Nonprofit Research on Gene Therapy for Rare Ocular Diseases

Gene therapy research and development has been progressing the field of genetic disease by striving to provide an effective therapeutic approach to correcting the genes involved with specific conditions.

The mission of biotech company Odylia Therapeutics is to bring therapeutics to patients with rare diseases, regardless of prevalence or commercial opportunity.

Odylia is a nonprofit organization, setting it apart from traditional biotech and pharmaceutical companies. President and CSO, Ashley Winslow, PhD, explained that while the science and research functions the same way, the fundraising efforts are different and the organiztion utilizes its profits as an investment for furthering its mission.

“We are very focused, as a nonprofit, on the treatments we're trying to develop, or how we're helping other groups, so everything comes back to the mission,” she said.

The organization's current gene therapy pipeline is focused on 2 rare ocular diseases, according to Winslow. In this interview with HCPLive, she spoke about the genes each program is working with to address Leber congenital amaurosis (LCA) and Usher syndrome.

The transparent practices of a nonprofit like Odylia has opportunities that industry companies may not.

“I really love that aspect of my job,” Winslow said, “because having worked in industry before, you don't get the opportunity to talk every day about what you do. As a nonprofit, we have to talk about it. It's not only inspiring, it's easy to get up every day and do what we do.”

Education and awaress are escpecially critical when working with rare diseases.

“We have to speak to it in order to really attract people to our mission,” she said. “And that's what we're trying to do through fundraising efforts is speak to what we've accomplished, what we're seeking to accomplish, where we are pushing boundaries.”