FDA Grants Fast Track Designation for GS030 in the Treatment of Retinitis Pigmentosa
Officials with the FDA have granted Fast Track designation to GS030 (GenSight Biologics) for the treatment of retinitis pigmentosa (RP). According to GenSight, GS030 combines AAV2-based gene therapy with optogenetics to treat RP.
“The Fast Track designation granted by the FDA to GS030 highlights the significant unmet need for a safe and effective treatment of all forms of retinitis pigmentosa,” said Bernard Gilly, co-founder and chief executive officer of GenSight, in a press release. “Following the publication of a promising first case report in Nature Medicine in June, and with more data from the PIONEER trial expected later this year, we are in a great position in our quest to provide a cutting-edge treatment for retinitis pigmentosa patients.”
Optogenetic therapies combine cellular expression of light-sensitive opsins with light stimulation provided by a medical device. GS030 uses an optimized viral vector to express the light-sensitive opsin ChrimsonR in retinal ganglion cells. The viral vector is administered via an intravitreal injection. The treatment further uses proprietary light-stimulating goggles to project the right wavelength and intensity of light onto the treated retina.
The treatment is currently being studied in the PIONEER trial, a phase 1/2 first-in-human, multi-center, open-label dose-escalation clinical trial designed to evaluate the safety and tolerability of GS030 in subjects with late-stage RP. The study is being conducted in 3 centers across the United Kingdom, France, and the United States.
Study participants in the first 3 cohorts include those affected by end-stage non-syndromic RP with no light perception or light perception levels of visual acuity, with an extension cohort planned to include patients with hand motion and counting fingers levels of visual acuity.
RP is a family of orphan genetic diseases, which are caused by a number of mutations in multiple genes involved in the visual cycle. Typically, patients with RP begin experiencing vision loss as young adults, with a progression to blindness by age 40. There is currently no curative treatment for the condition.
GenSight Biologics announces FDA grant of Fast Track Designation for optogenetic therapy GS030 as treatment for retinitis pigmentosa [news release]. BusinessWire; October 12, 2021. Accessed October 12, 2021. https://www.businesswire.com/news/home/20211011005660/en