French gene therapy company GenSight Biologics SA, based in Paris, France, has announced the withdrawal of a planned IPO (initial public offering) on the US NASDAQ, citing market conditions. The company had filed in July 2015 to raise up to $100 million, following a private investment round of €36 million. The company currently has two Phase III trials underway with a gene therapy containing the human ND4 gene (NADH dehydrogenase subunit 4) for the treatment of Leber’s hereditary optic neuropathy (LHON). The experimental therapy has received orphan drug designation in both the US anf the EU.
Gensight was co-founded by Bernard Gilly, former CEO of both Fovea and Transgene, Jose-Alain Sahel and Serge Picaud, both from the Institut de la Vision at INSERM, Jean Bennett from the University of Pennsylvania, Luk Vandenberghe and Connie Cepko from Harvard Medical School and Botond Roska of the Freidrich Miescher Institute of Biomedical Research. The company was originally launched in the first half of 2013, with an investment of €32M in a Series A funding round, representing the second largest European Series A funding of that year. While gene therapy has traditionally been a difficult arena in which to raise funding, recent developments in the field, including a number of successful ophthalmic clinical studies and the approval of Europe’s first gene therapy product (Glybera alipogene tiparvovec gene therapy for lipoprotein lipase [LPL]), the industry’s outlook has altered significantly. As gene therapy’s image changes and novel gene editing tools are developed, new start-ups and initiatives have benefitted from an in-flow of funds seeking to access potentially attractive returns-on-investment. GenSight has focused its efforts on LHON with an estimated prevalence of 1 in 40,000 in Europe, and RP with a prevalence of 1 in 4,000. The company expects that 1,100 to 1,200 LHON patients will be seeking therapies for this disorder each year, a demand that GenSight hopes to address.
Commenting on the recent initiation of enrollment of patients for the company’s Phase III LHON study, Dr. Alfredo A. Sadun, MD, PhD, from the Doheny Eye Institute, UCLA, stated: “After three decades of study, I’m thrilled that we have reached this point where we may be able to offer effective treatment in LHON. We now have good reason to hope for a solution to this devastating bilateral cause of congenital blindness in young adults.”