Latest news with #LUMEVOQ®
Business Wire
26-06-2025
- Business
- Business Wire
GenSight Biologics Announces Significant Milestone in New Manufacturing Partnership with Catalent
PARIS--(BUSINESS WIRE)--Regulatory News: " ... a significant milestone in GenSight's strategy for securing the supply of LUMEVOQ for clinical use and for supporting the planned regulatory submissions ..." Share GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced the successful transfer of the upstream phase of the manufacturing process for LUMEVOQ ®, the Company's gene therapy candidate product for the rare mitochondrial disease Leber Hereditary Optic Neuropathy (LHON), to its new manufacturing partner, Catalent, Inc. ' 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,' commented Scott Jeffers, Chief Technical Officer of GenSight Biologics. ' 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. The level of collaboration and coordination between our teams has been truly impressive. ' Catalent, Inc. is a leading global contract development and manufacturing organization (CDMO) whose mission is to develop, manufacture, and supply products that help people live better and healthier lives. It is the only 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. Catalent successfully manufactured the drug product batch that was released as safe for human use in November 2024 and which will be the source of product supply for the named patient early access program (AAC) and dose-ranging study in France. After the tech transfer is completed, Catalent will also manufacture the drug for the planned global Phase III trial RECOVER and the regulatory submissions. The Partnership as a Critical Element of GenSight Biologics' Strategy Following agreement with the French agency ANSM to consider opening the AAC program expeditiously after approval of a dose-ranging study, the Company is currently implementing a financing strategy, as it continues to advance its global marketing authorization strategy: Preparation for regulatory consultations in the US and EU Planning for the global Phase III trial scheduled to begin in 2026, designed to meet FDA and EMA requirements Completing the transition to a new manufacturing partner (Catalent) to secure reliable clinical and commercial supply Advancing preparations for MHRA submission in the United Kingdom ' Our partnership with a manufacturing powerhouse like Catalent is a critical enabler of our global strategy,' explained Laurence Rodriguez, CEO of GenSight Biologics. ' The outputs from their work with our team will allow us to reassure regulators that we have surmounted the challenges we faced in the past and, ultimately, to fulfill our mission to provide LHON patients a safe and effective treatment for their urgent unmet need.' About GenSight Biologics GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics' pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics' lead product candidate, GS010 (lenadogene nolparvovec) is in Phase III in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics' product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery. About Leber Hereditary Optic Neuropathy (LHON) Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1 st eye, with the 2 nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of subjects have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. About LUMEVOQ ® (GS010; lenadogene nolparvovec) LUMEVOQ ® (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. 'LUMEVOQ' was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. LUMEVOQ ® (GS010; lenadogene nolparvovec) has not been registered in any country at this stage.
Business Wire
12-06-2025
- Business
- Business Wire
GenSight Biologics Reaches Agreement with ANSM for Opening of LUMEVOQ ® Named Early Access Program (AAC) in France
PARIS--(BUSINESS WIRE)--Regulatory News: GenSight Biologics ("GenSight Biologics" or the "Company") (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that it has reached agreement with the French medicines safety agency ANSM to consider expeditiously opening the French named early access program (AAC) for LUMEVOQ ® upon approval of a dose-ranging clinical study. Clear Path Forward for LUMEVOQ ® AAC Program in France Following extensive discussions, GenSight reached alignment with ANSM on a plan to open the LUMEVOQ ® AAC program. The agency has agreed that the authorization of a focused dose-ranging study could enable the opening of the early access program. The Company has submitted a preliminary design of the study to the agency and aims to finalize the protocol of a dose-ranging study in Q3 2025. In parallel, the Company will work with the agency to find a solution for patients who may not be included in the study but who may benefit from the AAC program. The AAC program is targeted to open in Q4 2025 at the latest. "In the interest of clearing the way for patients to be treated with LUMEVOQ, we are pleased to have achieved this important agreement with ANSM on a clear path forward for opening patient access to LUMEVOQ in France" said Laurence Rodriguez, Chief Executive Officer of GenSight Biologics."We are thankful for the ANSM's ongoing engagement and openness to discussion, as we work towards the shared goal of providing access to LHON patients who urgently need a safe and effective treatment." Financing Strategy to Support Regulatory Milestones GenSight is implementing a financing strategy to bridge operations through the opening of the AAC program and prepare for the new global Phase III clinical trial. The Company is actively pursuing various funding initiatives to support these critical regulatory milestones. ' We are working on our financing strategy to manage the bridge between now and the opening of the AAC program and the initiation of the Phase III study,' commented Jan Eryk Umiastowski, CFO of GenSight Biologics. "Our focus remains on securing the resources needed to execute our regulatory strategy while exploring additional funding opportunities." Advancing Core Marketing Authorization Strategy In parallel with the French AAC program developments, GenSight will continue to advance its global marketing authorization strategy: Preparation for regulatory consultations in the US and EU Planning for the global Phase III trial scheduled to begin in 2026, designed to meet FDA and EMA requirements Completing the transition to a new manufacturing partner to secure reliable clinical and commercial supply Advancing preparations for MHRA submission in the United Kingdom During this period, the Company will actively pursue additional non-dilutive funding opportunities, including licensing arrangements outside North America and Europe and potential M&A activities. Multiple Value Catalysts GenSight anticipates a number of significant value-creating events in 2025-2026: Financial Position The Company received €0.7 million of its Research Tax Credit (CIR) end of May, with the remaining €0.4 million to be paid in July 2025. Based on current operations and projections, GenSight's cash will support operations until mid-July 2025. The Company acknowledges that current funds are insufficient to cover operational requirements for the next 12 months. The planned financing initiatives are designed to extend the cash runway and enable the initiation of the Phase III clinical trial and UK MHRA marketing application for LUMEVOQ ®. As of June 12, 2025, the number of outstanding GenSight Biologics shares was 131,466,495 ordinary shares. Risk factors Detailed information regarding the Company, including its business, financial information, results, perspectives and related risk factors are contained (i) in the Company's 2024 Universal Registration Document filed with the AMF on April 8, 2025 under number D.25-0234. This document, as well as other regulated information and all of the Company's press releases, can be accessed on the Company's website ( and/or AMF ( The reader's attention is drawn to the risk factors related to the Company and its activities presented in chapter 3 of its 2024 URD in particular the liquidity risk presented in the chapter 3.1.1. About GenSight Biologics GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics' pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics' lead product candidate, GS010 (lenadogene nolparvovec) is in Phase III in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics' product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery. Forward-Looking Statements This press release contains forward-looking statements, including statements regarding product development prospects and financial projections. These statements do not constitute guarantees of future performance and involve risks and uncertainties. While the Company is actively pursuing financing solutions, regulatory requirements necessitate disclosure that material uncertainty exists regarding the Company's ability to continue as a going concern if sufficient funding cannot be secured. A further list and description of risks and uncertainties that could cause actual results to differ materially from those set forth in the forward-looking statements in this press release can be found in GenSight Biologics' regulatory filings with the French Autorité des Marchés Financiers. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements and estimates, which speak only as of the date hereof. Other than as required by applicable law, GenSight Biologics undertakes no obligation to update or revise the information contained in this press release.
Business Wire
13-05-2025
- Business
- Business Wire
GenSight Biologics Announces Approval of All Resolutions Supported by the Board of Directors at the Combined General Meeting of May 13, 2025
PARIS--(BUSINESS WIRE)--Regulatory News: The Combined General Meeting of shareholders of GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, took place on May 13, 2025, at 2:00 pm CEST at the Company's headquarters, 74 rue du Faubourg Saint-Antoine, 75012 Paris, France. The General Meeting was chaired by Laurence Rodriguez, Chief Executive Officer of the Company. All resolutions recommended by the Board of Directors and submitted to the Combined General Meeting were adopted, with the exception of Resolution A, which was rejected, in accordance with the recommendations of the Board. The quorum on first convening amounted to 56.51%. During the General Meeting, the Chief Executive Officer reviewed the key achievements of 2024, a year marked by careful cash management, strengthened financial structure with the participation of new investors and renewed support from historical shareholders, as well as the renegotiation of the Company's financial obligations. She also highlighted progress in manufacturing, including the optimization of GenSight's technology, preparation for technology transfer to a new manufacturing partner, and the successful mixing and pharmaceutical release of two active substance batches. On the regulatory and clinical fronts, the year saw the preparation of the Phase III RECOVER protocol, the planned submission of the Early Access Program (AAC) application in November 2024, and the continuation of strong scientific communication through international congresses and high-impact journal publications. Looking ahead to 2025, the Company will maintain strict spending discipline while actively pursuing refinancing opportunities. Strategic priorities include the finalization of the Phase III RECOVER protocol in alignment with health authorities, the submission of the LUMEVOQ ® registration dossier to the UK's Medicines and Healthcare products Regulatory Agency (MHRA), and the resumption of the Early Access Program in France through continued engagement with the French agency ANSM. On the manufacturing side, efforts will focus on successfully completing the technology transfer initiated at the end of 2024, optimizing LUMEVOQ ® production yields, and finalizing the quality control plan. In parallel, the Company will continue to explore strategic options through discussions already underway. The results of the vote by resolution and the videocast of the Annual General Meeting will be available on the Company's website in the Investors section ( About GenSight Biologics GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics' pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics' lead product candidate, GS010 (lenadogene nolparvovec) is in Phase III in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics' product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.
Associated Press
17-03-2025
- Business
- Associated Press
GenSight Biologics Announces LUMEVOQ® Scientific Updates at NANOS 2025
Regulatory News: GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that new scientific data and analyses on the gene therapy LUMEVOQ ® will be presented at the 51 st Annual Meeting of the North American Neuro-Ophthalmology Society (NANOS) in Tucson, Arizona, USA (March 15-20, 2025). Leading Leber Hereditary Optic Neuropathy (LHON) Key Opinion Leaders will share new data on predictive factors of response to LUMEVOQ ® treatment; on a comparison of the treatment outcomes from idebenone and LUMEVOQ ®; on real-world experience with LUMEVOQ ®; and on long-term outcomes from bilateral treatment with the gene therapy. Poster presentation: ' Predictive Factors of Improved Final Visual Outcome in Patients with Leber Hereditary Optic Neuropathy Treated with Lenadogene Nolparvovec Gene Therapy' Presenter: Robert C. Sergott, MD, Wills Eye Hospital, Philadelphia, USA Poster Number 209 Time: Monday, March 17th, 2025, 5:00 pm – 6:00 pm (MDT) Location: Arizona Ballroom 1-6 Poster presentation: 'Efficacy of Lenadogene Nolparvovec Gene Therapy Versus Idebenone: Two Matched Adjusted Indirect Comparisons' Presenter: Patrick Yu-Wai-Man, MD, PhD, University of Cambridge, Moorfields Eye Hospital, and the UCL Institute of Ophthalmology, UK Poster Number 186 Time: Monday, March 17th, 2025, 6:00 pm – 7:00 pm (MDT) Location: Arizona Ballroom 1-6 Poster presentation: ' Efficacy and Safety of Lenadogene Nolparvovec Gene Therapy for Leber Hereditary Optic Neuropathy in the Real-Life Setting ' Presenter: Mark L. Moster, MD, Wills Eye Hospital, Philadelphia, USA Poster Number 12 Time: Sunday, March 16th, 2025, 2:00 pm – 3:00 pm (MDT) Location: Arizona Ballroom 1-6 Platform presentation: 'Long-Term Outcomes of Bilateral Injection of Lenadogene Nolparvovec Gene Therapy for Leber Hereditary Optic Neuropathy' Presenter: Nancy J. Newman, MD, Emory University School of Medicine, Atlanta, USA Scientific Platform Session I Time: Monday, March 17th, 2025, 11:45 am – 12:00 pm (MDT) Location: Tucson Ballroom About GenSight Biologics GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics' pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics' lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics' product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery. About Leber Hereditary Optic Neuropathy (LHON) Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1 st eye, with the 2 nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of subjects have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. About LUMEVOQ ® (GS010; lenadogene nolparvovec) LUMEVOQ ® (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. 'LUMEVOQ' was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. LUMEVOQ ® (GS010; lenadogene nolparvovec) has not been registered in any country at this stage. Chief Financial Officer Jan Eryk Umiastowski [email protected] Advisors Investor Relations Guillaume van Renterghem +41 (0)76 735 01 31 SOURCE: GenSight Biologics Copyright Business Wire 2025. PUB: 03/17/2025 02:30 AM/DISC: 03/17/2025 02:29 AM
Yahoo
17-03-2025
- Business
- Yahoo
GenSight Biologics Announces LUMEVOQ® Scientific Updates at NANOS 2025
PARIS, March 17, 2025--(BUSINESS WIRE)--Regulatory News: GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, today announced that new scientific data and analyses on the gene therapy LUMEVOQ® will be presented at the 51st Annual Meeting of the North American Neuro-Ophthalmology Society (NANOS) in Tucson, Arizona, USA (March 15-20, 2025). Leading Leber Hereditary Optic Neuropathy (LHON) Key Opinion Leaders will share new data on predictive factors of response to LUMEVOQ® treatment; on a comparison of the treatment outcomes from idebenone and LUMEVOQ®; on real-world experience with LUMEVOQ®; and on long-term outcomes from bilateral treatment with the gene therapy. Poster presentation: "Predictive Factors of Improved Final Visual Outcome in Patients with Leber Hereditary Optic Neuropathy Treated with Lenadogene Nolparvovec Gene Therapy" Presenter: Robert C. Sergott, MD, Wills Eye Hospital, Philadelphia, USA Poster Number 209 Time: Monday, March 17th, 2025, 5:00 pm – 6:00 pm (MDT) Location: Arizona Ballroom 1-6 Poster presentation: "Efficacy of Lenadogene Nolparvovec Gene Therapy Versus Idebenone: Two Matched Adjusted Indirect Comparisons" Presenter: Patrick Yu-Wai-Man, MD, PhD, University of Cambridge, Moorfields Eye Hospital, and the UCL Institute of Ophthalmology, UK Poster Number 186 Time: Monday, March 17th, 2025, 6:00 pm – 7:00 pm (MDT) Location: Arizona Ballroom 1-6 Poster presentation: "Efficacy and Safety of Lenadogene Nolparvovec Gene Therapy for Leber Hereditary Optic Neuropathy in the Real-Life Setting" Presenter: Mark L. Moster, MD, Wills Eye Hospital, Philadelphia, USA Poster Number 12 Time: Sunday, March 16th, 2025, 2:00 pm – 3:00 pm (MDT) Location: Arizona Ballroom 1-6 Platform presentation: "Long-Term Outcomes of Bilateral Injection of Lenadogene Nolparvovec Gene Therapy for Leber Hereditary Optic Neuropathy" Presenter: Nancy J. Newman, MD, Emory University School of Medicine, Atlanta, USA Scientific Platform Session I Time: Monday, March 17th, 2025, 11:45 am – 12:00 pm (MDT) Location: Tucson Ballroom About GenSight Biologics GenSight Biologics S.A. is a clinical-stage biopharma company focused on discovering and developing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics' pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics' lead product candidate, GS010, is in Phase III trials in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics' product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery. About Leber Hereditary Optic Neuropathy (LHON) Leber Hereditary Optic Neuropathy (LHON) is a rare maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells that results in brutal and irreversible vision loss that can lead to legal blindness, and mainly affects adolescents and young adults. LHON is associated with painless, sudden loss of central vision in the 1st eye, with the 2nd eye sequentially impaired. It is a symmetric disease with poor functional visual recovery. 97% of subjects have bilateral involvement at less than one year of onset of vision loss, and in 25% of cases, vision loss occurs in both eyes simultaneously. About LUMEVOQ® (GS010; lenadogene nolparvovec) LUMEVOQ® (GS010; lenadogene nolparvovec) targets Leber Hereditary Optic Neuropathy (LHON) by leveraging a mitochondrial targeting sequence (MTS) proprietary technology platform, arising from research conducted at the Institut de la Vision in Paris, which, when associated with the gene of interest, allows the platform to specifically address defects inside the mitochondria using an AAV vector (Adeno-Associated Virus). The gene of interest is transferred into the cell to be expressed and produces the functional protein, which will then be shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. "LUMEVOQ" was accepted as the invented name for GS010 (lenadogene nolparvovec) by the European Medicines Agency (EMA) in October 2018. LUMEVOQ® (GS010; lenadogene nolparvovec) has not been registered in any country at this stage. View source version on Contacts GenSight Biologics Chief Financial OfficerJan Eryk Umiastowskijeumiastowski@ LifeSci Advisors Investor RelationsGuillaume van Renterghemgvanrenterghem@ +41 (0)76 735 01 31 Sign in to access your portfolio
