Diseases of the eye : a prosperous period for gene-based therapies

After a first clinical study initiated against Leber congenital amaurosis, Horama is preparing a new test to fight other disease causing blindness. The biotech nantes announced on Wednesday, October 11, having obtained the permission to conduct a clinical study of phase I/III to demonstrate the non-toxicity and initial evidence of effectiveness of the Hora Pde6b. It is a gene therapy against retinitis pigmentosa induced by the mutation in the gene PDE6B. The method of Horama is injected under the retina a vector derived from a virus non-pathogenic.

“The first patient will benefit from treatment before the end of the year”, explains to La Tribune Christine Placet, ceo and president of the company created by researchers from Inserm and CHU of Nantes in 2014. This clinical trial will be conducted within the CHU of Nantes, the place of establishment of the biotech, on 12 patients in total, with “12 months of follow-up per patient”. If the trial is conclusive, Horama plans to proceed directly to phase III, the last stage before a possible commercialization. The test could include a quarantine of the patients. In a year, Horama intends to initiate a phase I/II for another gene therapy against retinitis punctate albescente, another rare disease, often to the point of blindness.

The moment is particularly well chosen : an expert committee FDA (u.s. Agency of medicines) has given yesterday a positive opinion on a gene therapy-Spark Therapeutics against Leber congenital amaurosis, an eye disease due to mutations in the gene RPE65. This treatment has improved to 93% of patients, based on the clinical trials conducted by the biotech american. Except surprise, the institution will permit the marketing of the product in a few months. This would be the first gene therapy dedicated to a rare disease affecting the vision. Still remains the question posed by the committee of experts of the FDA on the duration of the effectiveness of the treatment after one year.

A decision encouraging strategies based on gene therapy

The favorable opinion of the FDA is a satisfaction for Christine Placet. A negative decision could have impacted the strategy of the clinical development. “This shows us part of the way forward. Even if their method is intended for a different indication, we have a comprehensive approach similar.” This decision of the american Agency would promote the ongoing process of fund-raising in the biotech nantes.

Other French biotech that could see their horizon clear : Gensight. It has raised € 22.5 million in its ipo in late June to launch the first gene therapy French. The society hopes to publish in 2018 the results of the phase III study of its therapeutic product flagship, GS010, against the Leber hereditary optic neuropathy, another rare eye disease. The company had shown a significant improvement in the vision of patients in clinical trials.

Gene therapy could well have reached a phase of maturity, as the progressed the scientific director of AFM-Telethon a few months ago…

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