Lenadogene nolparvovec

LUMEVOQ是GenSight Biologics公司开发的一种基因疗法，用于治疗Leber遗传性视神经病变（LHON）。 治疗效果：根据GenSight Biologics发布的REFLECTIII期临床试验五年疗效和安全性的最终结果，单次注射LUMEVOQ后五年，患者视力改善持续存在，且安全性良好。双侧注射相比单侧注射，具有额外获益。所有接受LUMEVOQ治疗的眼睛的平均视力相对于最低点都增加了超过+15个字母，达到了临床意义上的改善阈值。双侧注射患者的第一只受影响的眼睛平均改善了+19个ETDRS字母，第二只受影响的眼睛平均改善了+18个ETDRS 字母。单侧注射患者的第一只受影响的眼睛平均改善了+20个ETDRS字母，而安慰剂组的眼睛平均改善了+14个ETDRS字母。此外，79% 接受双侧治疗的患者能够在屏幕上阅读字母，而单眼治疗的患者这一比例为72%。 临床信息：LUMEVOQ的临床研究包括 I/IIa 期临床试验REVEAL以及III期临床试验 REFLECT、RESCUE 等。REVEAL 试验主要评估了LUMEVOQ在LHON患者中的安全性，结果显示其具有良好的安全性和耐受性，且在长期随访中得到了证实。REFLECT是一项随机、双盲、安慰剂对照的 III 期临床试验，共招募了98名因 ND4基因突变引起的LHON患者（其中最具代表性的是m.11778G>A位点突变，该位点突变是导致Leber遗传性视神经病变（LHON）最常见的病因之一，会破坏线粒体能量代谢，引发视神经萎缩），他们的视力丧失时间不超过一年。 RESCUE 试验则进一步证实了LUMEVOQ治疗LHON的有效性和安全性。 LUMEVOQ的适用对象是确诊为ND4基因突变引起的Leber遗传性视神经病变（LHON），且处于特定疾病阶段的成年患者。 1. 核心适用条件 明确的基因诊断：必须通过基因检测，确认存在导致LHON的ND4基因突变（其中最具代表性的是m.11778G>A位点突变）。这是使用LUMEVOQ的首要前提，因该疗法专门针对此基因突变类型设计。 特定疾病阶段：患者需处于视力丧失后的早期阶段。根据关键临床试验（如REFLECTIII 期）的入组标准，通常要求视力丧失时间不超过1 年，此时视神经损伤尚有恢复潜力，是治疗的最佳窗口期。 目标患者群体：主要适用于成年患者，目前公开的临床数据和获批信息中，核心研究对象为成年LHON患者，儿童或青少年患者的适用性需等待进一步临床数据支持。 2. 需排除的情况（非适用对象） 非 ND4基因突变导致的LHON患者，如由 ND1、ND6 等其他基因突变引发的 LHON，LUMEVOQ无法针对性起效。 视力丧失时间超过1年，且视神经已出现不可逆严重损伤的患者，治疗获益可能显著降低。 存在其他眼部疾病（如青光眼、视网膜脱离）或全身性严重疾病，可能影响治疗安全性或疗效的患者。 对LUMEVOQ成分,如腺相关病毒载体 AAV2过敏的患者，属于绝对禁忌。 GenSight Biologics是一家临床阶段的生物制药公司，专注于开发和商业化用于视网膜神经退行性疾病和中枢神经系统疾病的创新基因疗法。公司的产品线利用两个核心技术平台，即线粒体靶向序列（MTS）和光遗传学，来帮助保护或恢复患有致盲性视网膜疾病的患者的视力。除了 LUMEVOQ，公司还有处于I/II期临床开发阶段的GS030，用于治疗视网膜色素变性。 声明：本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

FDA authorization and Institutional Review Board (IRB) approval followed by QP release for expanded access treatment of one patient in the US Treatment scheduled in November 2025 at the University of Pittsburgh School of Medicine Ongoing review of early access dossier by French agency ANSM Technology transfer to new manufacturing partner on track to produce new batches for 2026 milestones PARIS--(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 that the Company’s gene therapy GS010/LUMEVOQ® has been granted regulatory authorizations for an Individual Patient Expanded Access in the United States. The authorizations mark the first set of regulatory green lights for the candidate product since the Company’s withdrawal of its European marketing authorization application in 2023. FDA authorization was granted for expanded access treatment of one eligible patient, based on an application by a physician at the University of Pittsburgh School of Medicine (UPMC) to the agency. The required approval of the school’s Institutional Review Board (IRB) was also granted. The patient is scheduled to be treated in November 2025. GS010/LUMEVOQ® is in Phase III of its clinical development as a treatment for the rare blinding disease Leber Hereditary Optic Neuropathy (LHON) caused by a mutated ND4 mitochondrial gene and has not received marketing authorization in any country. Company Maintains Strategic Focus on Manufacturing and Clinical Development “We are very pleased to be able to provide GS010/LUMEVOQ for the individual patient expanded access treatment at UPMC,” said Laurence Rodriguez, Chief Executive Officer of GenSight Biologics. “At this time, the company’s highest priority is on ensuring adequate supply for the study already under discussion with the ANSM and to support our discussions about an Early Access Program in France. These programs represent essential steps towards our ultimate goal of maximizing patient access to GS010/LUMEVOQ, given the high unmet need of those impacted by LHON.” The Company is currently completing the technology transfer to its new manufacturing partner, Catalent, which is expected to be finalized by year-end 2025. The transition will enable the production of new batches in 2026 to address the full scope of projected clinical and early access needs. GenSight Biologics is preparing for the launch in H2 2026 of the pivotal Phase III study RECOVER for GS010/LUMEVOQ®, while engaging with the French medicines agency ANSM regarding a dose-ranging study the agency requested in connection with an Early Access Program (AAC) in France. The study protocol submitted in August 2025, is under review with well-defined regulatory timelines. In parallel, the Company is pursuing opportunities to out-license GS010 in markets outside the USA and Europe, while exploring paid Early Access Programs worldwide. About Leber Hereditary Optic Neuropathy (LHON) LHON is a rare, maternally inherited mitochondrial genetic disease, characterized by the degeneration of retinal ganglion cells, which results in precipitous and usually irreversible vision loss and typically leads to legal blindness. The ND4 mitochondrial mutation is the most common of the mutations that cause LHON and is associated with the worst prognosis among the leading mutations. 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. GS010 is currently in clinical development, has not to date been granted marketing authorization in France or any other jurisdiction, and is therefore not available commercially. 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 GS010/LUMEVOQ® (lenadogene nolparvovec) GS010/LUMEVOQ® (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 is then shuttled to the mitochondria through specific nucleotidic sequences in order to restore the missing or deficient mitochondrial function. GS010/LUMEVOQ® (lenadogene nolparvovec) is in Phase III of its clinical development. It has not been granted marketing authorization in any country and is not available commercially.