GenSight transfers to Catalent upstream phase of manufacturing for gene therapy candidate

Biopharma company GenSight Biologics announced the transfer to Catalent of the upstream phase of the manufacturing process for LUMEVOQ, GenSight’s gene therapy candidate for the rare mitochondrial disease Leber Hereditary Optic Neuropathy.

GenSight said Catalent is a new partner and the only contract development and manufacturing organization (CDMO) with a successfully commercialized gene therapy produced in their facility.

“Catalent offers gene therapy companies both production and in-house testing capabilities, and the GMP capacity at the facility used for LUMEVOQ will provide GenSight Biologics greater flexibility in the manufacture of the gene therapy,” according to the announcement.

The CDMO successfully manufactured the drug product batch that was released as safe for human use in November 2024, which will be the source of product supply for the named patient early access program (AAC) and dose-ranging study in France. Additionally, Catalent will manufacture the drug for a global Phase III trial and the regulatory submissions.

“This outstanding result is a significant milestone in GenSight’s strategy for securing the supply of LUMEVOQ for clinical use and for supporting the planned regulatory submissions,” Scott Jeffers, GenSight chief technical officer, said in a statement. “Our new partnership is proving to be highly effective, not just in completing the tech transfer process successfully, but also in improving the yield and upgrading the analytical methods used to reinforce control over the safety and quality of each batch.”

Leber hereditary optic neuropathy (LHON) is a genetically inherited disease that causes vision loss, with most people who inherit the condition becoming legally blind. Currently, there’s no cure for LHON. However, GenSight is aiming to provide patients with a safe and effective treatment for their urgent unmet needs.