AAVantgarde Closes $70.7 Million Financing

Rare Daily Staff

Italy-based AAVantgarde Bio said it completed a $70.1 million (€61 million) series A financing to advance its two proprietary adeno-associated viral vector platforms for large gene delivery.

Atlas Venture and European life sciences venture capital firm Forbion co-led the round, with participation from Longwood Fund and AAVantgarde’s founding investor, Sofinnova Partners, through its Sofinnova Telethon Fund.

AAVantgarde has two proprietary AAV-based large gene delivery platforms: one leveraging DNA recombination, named Dual Hybrid, and one using protein trans-splicing, named AAV Intein. The company is validating the platforms in two lead programs: Usher syndrome type 1B–associated retinitis pigmentosa (Usher1B), using Dual Hybrid, and Stargardt disease, using AAV Intein.

The financing will fund completion of a first-in-human proof-of-concept study in subjects with Usher1B, as well as further development of the AAV Intein program and entry into the clinic for Stargardt disease. The company also plans to pursue programs beyond ophthalmology.

Usher syndrome type 1B is an inherited disease that affects the retina and the inner ear. It is caused by mutations in the MYO7A gene. The therapeutic gene to treat Usher1B is 6.7 kilobases long and therefore too large to fit inside a standard AAV vector. Children with the condition are born deaf, have vestibular dysfunction, and begin to progressively lose vision in their first decade of life. Although surgical treatments are available to address deafness in these patients, there are no treatments for the progressive vision loss and blindness associated with the condition.

Stargardt disease is the most common inherited form of macular degeneration. In most cases inherited as an autosomal recessive disorder, it is caused by mutations in the ABCA4 gene. The therapeutic ABCA4 gene is too large to fit inside a standard AAV vector. Currently, there are no treatments for the blindness caused by Stargardt disease.

In connection with the Series A financing, Jason Rhodes of Atlas Venture, Dmitrij Hristodorov of Forbion, and David Steinberg of Longwood Fund will join AAVantgarde’s board of directors.

“This financing represents a hallmark moment in our mission to help patients, and we are excited to work with both our new and founding investors to advance our platforms and pipeline,” said Natalia Misciattelli, CEO of AAVantgarde. “The completion of this round with such a high-caliber group of global life science investors is a recognition of the significance of AAVantgarde’s unique technology, which will initially be utilized to help patients suffering from the debilitating impact of blindness associated with Usher1B syndrome and Stargardt disease.”

Photo: Natalia Misciattelli, CEO of AAVantgarde