Leveraging Non-Dilutive Funding for Ultra-Rare Disease Development with Dr. George Magrath Opus Genetics

Dr. George Magrath, CEO of Opus Genetics, discusses the company's experience in obtaining non-dilutive funding for its ultra-rare inherited retinal disease program. He highlights the challenges and advantages of this funding source, as well as the importance of establishing relationships with patient advocacy groups and foundations to mitigate risk and increase their attractiveness to traditional investors. The drive for non-dilutive funding for ultra-rare diseases is expected to become increasingly important in helping to get drugs to clinical trials.

George explains, "Opus Genetics is a gene therapy company for eye disorders that occur in children. And these disorders are ultra-rare. It's 200 patients, a thousand patients per indication, and it's really good science. It's from Gene Bennett, who was the inventor of Luxturna, which was the first approved genetic medicine. And it does require some non-conventional thought on the financing, though, because these are so rare. And what we've done at Opus Genetics and have been really fortunate to be a part of is non-diluted funding from external sources. And that comes in the form of partnerships with patient families and organizations, like we just announced last week with RDH 12 Alliance."

" It also comes with the Foundation Fighting Blindness, which has given us a number of grants and non-dilutive funding deals. And so those have been really important to Opus and have really helped us progress the programs. The way we think about that is the first principle is the clinical data we typically generate using our equity dollars. The preclinical work we try to do with non-dilutive funding, and that way we're able to focus on spending in rare scenarios."

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