An Open-label, Single-arm Study to Provide Efficacy and Safety Data of Voretigene Neparvovec Administered as Subretinal Injection in Japanese Patients With Biallelic RPE65 Mutation-associated Retinal Dystrophy

The purpose of this study is to provide safety and efficacy data for voretigene neparvovec, administered as subretinal injection, in Japanese patients with biallelic RPE65 mutation-associated retinal dystrophy.

开始日期2020-11-24

申办/合作机构

Novartis Pharmaceuticals Corp.

NCT03597399

/ Active, not recruitingN/A

A Post-Authorization, Multicenter, Longitudinal, Observational Safety Registry Study for Patients Treated With Voretigene Neparvovec in US

The objective of this study is to collect long-term safety information (i.e., for 5 years after treatment) associated with voretigene neparvovec-rzyl (vector and/or transgene), its subretinal injection procedure, the concomitant use of corticosteroids, or a combination of these procedures and products.
The enrollment period will last for two years from the first treatment following product approval (through 31March2020) and include a minimum of 40 patients.

开始日期2019-01-10

申办/合作机构

Spark Therapeutics, Inc.

NCT03602820

/ Active, not recruitingN/A

A Long-Term Follow-Up Study in Subjects Who Received an Adenovirus-Associated Viral Vector Serotype 2 Containing the Human RPE65 Gene (AAV2-hRPE65v2, Voretigene Neparvovec-rzyl) Administered Via Subretinal Injection

Multi-site, non-randomized, observational study, for up to 15 years after subretinal AAV2-hRPE65v2 administration for each subject. The study is a non-interventional, follow-up study of subjects who participated in previous AAV2-hRPE65v2 gene therapy clinical trials.

开始日期2015-06-01

申办/合作机构

Spark Therapeutics, Inc.

100 项与 Voretigene Neparvovec 相关的临床结果

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100 项与 Voretigene Neparvovec 相关的转化医学

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100 项与 Voretigene Neparvovec 相关的专利（医药）

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153

项与 Voretigene Neparvovec 相关的文献（医药）

2025-04-01·Journal of Genetic Counseling

Genetic counselors' awareness and attitudes regarding gene therapies

Article

作者: Ortiz, Damara ; Alabek, Michelle ; Durst, Andrea L. ; Walsh, Chelsey ; Miller, Rachel G.

Abstract:

There are 10 gene therapies (GTs) for hereditary conditions that are currently approved by the Food and Drug Administration (FDA). While prior research demonstrates that the majority of healthcare providers lack knowledge regarding GTs, this has not been explored within the genetic counseling profession. The authors hypothesize that the availability of GTs impacts the genetic counseling profession and that there is variable awareness on this topic among genetic counselors (GCs). We conducted a survey to assess GCs' familiarity with, comfort with, and frequency of discussing FDA‐approved GTs at the time of the survey, as well as GCs' perceived impact of and educational experiences related to GT. The survey was distributed through listservs and word of mouth from January through March 2021. One hundred of the 109 responses met eligibility criteria. Respondents were more familiar with onasemnogene abeparvovec‐xioi (ZOLGENSMA; Novartis Gene Therapies, Inc., Durham, NC, USA) than voretigene neparvovec‐rzyl (LUXTURNA; Spark Therapeutics, Inc., Philadelphia, PA, USA; p < 0.001). Familiarity with, comfort with, and frequency of discussing both GTs varied by specialty but not by years of experience. Fifty‐nine percent of respondents (58/98) reported that GTs impact their work, with differences by specialty but not by years of experience. The majority of respondents (93%; 90/97) felt that GCs should be comfortable discussing GTs with patients, and most respondents (83%; 79/95) were interested in additional GT training. Only 38% of respondents (36/95) recalled GT being included in their genetic counseling training program's curriculum, which may be skewed by recent growth of this field. Our results suggest that GCs feel that GTs impact their practice, have discrepant awareness and comfort in this area, and desire additional training on this topic. Further investigation into the actual impact and models for addressing training is warranted and will be critical as the number of approved GTs increases.

2025-02-01·BRITISH JOURNAL OF OPHTHALMOLOGY

12-month outcomes after voretigene neparvovec gene therapy in paediatric patients with RPE65 -mediated inherited retinal dystrophy

Article

作者: Bremond Gignac, Dominique ; Batté, Emilie ; Robert, Matthieu P ; Daruich, Alejandra ; Valleix, Sophie ; de Vergnes, Nathalie ; Rateaux, Maxence

Aims:

To report main outcomes and complications following voretigene neparvovec (Luxturna) treatment in paediatric patients.

Methods:

Records of patients under the age of 17 treated by subretinal administration of voretigene neparvovec for confirmed biallelic RPE65 -mediated inherited retinal dystrophy were retrospectively reviewed. Best-corrected visual acuity (BCVA) and data from spectral-domain optical coherence tomography, ultra-wide-field fundus imaging and Goldmann visual field (VF) were analysed at 12 months follow-up.

Results:

12 eyes of six patients (mean age: 7.8 years) were analysed. No intraoperative complications occurred. BCVA significantly improved at 12-month follow-up (mean LogMAR (logarithm of the minimal angle of resolution) BCVA: 1.0±0.8 at baseline vs 0.6±0.3 at 12 months, p=0.001). Mean central macular thickness and central outer nuclear layer thickness did not change at 12 months follow-up. VF V4e isopter did not show significant changes. Postoperatively complications included: elevated intraocular pressure in two eyes of the same patient, a parafoveal lamellar hole at 3 months post-treatment and atrophy on the injection site observed in all eyes except one, which significantly enlarged during 12 months (p=0.008).

Conclusions:

Most paediatric patients treated by voretigene neparvovec showed a significant increase in visual function at 12 months follow-up. None of the postoperative complications prevented gains in visual function.

2025-01-01·CANADIAN JOURNAL OF OPHTHALMOLOGY-JOURNAL CANADIEN D OPHTALMOLOGIE

“Blindness” is not a contraindication for voretigene neparvovec-rzyl treatment: a review of 9 cases

Article

作者: Héon, Elise ; Seamone, Mark ; Parameswarappa, Deepika C ; Kertes, Peter J ; Stephenson, Kirk A J ; Muni, Rajeev H ; Qian, Cynthia X ; Vincent, Ajoy

OBJECTIVE:

Biallelic RPE65 pathogenic variants may cause Leber congenital amaurosis (LCA). Voretigene neparvovec-rzyl (VN, Luxturna) is the only approved subretinal gene therapy that demonstrated benefit and safety. The eligibility criteria are vague and variable between centres. This is the first comprehensive outcome report of RPE65-LCA patients with World Health Organization blindness criteria vision treated with VN.

DESIGN:

Multicentre retrospective case series.

PARTICIPANTS:

Patients meeting the treatment criteria for VN who had best-corrected visual acuity (BCVA) <20/400 or visual field (VF)III4e isopter <10°.

METHODS:

Patients were followed for a mean of 11.1 ± 4.7 months. Age, sex, BCVA, central retinal thickness (CRT), retinal atrophy, VF, full-field stimulus testing (FST), and subjective impressions were assessed.

RESULTS:

Nine patients met the inclusion criteria (mean: BCVA 1.89 LogMAR, range: 1.4 - 2.7 LogMAR, mean age: 28.7-years-old, range: 17-59 years). Though VF area did not improve, FST improved in patients with better baseline FST (-8.83 dB vs -0.56 dB; p = 0.010), and better VFV4e (7245 vs 341^o2; p < 0.001) and III4e (596.1 vs 24.8^o2; p = 0.011) area. VA improved in younger (20 vs 32 years; p = 0.011) patients with thinner CRT^1mm (155 vs 193 µm; p = 0.038). VFV4e loss occurred in older (38 vs 19 years; p = 0.001) patients with worse baseline V4e area (1728 vs 8159^o2; p < 0.001). Subjective improvement in dim light navigation skills occurred in younger patients (20.3 vs 45.3 years; p < 0.001).

CONCLUSIONS:

Blindness is not a contraindication to VN treatment for RPE65-LCA. Superior results correlated with greater baseline FST but not with CRT^1mm, provided that measurable outer retinal structures persist.

306

项与 Voretigene Neparvovec 相关的新闻（医药）

2025-03-24

·BiG生物创新社

天价基因疗法，狂热追捧中踩下急刹

作者｜与安 2025年3月，罗氏制药（Roche）宣布对旗下基因疗法子公司Spark Therapeutics启动“根本性重组”，全额减记24亿美元商誉。财务数据显示，罗氏已累计为 Spark 投入超10亿美元的重组成本，其中包括2024 年1.84亿美元的前期重组费用和2025年预计达3.4亿美元的追加支出。然而，这场持续六年的资本实验最终却惨淡收场，成为基因疗法领域最惨痛的收购案例之一。而这并非个例。过去三个月，基因治疗领域在希望与阵痛中交织前行，此前昭示熊熊野心的各家药企中，不乏惨淡收场的身影。跨国药企的管线重组、战略收缩与突破转型，再次将这一赛道的复杂性与不确定性推至台前，同时也代表着曾受资本狂热吹捧的基因疗法领域正逐步向理性发展回归转折。 01 43亿美元的深刻教训技术瓶颈下散场的资本盛宴 2019年，罗氏以43亿美元天价收购Spark Therapeutics，看中的是其全球首个获批的遗传性视网膜病变疗法Luxturna，以及血友病A基因疗法SPK-8011（dirloctocogene samoparvovec）的3期临床潜力。彼时Spark市值仅22亿美元，而罗氏以溢价125%的代价收购，创下当时基因疗法领域最大并购案，折射出行业对基因疗法商业化的狂热预期。然而截至 2024 年，Luxturna 年销售额仅 2050 万美元，同比暴跌 59%.此外，SPK-8011 项目则因安全性问题被紧急叫停，不达预期的商业表现和无法突破的技术难题，直接让这项大手笔并购案从"战略收购" 降级为 "产品交易"。迅速击穿资本泡沫的主要是三重瓶颈。首先是载体免疫性难题。Luxturna依赖的AAV2血清型载体，暴露了基因疗法最顽固的技术瓶颈之一，即预存免疫与后天免疫的双重绞杀。全球约30%-60%人群因自然感染携带AAV中和抗体，导致Luxturna治疗前必须进行抗体筛查。但Spark采用的"血清型切换"策略并未突破根本限制，数据显示超60%患者治疗后仍产生新中和抗体，直接导致疗效持久性不足。而后天免疫的影响在于，即使患者通过预存免疫筛查，治疗后仍可能因免疫系统激活产生新的抗AAV2免疫反应。其次是产能不足。Spark费城工厂病毒载体年产能仅5000剂，在适应症人群本就狭窄（全球约6000名患者）的情况下，覆盖率仍旧不足85%。患者等待期过长，市场渗透未达预期。最后则是临床表现的滑铁卢，完成收购时被列为重点的SPK-8011因安全性问题被叫停，于去年年底宣布终止研究。当病毒载体的免疫原性、生产工艺、长期安全性三大技术关卡未获突破时，资本堆砌反而加速了泡沫破裂。 02 火热势头下的行业寒冬难以走出的商业化困境与此同时，罗氏的受挫并非个例。从去年开始，基因治疗领域接连出现“利空”消息，整体成绩远低于预期，市场表现陷入疲软，多家行业明星宣布因进展不佳关停管线或业务重组。 2月21日，辉瑞宣布将停止进一步开发和商业化血友病B基因疗法Beqvez（SPK-9001）。这款药物于2024年4月刚获FDA批准，定价高达350万美元（约合人民币2537万元），在2024年全美最贵药物前十排行榜中，仅次于定价 425 万美元的 Lenmeldy，并列第二。 2024年全美最贵十款药物而Beqvez从获批上市到决定撤回，还不满一年时间。辉瑞表示，如此决定是因为“患者及医生对血友病基因疗法表现出的兴趣有限”，上市至今，尚未有患者接受过该药治疗。与此同时，辉瑞彻底清空了基因疗法管线。随着Beqvez退出，该司已没有任何基因疗法处于临床或商业化阶段。实际上，彼时将基因治疗领域列为“优先发展领域”的辉瑞，近三年来正在逐步撤离基因治疗领域。2023年7月，该司将早期研发阶段的基因疗法打包以高达10亿美元的价格卖给了阿斯利康旗下的罕见病部门Alexion；2024年6月，其针对杜氏肌营养不良症（DMD）的基因疗法fordadistrogene movaparvovec因CIFFREO Ⅲ期试验结果未达主要终点，陷入停滞；而到9月，更是撤回了市场上所有批次的镰状细胞贫血疗法Oxbryta，同时终止所有相关试验。因其临床数据表明，该药在镰状细胞患者群体中的总体效益不再足以覆盖其风险。至年底，辉瑞则终止了与Sangamo Therapeutics的全球合作及许可协议，放弃共同开发治疗A型血友病的基因疗法候选药物giroctocogene fitelparvovec（Giro-vec），尽管该药在去年完成的AFFINE III期临床试验中已取得积极结果，并已准备向FDA和EMA提交监管申请。临时放弃的主要原因大致有二，一是BioMarin同适应症的基因疗法Roctavian商业化进展不佳，市场表现惨淡，可谓现成的前车之鉴；二是辉瑞血友病新型抗体疗法Marstacimab于不久前才获批上市，作为给药便利性更加的抗体，对于基因疗法的市场空间也造成了一定冲击。总得来说，就是彼时看来，这款药物似乎难以实现大卖，而三个月后折戟的Beqvez也确实证实了这一点。而就在辉瑞宣布从基因疗法退场的前一天，曾经估值百亿的蓝鸟生物宣布卖身私募资金，一同踩下急刹。 2018年初，凭借β地中海贫血疗法Zynteglo（定价280万美元）、脑肾上腺脑白质营养不良疗法Skysona（定价300万美元）和镰状细胞病疗法Lyfgenia（定价310万美元），蓝鸟生物一时声名鹊起，股价飙升至每股2300美元，市值突破300亿美元。其“一次性治愈”叙事吸引了黑石、富达等顶级资本加持，成为首个基因疗法领域“十角兽”企业。然而，高昂定价和狭窄的患者群体让其商业化之路变得十分困难。受限于保险公司等支付方在高昂定价下的报销审批挑战，三款核心产品2023年总销售额为2910万美元，至今未实现盈利，其中，Zynteglo 2023年销售额为1670万美元，截至2024年11月累计治疗35例患者；Lyfgenia则仅完成4例患者治疗，虽与Vertex的CRISPR疗法同日获批，却因价格高出90万美元且因存在安全问题遭FDA发出黑框警告，市场份额遭到挤压。去年10月，蓝鸟宣布裁员25%并启动重组，但现金流缺口仍无法填补。最终，该司以2900万美元（不足巅峰市值0.1%）被私募基金凯雷/SK资本收购，对赌条款上限仅1亿美元，蓝鸟最终“断翅”。 03 市场洗牌中的希望与曙光大额盈利并非不可能一度受挫的基因疗法领域也并非毫无商业进展。与前文几款药物相比，Sarepta Therapeutics的Elevidys表现似乎截然不同。这是全球首款DMD基因疗法，可对携带任何类型DMD致病基因变异的患者生效，很好地填补了临床上的空白。值得注意的是，这还是和罗氏联合开发的疗法，同样高成本、高定价，售价320万美元，位列全美最贵药物第四（见图1）。2023年6月获FDA批准，8月正式上市销售，2024年便首度扭亏为盈。 2024年6月，Serepta扩大了该药的适应症，从原来的仅限于 4～5 岁具有行走能力的儿童，扩展为 4 岁及以上的儿童（无论是否有行走能力），进一步拓宽了市场覆盖面。至第三季度，Sarepta实现了净利润同比增长113%的好成绩，一举达到7619万美元。而诺华在AAV基因疗法上的投入则更是成效亮眼，针对脊髓性肌萎缩症（SMA）的Zolgensma可谓销冠，据其财报显示，2024年的年销售额仍然取得了12.14亿美元的好成绩。 Zolgensma2024年净销售额或许还有一大关键原因在于适应症的选择。反观让多位巨头或挣扎前行、或认栽止损的血友病基因疗法，市场表现不佳主要还是因为有凝血因子替代疗法和双抗药物可供患者选择。自身售价不菲，疗效也并无绝对优势，在可替代方案充足的情况下，难以打开市场的情况或许也并非难以预料。然而罗氏并未完全放弃。一方面，Spark的剩余业务将整合至集团，重组不影响Luxturna的持续供应，费城创新中心的一部分研发职能也仍然予以保留；另一方面，通过与Dyno Therapeutics达成的10亿美元合作，利用后者人工智能平台开发神经疾病新型AAV载体，罗氏将转向开发针对神经疾病的下一代基因疗法。基因疗法的故事远未终章。尽管挑战重重，部分企业仍以战略调整与技术升级寻找生机。2025年的春天，基因治疗领域仍在冰火之间跋涉。但随着临床技术的进步、监管政策的完善和支付体系的创新，当基因治疗的商业化摆脱纯资本驱动，构建起“技术-临床-支付”的完整闭环，这一领域仍然有望在迎来更为明朗开阔的前景，重唱高歌，再次高速前行。参考文献 https://www.fiercepharma.com/pharma/roche-overhauls-spark-gene-therapy-unit-recording-24b-full-impairment https://www.fiercepharma.com/pharma/pfizer-empties-gene-therapy-portfolio-discontinues-hemophilia-treatment-beqvez https://www.fda.gov/news-events/press-announcements/fda-expands-approval-gene-therapy-patients-duchenne-muscular-dystrophy https://www.pharmaceutical-technology.com/features/the-most-expensive-drugs-in-the-us/ 公众号文章 BioSeedin柏思荟辉瑞“泪洒”基因疗法 https://news.bioon.com/article/98498658028f.html 各家药企官网新闻动态和财报数据共建Biomedical创新生态圈！如何加入BiG会员？

基因疗法并购临床3期

2025-03-11

·生物制品圈

43 亿→24 亿！罗氏基因治疗折戟背后的行业寒冬

2025 年 3 月，罗氏制药（Roche）宣布对旗下基因治疗子公司 Spark Therapeutics 启动 "根本性重组"，并确认 24 亿美元的商誉减值损失。这一决策不仅为其六年前 43 亿美元的收购案画上句号，更折射出整个基因治疗行业从资本狂欢到理性回归的阵痛。一、重组背后的财务黑洞罗氏在 2019 年以 43 亿美元收购 Spark 时，曾将其视为布局基因治疗领域的关键棋子。当时 Spark 的核心资产包括已获批的 Luxturna（遗传性失明疗法）和处于 III 期临床的血友病 A 基因疗法 SPK-8011。然而截至 2024 年，Luxturna 年销售额仅 2050 万美元，同比暴跌 59%，而 SPK-8011 项目因安全性问题被紧急叫停。财务报告显示，罗氏已累计为 Spark 投入超 10 亿美元重组成本：2024 年 1.84 亿美元的前期重组费用，加上 2025 年预计的 3.4 亿美元追加支出。更致命的是，24 亿美元的商誉减值意味着罗氏彻底放弃了对 Spark 未来商业价值的期待，将其从 "战略收购" 降级为 "产品交易"。二、行业寒冬中的多米诺骨牌罗氏的困境并非孤例。辉瑞在 2025 年初因 III 期临床失败全面退出基因治疗领域，蓝鸟生物（bluebird bio）更是从巅峰时的百亿美元市值缩水至 2900 万美元卖身私募。这些事件共同勾勒出行业的残酷现实：商业化困境：基因治疗产品定价虽高（如 Luxturna 曾达 85 万美元 / 剂），但患者群体极小，市场渗透率远低于预期。研发风险：基因递送技术（如 AAV 载体）的安全性问题频发，导致多个项目折戟临床后期。资本退潮：2024 年全球基因治疗领域融资额同比下降 67%，投资者对 "天价疗法" 的可持续性产生质疑。三、罗氏的战略突围与行业启示面对困局，罗氏采取了 "收缩 + 转型" 的双轨策略：一方面将 Spark 的剩余业务整合至母公司，保留费城创新中心的部分研发职能；另一方面，通过与 Dyno Therapeutics 的 10 亿美元合作，转向开发下一代神经疾病基因疗法。这种调整释放出两个重要信号：从激进并购到自主研发：罗氏承认早期通过收购快速布局的模式存在缺陷，转而强调内部技术平台的重要性。聚焦精准领域：放弃泛病种开发，将资源集中于神经疾病等具有技术壁垒的细分赛道。四、未来展望：基因治疗的十字路口尽管当前行业处于低谷，但技术进步仍在持续。CRISPR 基因编辑技术的临床突破、新型递送系统的研发，都为基因治疗保留了想象空间。罗氏的选择代表了大型药企的典型路径 —— 在风险与机遇间寻找平衡点，通过战略合作降低试错成本。对于行业而言，罗氏的案例警示后来者：基因治疗的商业化不能仅依赖资本驱动，需构建 "技术 - 临床 - 支付" 的完整闭环。随着监管政策的完善和支付体系的创新，这一领域有望在 5-10 年内迎来真正的爆发期，但在此之前，企业必须经历残酷的洗牌与重构。识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观不本站。

基因疗法临床3期财报并购

2025-03-11

·BioSpace

Roche Absorbs $2.4B Impairment in Overhaul of Spark Gene Therapy Unit

iStock, Besiki Kavtaradze Roche acquired Spark Therapeutics in 2019 for $4.8 billion. Roche is currently enacting a “ fundamental reorganisation ” of its gene therapy subsidiary Spark Therapeutics amid broader shifts in its pharmaceuticals business. The move, first revealed in the company’s financial report published in January 2025, comes after a strategic review of Spark’s prospects in the second half of 2024. Roche at the time was simultaneously implementing “restructuring activities” at Spark, which cost the company about $185 million last year. Roche has yet to settle on the specifics of the reorganization initiative, though it will involve the integration of some of Spark’s operations into the group’s broader Pharmaceuticals Division, as per the financial report. Roche’s preliminary estimate puts its restructuring costs at around $341 million. In connection with Spark’s overhaul, Roche will incur approximately $2.4 billion in goodwill impairment costs. This sum takes into consideration the $4.8 billion it paid to acquire the gene therapy specialist in 2019, as well as the current and future revenues and costs associated with the unit. “There was no surplus from the estimated future revenues of the Spark Therapeutics business to support the carrying value of the goodwill,” Roche wrote in its financial report, “neither were there any significant future synergistic benefits to other products within the Pharmaceuticals Division.” Roche’s decision to acquire Spark in 2019 rested heavily on two assets: the FDA-approved gene therapy Luxturna, indicated for a rare and heritable form of blindness, and the investigational SPK-8011, a one-time treatment for hemophilia A. At the time, SPK-8011 had just elicited a 97% response rate in patients with the bleeding disorder, giving Spark the push it needed to take the asset into late-stage studies. In December 2024, however, Roche shelved SPK-8011, as per an update to its federal clinical trials page that tags the Phase III trial of the asset as “withdrawn.” In a statement to Fierce Biotech at the time, a Roche spokesperson said that the decision to discontinue the SPK-8011 study is connected to a newer investigational therapy with an “enhanced function FVIII variant.” Roche also previously axed the Spark assets SPK-8016 for hemophilia A and SPK-3006 for Pompe disease, a rare genetic condition that damages muscles and nerves. Spark is also conducting research into eye, liver and neurodegenerative diseases, according to its pipeline . Luxturna has similarly been disappointing. In 2024, the gene therapy brought in just under $20.5 million , representing a 59% year-on-year decline in sales. Elsewhere in its gene therapy efforts, Roche in October 2024 put $1 billion on the line —on top of a $50 million upfront payment—in a back-heavy deal with Dyno Therapeutics to develop next-generation gene therapies for neurological indications.

并购基因疗法临床3期

100 项与 Voretigene Neparvovec 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11008	-	S01	DB13932

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
遗传性视网膜营养不良	加拿大	Novartis Pharmaceuticals Canada, Inc.	2020-10-13
莱伯先天性黑蒙	英国	Novartis AG	2020-02-10
双等位基因RPE65突变相关的视网膜变性疾病	美国	Spark Therapeutics, Inc.	2017-12-19

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
早发性严重视网膜营养不良	临床3期	美国	The Children's Hospital of Philadelphia	2012-10-01
早发性严重视网膜营养不良	临床3期	美国	Spark Therapeutics, Inc.	2012-10-01
2型利伯先天性黑朦症	临床2期	美国	Spark Therapeutics, Inc.	2010-11-01

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临床结果

适应症

分期

评价

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


EURETINA2024	N/A	-	-	Voretigene neparvovec-rzyl	糧鹽窪顧鑰遞築鏇艱範(鏇醖構齋鹽鑰鹽壓鏇簾) = 遞齋壓醖壓積糧齋築願壓醖獵糧鏇鹽鬱鑰鏇餘 (膚鏇鏇壓襯範構積夢鹹 ) 更多	-	2024-09-19
EURETINA2024	N/A	-	-	Control group (no VN therapy)	醖齋簾選廠醖淵願築鹹(鏇簾獵糧鬱膚憲壓齋襯) = 繭淵範選壓壓積醖遞選網顧顧網網鏇鹽範鑰艱 (網製遞窪齋範衊衊憲鹹 )	-	2024-09-19
EURETINA2024	N/A	-	-	Voretigene Neparvovec	獵顧製構衊鑰網積繭築(鏇繭鏇夢鑰鏇醖糧製選) = 願積憲壓獵範糧廠願艱遞鑰醖繭遞鏇網齋夢選 (鏇膚繭艱膚夢襯鑰範齋 )	-	2024-09-19
APAO2024	N/A	-	-	Voretigene Neparvovec (AAV2-hRPE65v2)	醖廠憲蓋艱範廠顧鏇鹹(衊顧鏇製簾醖遞窪膚窪) = 壓鏇膚築襯願憲襯蓋鹽鬱憲艱鹹鏇壓齋製餘築 (艱艱壓顧夢齋繭鬱觸遞 )	-	2024-02-22
RPE65-related inherited retinal dystrophies (ARVO2023)	N/A	-	12	Voretigene Neparvovec	蓋鹹築齋鑰襯壓蓋繭淵(鑰觸選積夢窪網築醖餘) = 夢製糧廠蓋鏇艱選構製醖蓋憲夢壓艱願築觸積 (遞鑰遞獵憲餘繭鹹艱構 ) 更多	积极	2023-04-23
ARVO2023	N/A	Sveinsson脉络膜视网膜萎缩	-	Voretigene neparvovec	選糧網鬱蓋壓積積襯觸(壓壓鹽餘餘淵餘鹹鹽齋) = 製簾鏇觸窪網憲窪鏇簾觸壓廠齋繭範膚範獵鹽 (夢鹽糧願艱夢襯構壓鏇 )	-	2023-04-23
RPE65-IRD (ARVO2023)	N/A	-	30	Voretigene Neparvovec	膚壓鑰選網淵齋製淵壓(築願鏇壓簾選製鹽齋衊) = 築憲夢積鬱顧構鑰鏇蓋餘鑰壓膚鏇廠糧憲廠衊 (積網積膚築顧廠壓餘鑰, 0.8)	积极	2023-04-23
EURETINA2022	N/A	-	-	Voretigene neparvovec 260 μL	衊淵願鏇鹽窪構築蓋壓(積鏇窪選憲築積廠鏇淵) = 製鏇製構簾艱襯壓淵製築糧夢蓋鹹構齋鹹糧鑰 (襯遞網齋獵夢鹽鑰憲顧 ) 更多	-	2022-09-01
EURETINA2022	N/A	-	-	Voretigene neparvovec 300 μL	衊淵願鏇鹽窪構築蓋壓(積鏇窪選憲築積廠鏇淵) = 積鬱膚繭鹹築襯築餘衊築糧夢蓋鹹構齋鹹糧鑰 (襯遞網齋獵夢鹽鑰憲顧 ) 更多	-	2022-09-01
EURETINA2022	N/A	遗传性视网膜营养不良	-	Voretigene Neparvovec	醖積夢築艱齋窪窪構壓(獵築廠鹽願壓壓夢願顧) = No clinically meaningful change in the mean BCVA was observed over 6 months of follow-up 鏇選製願糧壓簾憲鑰選 (膚膚積簾夢遞獵繭淵築 ) 更多	-	2022-09-01
EURETINA2022	N/A	-	-	Voretigene neparvovec	築艱鬱簾襯製衊艱願鬱(鏇窪餘窪獵艱醖構艱鹽) = Eight patients had non-ocular AEs; the most frequent was headache (n=4) 築夢構夢觸淵範衊鬱憲 (鬱膚觸蓋壓廠壓鬱廠壓 ) 更多	-	2022-09-01
EURETINA2022	N/A	小儿感染	-	Voretigene Neparvovec	獵衊築構鏇構齋淵顧積(襯鏇夢繭觸顧艱窪齋餘) = 1 patient developed bilateral sub-foveal outer retinal atrophy; 1 had atrophy related to the injection site in one eye 願範襯願鬱繭窪鬱鏇膚 (衊淵範獵夢觸齋構觸蓋 ) 更多	-	2022-09-01