The attempt to commercialize gene therapy has been a multi-decade roller coaster, but its current upswing continues. Today Allergan, a company best known for skin and eye care products, has acquired a startup developing gene therapies for a range of eye diseases.
The deal is pocket change for a company that brought in over $4 billion in revenue in 2015, but Allergan (NYSE: [[ticker:AGN]]) paying $60 million up front for privately held Ann Arbor, MI, startup RetroSense Therapeutics signals another large company’s comfort with gene therapy—a technique that could offer long-lasting treatments for genetic diseases, if not outright cures. The deal includes other potential payouts for RetroSense shareholders if the company’s lead product, a treatment for the eye disease retinitis pigmentosa, progresses successfully.
Two gene therapies are already approved in Europe, and Spark Therapeutics (NASDAQ: [[ticker:ONCE]]) could soon become the first company to have one approved in the U.S.—a gene therapy for a form of genetic blindness. In addition, gene therapies have begun producing encouraging results in diseases like hemophilia, sickle cell disease, and beta-thalassemia. Years of ups and downs have given way to technological advancements that have led to more promising treatments.
Though questions still remain about gene therapy’s durability, how safe it will be over time, and how broadly applicable it’ll be as a method of drugmaking, it’s regained momentum. There are now a slew of publicly traded gene therapy companies, and large pharmaceutical companies both investing in and buying smaller gene therapy firms. Earlier this year, for instance, Pfizer, which already has a partnership with Spark, bought a privately held gene therapy company called Bamboo Therapeutics that is working on a treatment for Duchenne muscular dystrophy. In addition, two new gene therapy startups—Avrobio and Orchard Therapeutics—were launched earlier this year with the support of venture firms.
Retrosense has become the latest gene therapy company to draw interest. It’s developing a treatment for retinitis pigmentosa, an inherited condition in which photoreceptor cells in the retina die, leading to gradual vision loss. RP affects about 100,000 people in the U.S. and has no effective treatments.
Retrosense’s treatment, RST-001, is meant to help restore vision in patients with the disease. The company began the first human clinical tests of RST-001 earlier this year. Allergan said in a statement the therapy could be useful not just for RP, but other retinal diseases as well. According to Retrosense’s website, the company planned to test RST-001 as a treatment for the “dry” form of age-related macular degeneration.
“The team at Allergan is excited by the prospect of advancing an entirely new approach in the treatment of retinal diseases, and this technology is highly complementary to our ongoing development programs in this critical treatment area,” said David Nicholson, Allergan chief research and development officer, in the statement.
Retrosense was formed in 2009 around scientific work at Wayne State University, and was funded by the Foundation for Fighting Blindness, the Michigan Economic Development Corp., the National Neurovision Research Institute, and unspecified private investors.
