Recently, we talked with Scott Dorfman, CEO of Odylia Therapeutics. In this clip, Dorfman explains the origins and philosophy of Odylia Therapeutics, a non-profit company designed to find treatments for rare disease likely to be overlooked by for-profit organizations.

The company began just two years ago based on funding from Massachusetts Eye & Ear and Usher 2020 Foundation. The latter group is a patient organization, also started by Dorfman, to fund research for Usher syndrome, a rare disease that afflicts two of Dorfman’s children.

Usher disease is an autosomal recessive disease that leads to mutations in the USH1C gene that causes profound deafness at birth, vestibular disfunction and degenerative sight loss. Some of the research that Usher 2020 Foundation funded led Dorfman to meet Luk Vandenberghe, PhD and Eric Pierce, MD, PhD of Harvard Medical School and Massachusetts Eye & Ear. Together, the 3 men planted the seeds to form Odylia Therapeutics with the goal of facilitating treatments for low prevalence retinal disease with proof-of-concept science to clinical trials.

“We were fortunate enough to be given from Mass Eye &Ear, the IP for a unique novel vector called Anc80, which was invented by Luk Vandenberg out of Harvard. it’s a novel ancestral AV therapy genetic therapy for that had not existed in the past and it has long patent protection. It shows great expression in the retina as well as other systemic areas.”

Dorfman added that Odylia owns the rights to use Anc80 in any disease with a prevalence under 3000 (in the United States) while Lonzo Houston owns the rights for all diseases about 3000.

Among the gene therapies in development by Odylia is RPGRIP1, which is using the ANc80 vector to deliver a gene therapy into the retina. “We’ve been doing pre-clinical work on it. We’re starting IND-enabling studies now and we have a sponsor that’s going to take it forward a clinical trial over the next several months,” noted Dorman.

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