Latest news with #LHON
Medscape
08-08-2025
- Health
- Medscape
Rare Eye Disease Gets First NHS Drug Recommendation
The National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending idebenone (Raxone, Chiesi Pharmaceuticals) for treating visual impairment from Leber hereditary optic neuropathy (LHON) in people aged 12 years or older. This is the first time that NICE has recommended a licensed medicine for use on the NHS in England to target the underlying causes of this genetic eye condition. Around 250 individuals in England may be eligible for treatment. Rare Mitochondrial Disorder LHON is a rare mitochondrial genetic disorder caused by a gene mutation that prevents retinal cells from producing enough energy to function normally. This leads to the dysfunction of retinal ganglion cells. The condition typically begins with painless blurring of central vision, which can progress to bilateral blindness within months. It mainly affects boys and young men. Until now, the standard of care was limited to supportive measures such as nutritional supplements, counselling, and lifestyle management, with no licensed medicines available to address the underlying pathophysiology of the disease. How the Drug Works Idebenone is a synthetic short-chain benzoquinone thought to restore the cells' ability to produce energy. This may allow inactive retinal cells to function again and potentially improve vision. NICE's recommendation was based primarily on results from the RHODOS trial, which randomised 85 patients aged 14 or older to receive 900 mg/day of idebenone or placebo for 24 weeks. At 6 months, idebenone produced clinically meaningful improvements in visual acuity, especially in patients with differing vision between eyes at baseline. Visual recovery occurred in 30% of treated patients compared with 10% in the placebo group. Improvements seen as early as one month. A follow-up observational study, RHODOS-OFU, found that improvements were largely maintained, even after idebenone treatment ended. Additional evidence came from the non-randomised long-term EAP, LEROS, and PAROS studies. Safety and Availability Idebenone was generally well tolerated, with adverse events occurring at similar rates to placebo. Reported side effects were typically mild and included headache, nasopharyngitis, and cough. The drug will be available as 150 mg tablets. The recommended dose is two tablets taken three times a day with food. Professor Patrick Yu-Wai-Man, a NICE committee member and professor of ophthalmology at the University of Cambridge, said the recommendation would "come as a great relief to the LHON community". The treatment will be supplied through a confidential commercial arrangement with a patient access scheme that provides a discount to the NHS. NHS England will make the treatment available within 3 months of NICE publishing its final guidance.
Yahoo
26-06-2025
- Business
- Yahoo
GenSight Biologics Announces Significant Milestone in New Manufacturing Partnership with Catalent
Upstream phase of LUMEVOQ® manufacturing process successfully transferred Milestone follows successful manufacture of drug product batch to be used for named early access program (AAC) and dose-ranging study in France Partnership expected to improve yield and upgrade analytical methods ahead of clinical use and regulatory submissions PARIS, June 26, 2025--(BUSINESS WIRE)--Regulatory News: 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 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@ Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
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
