Eye On the Cure Research News

Aug 3, 2020

Foundation Invests $3 million in Atsena Therapeutics, New Company Developing GUCY2D-LCA1 and MYO7A-USH1B Gene Therapies

With a founding investment from Hatteras Venture Partners, Atsena has raised a total of $8.15 million for its launch.

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The Foundation Fighting Blindness is investing $3 million in Atsena Therapeutics, a newly formed company with three retinal disease gene therapies in development. The first is for Leber congenital amaurosis (LCA) caused by mutations in GUCY2D, which is in a Phase 1/2 clinical trial at the University of Pennsylvania. The second, in preclinical studies, is a dual-vector gene therapy for Usher syndrome type 1B (USH1B) which is caused by mutations in MYO7A. The third program, also in preclinical studies, will be disclosed in the coming months.

Shannon Boye has emerged as a leading innovator of gene therapies for inherited retinal diseases. Her advancements are having tremendous impact in the field.

Benjamin Yerxa, PhD, chief executive officer, Foundation Fighting Blindness

Atsena Founder and Chief Scientific Officer Shannon Boye, PhD, University of Florida, is the preclinical developer for the company’s emerging gene therapies. Founder Sanford Boye, MSc, University of Florida, serves as the company’s chief technology officer.

“Shannon Boye has emerged as a leading innovator of gene therapies for inherited retinal diseases. Her advancements are having tremendous impact in the field,” says Benjamin Yerxa, PhD, chief executive officer, Foundation Fighting Blindness. “We are delighted to see the Foundation’s investment in her research over the past 15 years lead to the formation of Atsena and its promising gene therapies for saving vision.”

“Sustained support from the Foundation Fighting Blindness throughout my career has been crucial for advancing multiple preclinical programs in my lab,” says Dr. Boye. “And now, the Foundation’s funding is actually helping us get treatments into clinical trials. I am extremely excited by our potential now to save and restore vision in patients affected by these challenging retinal diseases. I am also genuinely heartened by the team we’ve built at Atsena, their universal desire to put patients first, and my ability to advance these therapies along with my FFB ‘family’.”

The Foundation’s investment in Atsena is being made through its RD Fund, a venture philanthropy fund for emerging therapies that are approaching, or in, early-stage clinical trials. The RD Fund was launched in 2018 with an initial investment of $70 million. The fund currently has eight projects in its portfolio.

Atsena’s Phase 1/2 gene therapy clinical trial for GUCY2D-LCA1 is being led by Samuel Jacobson, MD, PhD. While the primary goal of the early stage trial is to evaluate safety, investigators will also be evaluating changes in retinal function and structure.

LCA is a severe retinal disease causing vision loss in young children. More than two dozen genes, when mutated, can each cause LCA. LCA1, caused by GUCY2D mutations, is a common form of LCA, accounting for about 20 percent of cases.

Atsena’s dual adeno-associated virus (AAV) delivery system for USH1B addresses the challenge of delivering large genes — those too large to fit within a single AAV capsid— into retinal cells.

Usher syndrome is a leading cause of combined vision and hearing loss. More than a dozen genes when mutated can each cause the condition. MYO7A-USH1B is a relatively common form, accounting for about 25 percent of cases.

With Atsena’s dual AAV vector system, the MYO7A gene is split in half. Each half is delivered by a separate AAV vector and once both halves are delivered to the retina, they re-assemble to form the full length gene. While Atsena is working on delivery of MYO7A, the dual AAV vector system may ultimately be used to deliver other large retinal genes including, but not limited to, ABCA4 (Stargardt disease) and CEP290 (LCA).

Dr. Boye’s opening address for the Foundation’s 2020 VISIONS Conference, held virtually, reviewed both the LCA-GUCY2D and USH1B projects.