In another sign that gene therapy has come of age, Paris-based GenSight Biologics is lifting off with a $41 million shot in the arm from a lineup of blue chip VCs, led by a Big Pharma venture arm--Novartis Venture Fund--along with Abingworth, Versant Ventures and Index Ventures.
The biotech upstart will announce later on Monday morning that it has the backing of an international group of scientists and is aiming to develop gene replacement therapies for a pair of orphan targets: Leber's hereditary optic neuropathy (LHON) and retinitis pigmentosa. The lead product is expected to enter the clinic later this year in LHON patients.
Former Fovea and Transgene CEO Bernard Gilly is helming this new biotech, which counts among its co-founders professor José-Alain Sahel, chairman of the Vision Institute in Paris; Botond Roska, a specialist in the structure and function of neural circuits at the FMI in Basel; and Jean Bennett, professor of ophthalmology at the University of Pennsylvania.
Bennett was the co-author of a recent micro study that used gene therapy to treat a handful of patients with Leber congenital amaurosis, which leads to blindness. Bennett used an adeno-associated virus to transport a corrective gene into the eyes of three adult patients. Bennett said the patients responded well, demonstrating improved vision. And significantly, the treatment was completed without any harmful immune response, which has raised questions in the past about the safety of gene therapy.
Now GenSight Biologics will take that same approach for Leber's hereditary optic neuropathy, along with its optogenetic therapy for retinitis pigmentosa. The new Leber's project will use the same vector Bennett used to transfer a corrective mitochondrial gene into the cells.
"It's interesting and amazing to see this comeback for gene therapies," Gilly tells FierceBiotech. Over the past four or five years, clinical trials have underscored the safety of the approach as well as the sustained efficacy patients can look forward to. A group of new gene therapy biotechs like bluebird bio have jumped into the clinic recently. Add the recent European approval for uniQure's pioneering gene therapy program, and the whole field has been transformed, making it easier to attract a significant round like this.
GenSight also has an added advantage in working on orphan eye diseases. "The eye is a small, closed system," Gilly explains, where small doses can have a big impact. The company also hopes to benefit from regulators' willingness on both sides of the Atlantic to consider a fast march through the clinic.
With a Phase I/II study prepped to launch in the fall, the biotech should now have enough money to make its way through 2016, says the CEO, enough time to complete a follow-up pivotal study with 100 to 120 patients and on to a conditional approval. And in the course of events, GenSight hopes to be in a position to choose between a partnership or going it alone, with only a small sales force needed to market the lead product.
For now, GenSight is operating with a virtual staff of 7. But Gilly expects that figure will more than double in the not-too-distant future.
- here's the press release