GenSight Receives $41.3 Million for Ocular Gene Therapy Development

April 08, 2013

GenSight Biologics, a Paris-based developer of gene therapies for ocular diseases, is receiving $41.3 million in Series A venture capital funding for the development of two ocular gene therapies. One is an optogenetic treatment for retinitis pigmentosa (RP), a disease that causes blindness from retinal degeneration. The other is a gene-correction therapy for Leber hereditary optic neuropathy (LHON), a condition that causes central vision loss from degeneration of the optic nerve.

Both treatments are slated to move into clinical trials with the LHON therapy study expected to start in 2013. GenSight has not yet announced a start date for the optogenetic RP therapy trial. The venture-capital funding is being provided by The Novartis Venture Fund, Abingworth LLP, Versant Ventures and Index Ventures.

GenSight scientific co-founders Drs. Jose Sahel, of the Institut de la Vision, and Botond Roska, of the Friedrich Miescher Institute, are currently receiving funding from the Foundation Fighting Blindness for the development of the optogenetic therapy. GenSight’s offices are housed within the Institut de la Vision.

“We are very pleased that our investment in this highly promising approach to vision restoration is attracting substantial venture-capital funding,” says Dr. Stephen Rose, chief research officer of the Foundation. “Our partnership with GenSight and the strong financial commitment to this project will help ensure that it moves into human studies as quickly as possible.”

The optogenetic treatment is designed to restore visual function to dormant cone cells, the retinal cells that provide central vision and the ability to perceive colors and details. The treatment consists of a human-engineered virus that carries copies of a gene called halorhodopsin to the cone cells, making them light-sensitive. The treatment is expected to work for most forms of RP, and perhaps other retinal diseases, which have caused significant cone loss. The therapy is delivered by an ocular injection. A single treatment is expected to be effective for several years.

GenSight scientific co-founders also include Drs. Jean Bennett, of the University of Pennsylvania and the Children’s Hospital of Philadelphia (CHOP); Luk Vandenberghe, of Massachusetts Eye and Ear Infirmary; Connie Cepko, of Harvard University; and Serge Picaud, of the Institut de la Vision.

Dr. Bennett’s Foundation-funded clinical trial of gene therapy at CHOP has led to vision restoration for children and young adults who were virtually blind from Leber congenital amaurosis (LCA), a severe form of RP. The Foundation is also funding Dr. Bennett to develop a gene therapy for choroideremia. Dr. Vandenberghe receives funds from the Foundation for the development of gene therapy vectors that can accept large genes.

“The knowledge and experience of the GenSight scientific team is extraordinary,” says Dr. Rose. “These scientists are truly the world’s top gene therapy developers. Their expertise, coupled with this boost in funding, gives us great momentum for moving forward.”


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