Targeting LHON and Retinitis Pigmentosa With Gene Therapy


Magali Taiel, MD, chief medical officer, GenSight Biologics, discussed the company’s work in ophthalmic diseases.

“We have 2 technology platforms. The first 1 is a technology platform to overcome mitochondrial mutation and we are developing it in Leber hereditary optic neuropathy. And the second technology platform is also important, we are developing optogenetic therapy; right now we are in early phase in retinitis pigmentosa and the beauty of this program is that it's independent to causal mutation.”

GenSight Biologics is aiming to use gene therapy methods to combat ophthalmic diseases. The company currently has programs in Leber hereditary optic neuropathy (LHON) and retinitis pigmentosa (RP) and is also setting its sights on dry age-related macular degeneration (AMD).

Recent data from GenSight’s phase 3 REFLECT trial (NCT03293524) showed that patients treated with lenadogene nolparvovec (Lumevoq; GS010) had statistically significant improvements in visual acuity from baseline in the first affected eye with both unilateral (+0.15 LogMAR score [+8 ETDRS letters]; P <.05) and bilateral (+0.23 LogMAR score [+12 ETDRS letters]; P = .001) injections. 

GeneTherapyLive spoke with GenSight’s chief medical officer, Magali Taiel, MD, about the company’s background and technology platforms. She also discussed the need for gene therapies in ophthalmic diseases and partnerships the company has with academic institutions.

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.
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