GenSight Biologics Announces Successful Completion of LUMEVOQ® Engineering Batch Validating Implemented Corrective Actions

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  • A successful engineering batch validates actions taken to correct low yields in previous runs

  • Significantly improved yield to a level above all previous batches

  • Opinion of the Committee for Medicinal Products for Human Use (CHMP) still expected by Q3 2023

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, announced today today the successful completion of the first batch of engineering incorporating improvements in the manufacturing process of LUMEVOQ®, the company’s gene therapy for Leber’s Hereditary Optic Neuropathy (LHON). The batch was the first to implement a set of targeted corrective actions around better process control and enhanced on-site supervision that were identified by the Company and its manufacturing partner in the United States in April of this year1.

“The return from this engineering batch is extremely positive and gives us greater confidence in our ability to consistently manufacture LUMEVOQ with the highest quality of workmanship in the future,” commented Bernard Gilly, Chairman and CEO and co-founder of GenSight Biologics. “While this manufacturing setback has delayed the original timeline, we would like to reiterate to LHON patients and their physicians that we continue to work tirelessly to get LUMEVOQ approved to provide them with a therapeutic solution as quickly and safely as possible. .”

The successful engineering trial generated a drug substance whose viral genome titer reached the acceptance threshold, confirming that the manufacturing process of LUMEVOQ® is sound at the defined batch size. Additionally, the results demonstrate that the corrective actions successfully resolved issues in the downstream process filtration steps, resulting in a significant improvement in product yield that had not previously been achieved.

The company is now moving forward with renewed confidence to resume the early access program and clinical development and prepare for commercialization. GenSight still expects to receive the opinion of the European Committee for Medicinal Products for Human Use (CHMP) by the third quarter of 2023, followed by a commercial launch by the end of 2023.

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, LUMEVOQ® (GS010; lenadogene nolparvovec), is an investigational compound and has not been registered in any country at this time; a marketing authorization application is currently under review by the EMA for the treatment of Leber’s hereditary optic neuropathy (LHON), a rare mitochondrial disease primarily affecting adolescents and young adults and resulting in 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 LUMEVOQ® (GS010; lenadogen nolparvovec)

LUMEVOQ® (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 AAV (Adeno-Associated Virus) 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. LUMEVOQ® (GS010; lenadogene nolparvovec), is an investigational compound and has not been registered in any country at this stage; a marketing authorization application is currently being examined by the EMA.

1 In April 2022, GenSight announced a delay in completing validation batches of the Company’s product for Leber’s Hereditary Optic Neuropathy (LHON). This delay, granted by the European Medicines Agency (EMA), was necessary to implement the necessary operational adjustments with its industrial partner in order to ensure reliable execution of its process. The success of this first engineering campaign demonstrates that the adjustments implemented by GenSight in close collaboration with its manufacturing partner have resolved execution issues and even improved the execution of its manufacturing process.

GenSight Biologics

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Clothilde Caillet

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Rooney Partners

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Jeanene Timberlake

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Guillaume van Renterghem

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James Palmer

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Source: GenSight Biologics SA

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