Five-year data show gene therapy safe for ND4-LHON

Woman having an eye test

GenSight Biologics has revealed pooled safety data from five clinical studies of lenadogene nolparvovec (Lumevoq), the largest cohort of ND4-LHON (Leber hereditary optic neuropathy) patients studied after gene therapy treatment.

The data confirms the good overall safety profile of the product in terms of systemic and ocular tolerability, humoral and cellular immune response. The results also highlight a comparable safety profile for unilaterally and bilaterally treated patients.

The data was published in a peer-reviewed article in the American Journal of Ophthalmology.

“The publication of this safety analysis is a key milestone, combining data from all five clinical studies of Lumevoq and representing the largest cohort of ND4-LHON patients ever treated with gene therapy,” said Dr Catherine Vignal-Clermont, Department of Neuro Ophthalmology and Emergencies at the Rothschild Foundation Hospital in Paris and lead author of the article.

“We are very pleased to see our work published in such a prestigious journal, which highlights the significance of our findings. Given the good safety profile demonstrated, we believe lenadogene nolparvovec is proving itself as a potential new treatment solution for ND4-LHON patients as a clinically relevant patient benefit has been established with minimal safety risks.”

The article analyses safety data collected from ND4-LHON patients who received single unilateral or bilateral intravitreal injections of lenadogene nolparvovec across five clinical studies (REVEAL, RESCUE, REVERSE, RESTORE and REFLECT) who were followed over five years after treatment.

Almost all patients (95%) received lenadogene nolparvovec at a dose of 9 x 1010 viral genomes per eye and 88% had at least two years of follow-up.

Related Articles

Join FREE today and become a member
of Drug Discovery World

Membership includes:

  • Full access to the website including free and gated premium content in news, articles, business, regulatory, cancer research, intelligence and more.
  • Unlimited App access: current and archived digital issues of DDW magazine with search functionality, special in App only content and links to the latest industry news and information.
  • Weekly e-newsletter, a round-up of the most interesting and pertinent industry news and developments.
  • Whitepapers, eBooks and information from trusted third parties.
Join For Free