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Apr 15, 2013
 |  BioCentury  |  Product Development

Gene therapy in sight

GenSight's big series A shows investor confidence in ophthalmic gene therapy

Gene therapy has been more or less off the radar for years, but last week's big series A round for GenSight Biologics Inc. reflects the investors' confidence that gene therapies for the eye can be brought to market quickly and at reasonable cost.

Abingworth, Index Ventures, Novartis Venture Funds and Versant Ventures put €32 million ($41.6 million) into GenSight. It is the second largest A round this year, behind cancer immunotherapy company Jounce Therapeutics Inc., which raised $47 million in February from Third Rock Ventures (see BioCentury, Feb. 18).

The money should allow GenSight to advance its preclinical Leber's hereditary optical neuropathy (LHON) product to registration in Europe as soon as late 2016 and its retinitis pigmentosa (RP) program to Phase IIa without additional financing. The company expects the funding to last through 2016.

GenSight was co-founded by veterans in gene therapy and ophthalmology: Executive Chairman Bernard Gilly, former CEO of Fovea Pharmaceuticals S.A. and Transgene S.A.; Jose-Alain Sahel, chairman of the Institut de la Vision of the Institut National de la Sante et de la Recherche Medicale (INSERM); Botond Roska of the Friedrich Miescher Institute for Biomedical Research; Jean Bennett, professor of ophthalmology at the University of Pennsylvania; Connie Cepko, professor of genetics at Harvard Medical School; Ernst Bamberg, professor at the Max Planck Institute for Neurological Research; Luk Vandenberghe of the Schepens Eye Research Institute and Harvard Medical School; and Serge Picaud of Institut de la Vision.

Gilly, Sahel and Picaud were also co-founders of Fovea, an ophthalmic play acquired by Sanofi in 2009 for €90 million ($131.5 million) up front and up to €280 million ($409 million) in milestones.

GenSight builds upon recent independent clinical successes of a team led by Bennett and two other academic groups showing that gene therapy can replace a defective protein and improve visual function in Leber's congenital amaurosis (LCA), a hereditary ocular disorder that leads to blindness by adulthood.

Gilly said Bennett's group has shown gene expression 11-12 years after a single retinal injection in dogs and after four to five years in man. The Children's Hospital of Philadelphia is sponsoring Phase III trials of AAV2-hRPE65v2 in individuals with LCA subtype 2.

While Gilly doesn't think that program is partnered, he said GenSight has chosen for commercial reasons not to...

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