Skip to main content
Premium Trial:

Request an Annual Quote

GenSight Biologics to File UK Application for LHON Gene Therapy, Mulls 'Strategic Opportunities'

NEW YORK – GenSight Biologics on Wednesday said it plans to file a marketing authorization application for its lead candidate Lumevoq (lenadogene nolparvovec) for the treatment of Leber hereditary optic neuropathy (LHON) in the UK next year. The firm still plans a new Phase III trial of the gene therapy to assuage regulatory concerns in the EU.

Paris-based GenSight also said it is in discussions with potential partners on "strategic opportunities" such as a merger, acquisition, or licensing deal. 

The company is developing Lumevoq as an adeno-associated virus vector-based gene therapy treatment for LHON, a rare inherited mitochondrial disorder associated with vision loss. The therapy delivers a functional copy of the ND4 gene, mutations in which are known to cause LHON.

The firm said it received a letter from experts with the UK Medicines and Healthcare Products Regulatory Agency (MHRA) stating that based on clinical data presented at a scientific advice meeting in November, the application package proposed by GenSight "appears comprehensive" and could support a marketing authorization application.

According to the MHRA experts, the data package could potentially support an application without including data from the upcoming RECOVER trial, a new Phase III trial the firm is launching after it received feedback from the European Medicines Agency's Committee for Advanced Therapies indicating that the data provided weren't sufficient for approval. GenSight, which had submitted an application to the EMA for Lumevoq, withdrew its application in April before the EMA's Committee for Medicinal Products for Human Use issued its final opinion.

GenSight said the initial application it plans to submit to MHRA in the second half of 2024 would not include data from the RECOVER trial, but that the data would later be added to complete the dossier.

The company said it is aiming to receive a decision from MHRA on Lumevoq in the second half of 2025, in the hopes of commercializing the product in the UK that same year.

GenSight on Wednesday also said it has extended its cash runway to mid-February 2024 but will require roughly €14 million ($15.5 million) to continue to fund its activities through July 2024. After that, the firm expects revenue from an early-access program in France to provide it with enough cash through the end of Q1 2025. The company said it is in ongoing discussions with several business partners and creditors.