PPQ campaign restart targeted for Q4 2022
Scott Jeffers joins as new technical director overseeing manufacturing and supply chain operations
PARIS–(BUSINESS WIRE)–Regulatory news:
GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharmaceutical company specializing in the development and commercialization of innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, announces today a report in the finalization of the validation (PPQ) batches for LUMEVOQÂ®, the Society’s gene therapy for the treatment of Leber’s Hereditary Optic Neuropathy (LHON). The delay is necessary to implement operational adjustments that will prevent the recurrence of issues with the latest PPQ campaign. The Company aims to restart the campaign in the fourth quarter of 2022.
The latest campaign, which was launched after the Company resolved an equipment issue that caused the 2021 campaign to fail, generated a drug substance whose viral genome titer fell below the acceptance threshold . The resulting investigations, conducted by external experts, traced the result to operational difficulties at specific stages of the downstream process. To prevent the recurrence of these issues, the Company is working with its manufacturing partner to implement targeted fixes around improved process control and tighter oversight within manufacturing suites. Additionally, the Company decided to manufacture smaller engineering batches to confirm the robustness of the corrective actions.
“We understand the difficult position this can place LHON patients in, so we are moving quickly but thoughtfully to act on the clear set of targeted corrections identified by our experts,” has explained Bernard Gilly, CEO and co-founder of GenSight Biologics. “We are more determined than ever to get it right. By performing additional checks throughout the engineering cycles, we will restart the PPQ campaign with added confidence that the manufacturing process will be performed reliably and to the highest standards of execution.
âIn the meantime, our teams will work with KOLs, treatment centers and local authorities to ensure that patients identified during this adjustment phase receive timely treatment,â adds Bernard Gilly. The supply of LUMEVOQÂ® the vials available for compassionate use have been exhausted, so treatments can only resume in early 2023 when test results confirm the quality of the product from the new PPQ campaign. âWe are strongly committed to providing LHON patients with access to our innovative therapy as soon as possible.â
The new technical director of the company, Scott Jeffers, who will assume the responsibilities of the former vice president of pharmaceutical operations. Scott joins GenSight with extensive experience holding senior management responsibilities over CMC operations at various pharmaceutical companies as well as contract manufacturing companies.
“I am excited to lead GenSight’s efforts to improve the executability of its established manufacturing process.commented Scott Jeffers. “Making AAV vectors is, in general, a relatively well-known process, but there are specifics for LUMEVOQ that need to be considered. The extra time we spent on corrections will ensure the impact and success of our action plan.â
Scott will be supported by Jay Stout, a leading expert and consultant in the field of biologics, cell and gene therapy manufacturing and supply chain. Both based in the USA, they will be able to collaborate intensively with the Company’s manufacturing partner on the site where LUMEVOQÂ® manufacturing operations are carried out.
GenSight has initiated discussions on the new manufacturing schedule with the European Medicines Agency, while finalizing all day 120 responses unrelated to the manufacturing of PPQ batches. The Company will provide an update once the Agency has given its opinion.
The GenSight leadership team will be hosting a live webcast today, Thursday, April 7, 2022to discuss the manufacturing plan. The webcast will be in English and simultaneous translation into French will also be available.
Thursday, April 7, 2022
8:00 a.m. EDT / 2:00 p.m. CEST
The webcast will be available as recordings using the same links used to access the live webcast.
GenSight Biologics will release its 2021 financial results on April 8, 2022.
About GenSight Biologics
GenSight Biologics SA is a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics’ pipeline leverages two primary technology platforms, mitochondrial targeting sequence (MTS) and optogenetics, to help preserve or restore vision in patients with blinding retinal diseases. GenSight Biologics lead product candidate, LUMEVOQÂ® (GS010; lenadogene nolparvovec), has been submitted for marketing authorization in Europe for the treatment of Leber hereditary optic neuropathy (LHON), a rare mitochondrial disease primarily affecting adolescents and young adults and causing irreversible blindness . Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be delivered as a single treatment to each eye by intravitreal injection to provide patients with long-lasting functional visual recovery.
About LUMEVOQÂ® (GS010; lenadogen nolparvovec)
LUMEVOQÂ® (GS010; lenadogene nolparvovec) targets Leber’s hereditary optic neuropathy (LHON) based on a proprietary mitochondrial targeting sequence (MTS) technology platform, derived from research conducted at the Institut de la Vision in Paris, which, when combined with the gene of interest, allows the platform to specifically address defects inside mitochondria using an Adeno-Associated Virus (AAV) vector. The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be transported to the mitochondria via specific nucleotide sequences in order to restore the missing or deficient mitochondrial function. “LUMEVOQ” was accepted as an invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018.
Corporate Communications Director
Guillaume van Renterghem
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