Gene therapy company needs to raise additional €10 million to sustain activities to next milestone.

GenSight Biologics, a biopharma specializing in gene therapies for retinal neurodegenerative diseases, has announced the successful confirmation of the second Good Manufacturing Practice (GMP) batch of its lead product candidate LUMEVOQ. This event means the French biotech is now eligible to draw down the €4 million second tranche of a bridge financing agreed with Sofinnova Partners, Invus and UPMC Enterprises.

GenSight is developing a pipeline of therapies using mitochondrial targeting sequence and optogenetics technologies to address retinal diseases and central nervous system disorders. The company is currently awaiting the review of its marketing authorization application by the European Medicines Agency for LUMEVOQ, a gene therapy targeting a rare mitochondrial disease leading to irreversible blindness.

Before a product like LUMEVOQ can be approved for human use, a validation campaign must be completed, consisting of at least three successful GMP batches manufactured sequentially to demonstrate the robustness, control, consistency and reproducibility of the commercial manufacturing process.

Earlier this year, following the successful completion of two batches, shares in GenSight fell dramatically after the third batch had to be terminated due to production issues at its contract development and manufacturing organization.

GenSight says the additional funding released by achieving the latest milestone will extend its cash runway until mid-December 2023. The company is seeking alternative financing sources to meet its working capital requirements and cover operating expenses beyond that date. GenSight estimates a total funding requirement of approximately €10 million to sustain its activities until the anticipated resumption of its early access program in France in the beginning of the second quarter of 2024.

“The two successful GMP batches confirm the robustness of our manufacturing process and exemplify the fantastic work achieved by our team with our manufacturing partner in the past 12 months,” said Bernard Gilly, CEO of GenSight. “These successes boost our confidence that we can proceed to a successful PPQ campaign in 2024.”