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GenSight trials in development

31 January 2017

The US Food & Drug Administration has granted GenSight’s developing drug, GS030, Orphan Drug Disease Designation for the treatment of retinitis pigmentosa.

The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases / disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

GenSight is a clinical-stage biotechnology company which develops novel therapies for mitochondrial and neurodegenerative diseases of the eye and central nervous system.

The GS030 drug, currently undergoing a Good Laboratory Practices (GLP) regulatory toxicity study, is expected to be tested in a Phase I / II clinical trial in retinitis pigmentosa patients in 2017.

GS030 is designed to restore vision to patients; it is a gene therapy product comprising a gene encoding a photoactivatable channel rhodopsin protein, delivered via a modified AAV2 known as AAV2 7m8.

GenSight is also developing biomimetic goggles that stimulate the engineered retinal cells. The images are projected by a light source that uses a specific wavelength onto the retina. The goggles mimic the normal retinal activity of capturing vision information will then amplify the light signal at the appropriate wavelength to enable vision restoration.

We will follow these developments and report when further information is announced.


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