• Confirms corrections implemented in remediation plan
  • All protocols successfully followed during manufacture of first GMP (Good Manufacturing Practice) batch
  • Batch may provide product supply in Q1 2024 for potential resumption of early access program and initiation of a possible new clinical trial, as well as additional data for planned regulatory submission
  • Manufacturing of a second GMP batch underway to provide additional product and data; results expected in October 2023

Paris, France, September 18, 2023, 7:30 am CEST – GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that the Company’s manufacturing partner in the US has successfully manufactured the drug substance (DS) for LUMEVOQ®, the Company’s gene therapy for Leber Hereditary Optic Neuropathy (LHON), compliant with Good Manufacturing Practice (GMP) standards.


Because the batch was manufactured according to GMP manufacturing protocols, which are the required standards for commercial batches, the gene therapy may be eligible for use with patients after passing all quality control tests and pending discussions with regulatory bodies. The manufacturing of a second GMP DS batch, which will increase the amount that can be made available to patients, is already underway, with vg titer results expected in October 2023.


“I would like to congratulate the entire GenSight team, as well as our manufacturing partner, for all the hard work that enabled us to cross this important milestone,” said Bernard Gilly, Chief Executive Officer and Co-Founder of GenSight Biologics. “With this achievement, we confirm that our robust manufacturing process can be executed at the best level of quality as required by authorities. LUMEVOQ should once again be available to patients and physicians in early 2024. The greater clarity in our timelines is great news for LHON patients struck blind by the disease.”


GenSight is planning to manufacture at least 3 GMP batches at commercial scale outside the context of a validation campaign[1], to generate more process data for a future Marketing Authorisation Application (MAA) submission, to provide more experience of the manufacturing process to the operating teams, and to fulfil the immediate requirement of supplying product for a possible new clinical trial and for the potential resumption of an early access program for patients in Q1 2024.


[1] A validation campaign, or Process Performance Qualification (PPQ) campaign, consists of at least 3 successful GMP (Good Manufacturing Practice, required standards for human use outside of a clinical trial) batches manufactured sequentially to demonstrate and document the robustness, control, consistency and reproducibility of the commercial manufacturing process at the designated commercial facility. This exercise is required only as part of a Marketing Authorisation Application with the EMA and a Biologics License Application (BLA) with the FDA.