GenSight Biologics Reports Second Patient Case Showing Significant Visual Recovery after GS030 Optogenetic Treatment
GenSight Biologics reported a second case of a patient with late-stage retinitis pigmentosa (RP) who partially recovered her visual function after treatment with GS030 optogenetic therapy.
“We are delighted by the highly encouraging signs emerging from PIONEER, which are demonstrating the promise of optogenetics to treat one of the most common blinding genetic disorders,” Bernard Gilly, Co-Founder and Chief Executive Officer of GenSight, said in a company news release. “We will eagerly push forward towards the realization of a treatment for retinitis pigmentosa patients.”
The GS030 optogenetic treatment, which combines gene therapy with the use of light-stimulating goggles, led to the patient being able to perceive and count objects 1 year after injection with GS030’s gene therapy component. The patient is a participant in the ongoing PIONEER phase 1/2 clinical trial of GS030, like another patient whose partial recovery was published as a case report in Nature Medicine in May 2021.
The patient, whose improvement is documented in a video, had been diagnosed with retinitis pigmentosa 20 years prior to enrollment and, at the time of injection, was barely able to perceive light. She received a single intravitreal (IVT) injection with the medium dose (1.5E11 vector genomes) of GS030 gene therapy in her worse-seeing eye and, after 4 months, started training on the use of the device.
Twelve months after injection, the patient could detect and correctly locate objects of different sizes and contrasts placed on a white table in front of her, with a 57% success rate when wearing the GenSight light-stimulating goggles, compared to only 24% without the device.
A video of the patient performing the tests can be viewed at www.gensight-biologics.com.
The patient was featured in an update on PIONEER that was presented by Dr. José-Alain Sahel, MD, Co-founder of GenSight Biologics and of the Institut de la Vision (Sorbonne-Université/Inserm/CNRS), Paris, France, and Distinguished Professor and Chairman of the Department of Ophthalmology at University of Pittsburgh School of Medicine, Pittsburgh, PA, USA, at the Retina Sub-Specialty Day of the American Academy of Ophthalmology (AAO) annual meeting (November 12-15, 2021).
Dr. Sahel also provided an update on the safety of GS030. The optogenetic therapy has been well-tolerated up to 3 years after the single intravitreal injection administered to the 9 subjects treated to date. There have been no systemic issues related to gene therapy, no adverse events leading to study discontinuation, and no withdrawal of participants.
In September 2021, after reviewing the safety data for the first three cohorts of the PIONEER trial, the independent Data Safety Monitoring Board recommended the use of the highest dose of the gene therapy (5E11 vector genomes) in the extension cohort. The trial’s topline results are expected to be available in H2 2022.