that can support the company’s growth.

AAVantgarde previously raised a €61 million Series A round co-led by Atlas Venture and Forbion, with participation from Longwood Fund and Sofinnova Partners via its Sofinnova Telethon Fund, to support early clinical development and organizational expansion.

With the fresh capital secured, AAVantgarde is poised to accelerate its clinical programs, engage regulatory authorities, and expand its research portfolio with the long-term goal of bringing new gene-based treatments to patients worldwide.