GenSight announces good news on Lumevoq
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(News Bulletin 247) – The price of the French pioneer in cellular therapies targeting vision diseases rebounded strongly on Monday. GenSight announces the successful production of the first pilot batch incorporating improvements in the manufacturing process of Lumevoq, a cell therapy aimed at correcting a degenerative disease responsible for sudden blindness.

Good news on “lenadogene nolparvovec”, or Lumevoq, one of the first gene therapies – that is, a treatment aimed at resolving the genetic defect causing a given disease, in this case Leber’s hereditary optic neuropathy (LHON). ), a rare disease causing a sudden loss of vision generally in young subjects.

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GenSight Biologics, the originator of this innovative treatment, saw its share price rebound by nearly 20% to 3.886 euros, around 2:30 p.m. on Monday after the “success” of the production of the first pilot batch (“engineering run”) integrating improvements in the Lumevoq manufacturing process.

This batch succeeded in generating a product (“drug substance”) whose viral titer reached the acceptance threshold, thus confirming that the manufacturing process of Lumevoq is robust to the defined batch size. GenSight states that the yield is significantly improved to a level above all previous batches.

The company recalls that the pilot batch was the first to incorporate a series of targeted corrective measures identified by GenSight and its production partner in the United States in April of this year, and relating to the reinforcement of the control of the procedures as well as the implementation place of reinforced on-site supervision.

A quality problem to solve

In April 2022, GenSight announced a delay in the production of validation batches (PPQ) of its product in Leber hereditary optic neuropathy (LHON). The delivery of the last batches of the treatment was then not satisfactory, a quality problem had occurred at the last stage of production at the end of 2021.

The company had quickly identified and thought to solve the problem, due to a change of model of a TFF filter (tangential flow filtration, intended to harvest cells modified to correct a genetic defect). But the production campaign which had resumed in December 2021 once this equipment problem had been solved did not make it possible to generate a product in line with expectations, the viral title being below the acceptance threshold.

To put the odds on its side, the company had thus decided to produce several pilot batches of reduced size in order to confirm the robustness of the corrective measures. Today’s announcement confirms the success of the steps taken by GenSight to resolve the execution issues in close collaboration with its partner. The adjustments undertaken by both parties have even led to an improvement in the execution of the manufacturing process. GenSight’s patience has been rewarded.

“While this manufacturing setback has delayed the original schedule, we would like to reaffirm to LHON patients and their healthcare professionals that we continue to work tirelessly to get Lumevoq approved to provide them with a therapeutic solution. as quickly as possible and in the safest way”, commented Bernard Gilly, CEO and Co-Founder of GenSight Biologics.

The company is now confident in the resumption of its early access program as well as in the clinical development and preparation for the commercialization of the product. GenSight still expects to obtain an opinion from the European Committee for Medicinal Products for Human Use (CHMP) by the third quarter of 2023, followed by commercial launch by the end of 2023.