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On November 17, 2021, GenSight Biologics announced that a second patient with advanced retinitis pigmentosa (RP) partially restored her visual function after receiving GS030 optogenetic therapy
Retinitis pigmentosa is a neurodegenerative eye disease in which vision deteriorates, usually in childhood
GS030 combines a single intravitreal injection of gene therapy (GS030-DP) with a wearable optoelectronic visual stimulation device (GS030-MD) using an optimized adeno-associated virus (AAV) vector that encodes the genetic instructions for the light-sensitive opsin ChrimsonR Ganglion cells fed into the fovea of the patient's retina
▲The mechanism of action of GS030 (Image source: GenSight official website)
A few months ago, Nature Medicine published the first case report of vision recovery in a patient receiving low-dose (5^10 vg) gene therapy: a blind patient who was diagnosed with the disease 40 years ago, after treatment, has been able to identify, Count, locate, touch different objects
The second patient was diagnosed with retinitis pigmentosa 20 years before enrollment and was treated with little light perception
In terms of safety, optogenetic therapy was well tolerated within 3 years of a single intravitreal injection in 9 subjects treated to date
References:
[1] GenSight Biologics Reports Second Patient Case Showing Significant Visual Recovery after GS030 Optogenetic Treatment.