bridge the early stage funding gap for these gene therapies, according to Dorfman, the company’s CEO. Odylia negotiates discounts with industry participants, among them contract research organizations, to cut down the costs of manufacturing the experimental gene therapies and running the preclinical studies needed to convince the FDA they are safe enough to be tested in humans. With an in-house team, Odylia will locate gene therapy experts to run clinical trials and patients to enroll, and help craft licensing agreements when the time comes.
Odylia is starting with a focus on gene therapies for inherited retinal dystrophies, but intends to expand beyond that, to diseases that don’t involve the eye, if it shows its model can work. “We need to walk before we run,” Dorfman says.
The group plans to offer a carrot to for-profit developers as well: donate money into the Odylia system, and get the right of first refusal to license a gene therapy that they like. Proceeds from any licensing deal will be reinvested into the next programs Odylia moves forward.
“The goal is just to have [these therapies] picked up and moved,” Pierce says.
The trio concedes there are major questions about whether they can raise the type of cash it would take to fund this work. While venture dollars are flowing into biotech startups at a record pace, raising even a small amount of cash is a much harder task for a non-profit. Odylia started up with the help of $1.3 million in seed money from Mass Eye and Ear and the non-profit Usher 2020 Foundation, a patient group Dorfman runs for children with ultra-rare inherited retinal diseases. Those donations are enough to keep Odylia going for “a long time,” Dorfman says. But to really “move the needle,” Odylia needs the benevolence and cash of companies and philanthropists, Dorman says. “Those are the two wild cards that we have not yet proven,” he says.
“While you can certainly accuse us of some level of naiveté in setting this up,” Vandenberghe says, “we’re not naïve in the sense that we all acknowledge this is an highly challenging effort and space to operate in.”
Since launching last year, the group has gained some traction. Dorfman says three companies within the biopharma industry have begun working with Odylia, though he can’t disclose their names. Roche head of rare disease drug discovery Mathew Pletcher has joined Odylia’s board, as have two other well-known gene therapy experts: University of Pennsylvania gene therapy pioneer Jean Bennett, who, along with Katherine High, co-led the early research that created Luxturna; and Telethon Institute for Gene Therapy principal investigator Albert Auricchio, another Luxturna contributor and the inventor of a variety of gene therapy-delivering viral “vectors.” The two may funnel programs into Odylia, though Dorfman says hasn’t happened as of yet.
The company is also amassing a database of academic research and stalled gene therapies for inherited retinal dystrophies within companies and expanding its network of collaborators. Being a non-profit has helped open these doors; people have been receptive to Odylia’s mission, Dorfman says.
“If we were a for-profit, everyone would be looking for their own advantage and for ways to personally gain,” he says.
But the real proof Odylia can succeed will be with the progress of the Mass Eye and Ear program, which helps treat retinal degeneration triggered by a mutation to the gene RPGRIP1. The mutation affects around 2,000 people in the U.S., says Pierce–similar to the number of people who have the RPE65 mutation treated by Spark’s Luxturna. Yet while Luxturna is now a product, the Mass Eye and Ear program and others like it are sitting, waiting to be put through further testing.
“Luxturna had the benefit of being first,” Vandenberghe says. “It’s much more challenging to be a Luxturna-like second. It is Odylia’s mission to get more programs to overcome that challenge.”
