Luk Vandenberghe calls it a “sad irony.”
After decades of ups and downs, gene therapy, which offers a long-lasting genetic fix for a disease, has arrived. A product from Spark Therapeutics called Luxturna, for a rare, inherited type of vision loss, recently became the first-ever approved gene therapy in the U.S., adding to two others in Europe. More products are likely on the way.
And yet Vandenberghe (pictured), the co-director of the Grousbeck Gene Therapy center at Massachusetts Eye and Ear, and his colleagues saw one investor or company after another pass on what they feel could very well be the next Luxturna. The work is too early, he and colleague Eric Pierce were told, and the targeted genetic defect—a mutation causing retinitis pigmentosa in young adults—is too rare.
Vandenberghe says this is part of a trend. There are “dozens, if not hundreds” of inherited retinal dystrophies, as they’re known, that could be amenable to a Luxturna-like treatment, he says, but gene therapies are only being pursued for a handful of them—the ones that affect the greatest number of patients and therefore present the biggest market opportunity.
Luxturna is an exception, in that targets a very rare mutation. But the treatment was bolstered by data from studies in large animals and a “clear indication” of safety and effectiveness in humans when Spark (NASDAQ: [[ticker:ONCE]]) formed in 2013 and plucked it from early work at the University of Pennsylvania and the Children’s Hospital of Philadelphia, Vandenberghe says. Similar gene therapies, like the Mass Eye and Ear program, usually can’t get the financial backing to progress to the same point.
As a result, says Vandenberghe, a co-founder of GenSight Biologics and an advisor to Nightstar Therapeutics (NASDAQ: [[ticker:NITE]]), treatments that could help people with other rare diseases are being “stranded in the lab never to reach the clinic.”
So rather than see their program stall, Vandenberghe, Pierce, and Scott Dorfman—the founder of the Usher 2020 Foundation, whose two children have the rare Usher Syndrome and are patients of Pierce—have tried something unusual. They’ve formed a non-profit company, Odylia Therapeutics, that aims to advance gene therapies that are getting left behind, among them Mass Eye and Ear’s retinitis pigmentosa program. Odylia’s focus: gene therapies with scientific promise yet limited commercial opportunity that may be gathering dust on the shelves of academic labs or even within companies.
Odylia is trying to set up a system that can move these gene therapies into human testing. The hope is as Odylia removes more of the risk and early expense involved in developing these treatments, they will become more attractive to companies to buy and turn into products—even with the small potential market. The nonprofit is testing the theory first with the Mass Eye and Ear retinitis pigmentosa therapy (which has gotten funding help from the Foundation Fighting Blindness), but Vandenberghe and Pierce are quick to say Odylia would fail if it were just an extension of their academic labs. It’s for others too. “We think the concept has legs,” Vandenberghe says.
Non-profit drug development work isn’t new. A number of initiatives and consortiums have been set up over the past few decades to try to address drug development bottlenecks and global health problems like malaria and AIDS.
And powerful non-profit organizations like the Bill & Melinda Gates Foundation help sponsor clinical development and even invest in companies with emerging technologies. Odylia’s is different because of its gene therapy-for-ultra-rare-disease focus. Another difference is its plan to stay involved as programs progress and sponsor clinical studies, Dorfman says.
Odylia plans to use a web of collaborations to
