Rare Disease Report

Retinitis Pigmentosa Drug and Goggles Treatment in Development

JANUARY 31, 2017
RDR Staff
The FDA has granted GenSight’s developing drug, GS030, Orphan Drug Disease Designation for the treatment of retinitis pigmentosa. 
GS030 currently undergoing a Good Laboratory Practices (GLP) regulatory toxicity study, and is expected to be tested in a Phase I/2 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 channelrhodopsin protein, delivered via a modified AAV2 known as AAV2 7m8. 
GeneSight 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.

About Retinitis Pigmentosa

Retinitis Pigmentosa is a group of inherited diseases causing retinal degeneration that often leads to blindness. In people with RP, cones and rods on photoreceptor cells die, leading to progressive loss of peripheral vision, night vision difficulties, degeneration in color perception, and loss of central vision. The condition can manifest at any age, but is typically diagnosed during adolescence and young adulthood. There is great variability in the rate of progression and degree of visual loss, but most patients with RP are legally blind by age 40 years.

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