Amgen Ventures-backed AAVantgarde Bio has raised $141 million in series B funding that the Italian biotech will use to push its two eye disease gene therapies through the clinic.
One of these assets, dubbed AAVB-039, is currently undergoing a phase 1/2 trial in patients with Stargardt disease, a leading cause of vision loss in children and young adults. By delivering the full-length ABCA4 protein, AAVantgarde hopes that AAVB-039 can address the root cause of the inherited eye condition.
There’s also AAVB-081, which is being tested in a phase 1/2 study in patients with retinitis pigmentosa associated with Usher syndrome Type 1B. Usher syndrome is a rare genetic disease that affects both hearing and vision, with the 1B variety caused by mutations in the MYO7A gene.
AAVB-081 is designed to deliver the MYO7A protein by gene augmentation. The biotech brought early data from a handful of patients in the AAVB-081 trial to a retinal specialists conference in France last month, where AAVantgarde pointed to early “signals of clinical benefit.”
The company will use the proceeds from the $141 million series B to fund both trials, as well as an additional observation study of patients with Stargardt disease. The raise was led by Atlas Venture, Forbion and a new backer in the form of Schroders. Other new investors who participated in the round include Amgen Ventures, Athos Capital, CDP Venture Capital, Columbia IMC, Neva SGR, Sixty Degree Capital, XGen Venture and Willett Advisors.
Meanwhile, previous backers Longwood Fund and Sofinnova Telethon Fund returned for the latest round after participating in a 61 million euros ($71 million) series A in 2023.
The investment "is a strong endorsement of the biotech's team, science and two clinical inherited retinal disease programs, AAVantgarde CEO Natalia Misciattelli, Ph.D., said in the release.
“Both programs address the root genetic causes of devastating conditions and offer hope of improvement to patients and families living with progressive vision loss," the CEO added.
AAVantgarde spun out of the Naples, Italy-based Telethon Institute for Genetics and Medicine (TIGEM). The company was founded by Alberto Auricchio, M.D., Ph.D., TIGEM’s scientific director and professor of medical genetics at Federico II University in Naples, who currently serves as the biotech’s chief scientific officer.
It’s been a busy year for eye-related gene therapies. While Johnson & Johnson’s $130 million bet on an X-linked retinitis pigmentosa candidate suffered a phase 3 setback in May, the likes of Opus Genetics, MeiraGTx, Beacon Therapeutics and 4D Molecular Therapeutics have seen better news in the clinic.
Meanwhile, the $135 million series B and Big Pharma backing secured by Stargardt disease-focused SpliceBio in June suggests investors can still be enthused by the potential for gene therapies to treat rare ocular diseases, while Eli Lilly bought Adverum Therapeutics last month.