Gene Therapy for Retinal and Neurological Diseases


Magali Taiel, MD, chief medical officer, GenSight Biologics, discussed future research the company plans to pursue.

"[We're considering] neurologic diseases or marginal diseases affecting mitochondria but we're not yet there. As soon as you have a technology that can overcome mitochondrial mutations, you can think about other diseases... mitochondrial mutations can lead to many diseases."

GenSight Biologics is targeting ophthalmic diseases with their gene therapy techniques. The company is investigating therapies in Leber hereditary optic neuropathy (LHON) and retinitis pigmentosa (RP) and plans to do future research in dry age-related macular degeneration (AMD).

Data from the phase 3 REFLECT trial (NCT03293524) showed that treatment with lenadogene nolparvovec (Lumevoq; GS010) yielded statistically significant improvements in visual acuity from baseline in the first affected eye.1 These improvements were seen with both unilateral (+0.15 LogMAR score [+8 ETDRS letters]; P <.05) and bilateral (+0.23 LogMAR score [+12 ETDRS letters]; P = .001) injections. 

Based on these promising preclinical data, the UK's Medicines and Healthcare products Regulatory Agency has granted the investigational therapy Promising Innovative Medicine designation as of September 6, 2021.2

GeneTherapyLive spoke with GenSight’s chief medical officer, Magali Taiel, MD, about further research the company plans to pursue if GS010 is approved. She also discussed how the company’s technology could be applied to neurological indications in the future.

1. GenSight Biologics reports topline results from REFLECT phase III clinical trial, confirming Lumevoq efficacy including better efficacy with bilateral treatment. News release. GenSight Biologics. June 30, 2021.
2. UK MHRA grants GenSight Biologics’ LUMEVOQ ophthalmic gene therapy promising innovative medicine designation. News release. GenSight Biologics.
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