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Gene therapy progresses through efforts of small companies, attracts big investments
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BARCELONA — Gene therapy has greatly progressed in rare disorders, such as Leber’s congenital amaurosis, but several applications are under evaluation and show promise, according to Jose-Alain Sahel, MD, who chaired a symposium on the topic at the Ophthalmology Futures European Forum.
“Gene therapy has also been tested as a novel form of drug delivery to the retina for AMD with anti-VEGFs. Results are so far not perfectly convincing, but there is a rationale to continue research in this direction,” he said.
Jose-Alain Sahel
Gene therapy can also be used to prevent retinal degeneration by neuroprotection strategies and to reactivate the retina “as a sort of biological prosthesis, to deliver to the retina proteins that are sensitive to light,” Sahel explained. Further potential lies in optogenetics, wherein retina cells can be used to create an artificial retina, he said. Gensight, a small company devoted to research and development of gene therapies, has already completed a phase 2a trial of optogenetics in treatment of Leber’s optic neuropathy and is moving this project into phase 3 by the end of 2015, according to Sahel. Furthermore, a trial of optogenetics for treatment of advanced retinitis pigmentosa will start in 2016, he said.
“This is high risk research and high risk development, which does not attract big companies. For optogenetics, for instance, you need a variety of skills and competencies in vectors, in clinical trials, and in prosthetics and devices. To gather such forces you need an integrated company that is fully dedicated to such a project. Startups play a key role in the development of this kind of innovation,” Sahel said.
Gensight, however, has attracted major investors, who have recently raised €32 million.
“There might be a need at some point for startups to partner with big pharma, for investments and to deliver their product to more patients. Development goes through these stages, from academia to startups to eventually big pharmas,” Sahel said. – by Michela Cimberle
Disclosure: Sahel reports he is founder of Gensight.