GS030 for Retinitis Pigmentosa Earns Fast Track Designation

Fast Track designation is requested by the drug company; this process and designation are designed to facilitate the development and review of therapies which can treat serious or underserved conditions, as well as fill unmet medical needs. According to Pharmacy Times, GS030, a gene and optogenetics therapy designed to treat retinitis pigmentosa (RP), recently earned Fast Track designation.


According to GenSight Biologics (“GenSight”), GS030 is:

an innovative combination of…a gene therapy product encoding a photoactivatable channelrhodopsin protein, delivered via a modified AAV2 vector…and biomimetic goggles that stimulate the engineered retinal cells.

Basically, GS030, delivered via an adeno-associated viral vector, helps neuronal cells respond to light stimulation, preserves retinal ganglion cells, and works to prevent retinal degeneration. The biomimetic goggles help increase light stimulation so that patients can better see.

Fast Track designation hinges on:

whether the drug will have an impact on factors such as survival, day-to-day functioning, or the likelihood that the condition will progress from a less severe condition to a more serious one…[and] providing a therapy where none exists or which may be potentially better than available therapy.

Thus, both GenSight and the FDA believe that GS030 shows the potential to improve patient outcomes and fulfill an unmet need within the RP patient community. Currently, researchers are evaluating GS030 within the Phase 1/2 PIONEER clinical trial.

Retinitis Pigmentosa (RP)

Retinitis pigmentosa (RP) comprises a group of inherited disorders leading to retinal degeneration. Altogether, over 60 genetic mutations have been linked to RP. RP can be inherited in an autosomal dominant pattern (requiring only one defective gene), autosomal recessive pattern (requiring two defective genes), or an X-linked pattern (which usually affects males more significantly than females). Regardless, patients with RP experience photoreceptor cell degeneration. Ultimately, this causes progressive vision loss. Most patients are legally blind by 40 years old. Symptoms vary depending on whether rods or cones are affected first. However, symptoms may include:

  • Loss of peripheral vision
  • Decreased central vision
  • Night blindness
  • Inability to accurately perceive colors and details
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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