更新于：2024-11-21

Olenasufligene relduparvovec(Lysogene SA)

更新于：2024-11-21

概要

研发状态

概要

基本信息

药物类型

腺相关病毒基因治疗

别名

Aavrh10-hsgsh、Dna (recombinant adeno-associated virus 2 vector lys-saf302 human n-sulfoglucosamine sulfohydrolase cdna-specifying)、Olenasufligene relduparvovec

+ [4]

靶点

SGSH

作用机制

SGSH替代物(磺氨基葡糖硫酸胺酶替代物)、基因转移

治疗领域

遗传病与畸形内分泌与代谢疾病皮肤和肌肉骨骼疾病+ [1]

在研适应症-

非在研适应症

黏多醣贮积症III型

原研机构

Lysogene SA

在研机构-

非在研机构

Lysogene SA

最高研发阶段无进展临床2/3期

首次获批日期-

最高研发阶段(中国)-

特殊审评快速通道 (美国)、孤儿药 (欧盟)

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序列信息

Sequence Code 612039214

关联

项与 Olenasufligene relduparvovec(Lysogene SA) 相关的临床试验

NCT03612869 / Unknown status临床2/3期

Open-label, Single-arm, Multi-center Study of Intracerebral Administration of Adeno-associated Viral (AAV) Serotype rh.10 Carrying Human N-sulfoglucosamine Sulfohydrolase (SGSH) cDNA for Treatment of Mucopolysaccharidosis Type IIIA

MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance. LYS-SAF302 is a gene therapy which is intended to deliver a functional copy of the SGSH gene to the brain. This is a phase 2-3 study to assess the efficacy in improving or stabilizing the neurodevelopmental state of MPS IIIA patients.

开始日期2018-12-17

申办/合作机构

Lysogene SA

100 项与 Olenasufligene relduparvovec(Lysogene SA) 相关的临床结果

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100 项与 Olenasufligene relduparvovec(Lysogene SA) 相关的转化医学

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100 项与 Olenasufligene relduparvovec(Lysogene SA) 相关的专利（医药）

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项与 Olenasufligene relduparvovec(Lysogene SA) 相关的文献（医药）

2020-06-01·Molecular Therapy - Methods & Clinical Development2区 · 医学

AAVrh10 Vector Corrects Disease Pathology in MPS IIIA Mice and Achieves Widespread Distribution of SGSH in Large Animal Brains

2区 · 医学

ArticleOA

作者: Tamang, Sarah J ; Beard, Helen ; King, Barbara M ; Hemsley, Kim M ; Lau, Adeline A ; Hocquemiller, Michaël ; Snel, Marten F ; Gomila, Cathy ; Mei, Xin ; Giersch, Laura ; Laufer, Ralph ; Winner, Leanne K ; Plavsic, Mark ; Trim, Paul J ; Ausseil, Jérôme ; Douglass, Meghan L ; Neumann, Daniel

Patients with mucopolysaccharidosis type IIIA (MPS IIIA) lack the lysosomal enzyme sulfamidase (SGSH), which is responsible for the degradation of heparan sulfate (HS). Build-up of undegraded HS results in severe progressive neurodegeneration for which there is currently no treatment. The ability of the vector adeno-associated virus (AAV)rh.10-CAG-SGSH (LYS-SAF302) to correct disease pathology was evaluated in a mouse model for MPS IIIA. LYS-SAF302 was administered to 5-week-old MPS IIIA mice at three different doses (8.6E+08, 4.1E+10, and 9.0E+10 vector genomes [vg]/animal) injected into the caudate putamen/striatum and thalamus. LYS-SAF302 was able to dose-dependently correct or significantly reduce HS storage, secondary accumulation of GM2 and GM3 gangliosides, ubiquitin-reactive axonal spheroid lesions, lysosomal expansion, and neuroinflammation at 12 weeks and 25 weeks post-dosing. To study SGSH distribution in the brain of large animals, LYS-SAF302 was injected into the subcortical white matter of dogs (1.0E+12 or 2.0E+12 vg/animal) and cynomolgus monkeys (7.2E+11 vg/animal). Increases of SGSH enzyme activity of at least 20% above endogenous levels were detected in 78% (dogs 4 weeks after injection) and 97% (monkeys 6 weeks after injection) of the total brain volume. Taken together, these data validate intraparenchymal AAV administration as a promising method to achieve widespread enzyme distribution and correction of disease pathology in MPS IIIA.

2019-09-01·Human Gene Therapy2区 · 医学

An Improved Adeno-Associated Virus Vector for Neurological Correction of the Mouse Model of Mucopolysaccharidosis IIIA

2区 · 医学

Article

作者: Hocquemiller, Michaël ; Liao, Aiyin ; Danos, Olivier ; Parker, Helen ; Agúndez, Leticia ; Taylor, Jessica T. ; Palomar, Nuria ; Henckaerts, Els ; Gray, Anna L. ; Linden, R. Michael ; Holley, Rebecca J. ; O'Leary, Claire ; Youshani, Amir S. ; Bigger, Brian W. ; Gleitz, Hélène F.

Patients with the lysosomal storage disease mucopolysaccharidosis IIIA (MPSIIIA) lack the lysosomal enzyme N-sulfoglucosamine sulfohydrolase (SGSH), one of the many enzymes involved in degradation of heparan sulfate. Build-up of un-degraded heparan sulfate results in severe progressive neurodegeneration for which there is currently no treatment. Experimental gene therapies based on gene addition are currently being explored. Following preclinical evaluation in MPSIIIA mice, an adeno-associated virus vector of serotype rh10 designed to deliver SGSH and sulfatase modifying factor 1 (SAF301) was trialed in four MPSIIIA patients, showing good tolerance and absence of adverse events with some improvements in neurocognitive measures. This study aimed to improve SAF301 further by removing sulfatase modifying factor 1 (SUMF1) and assessing if expression of this gene is needed to increase the SGSH enzyme activity (SAF301b). Second, the murine phosphoglycerate kinase (PGK) promotor was exchanged with a chicken beta actin/CMV composite (CAG) promotor (SAF302) to see if SGSH expression levels could be boosted further. The three different vectors were administered to MPSIIIA mice via intracranial injection, and SGSH expression levels were compared 4 weeks post treatment. Removal of SUMF1 resulted in marginal reductions in enzyme activity. However, promotor exchange significantly increased the amount of SGSH expressed in the brain, leading to superior therapeutic correction with SAF302. Biodistribution of SAF302 was further assessed using green fluorescent protein (GFP), indicating that vector spread was limited to the area around the injection tract. Further modification of the injection strategy to a single depth with higher injection volume increased vector distribution, leading to more widespread GFP distribution and sustained expression, suggesting this approach should be adopted in future trials.

项与 Olenasufligene relduparvovec(Lysogene SA) 相关的新闻（医药）

2023-05-11

·BioSpace

Forge Biologics Announces Eight Presentations at the Upcoming 2023 Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Four oral presentations and four scientific poster presentations highlight Forge’s AAV manufacturing capabilities and a clinical update on its FBX-101 AAV gene therapy for patients with Krabbe disease COLUMBUS, Ohio--(BUSINESS WIRE)--Forge Biologics, a leading manufacturer of genetic medicines, announced today that eight presentations, including four oral presentations, will be delivered during the upcoming 26th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), taking place May 16-20, 2023, in Los Angeles, California. “Forge’s presentations at ASGCT highlight the 2023 accomplishments of our team, particularly cell line and plasmid developments, as we expand our bespoke AAV manufacturing platform capabilities to deliver advanced solutions for gene therapy developers,” said Timothy J. Miller, Ph.D., CEO, President, and Co-Founder of Forge Biologics. “We are delighted to showcase our team’s contributions and Forge’s progress for patients as well we continue to provide solutions for industry-wide gene therapy manufacturing challenges.” SESSION PRESENTATIONS Title: The CDMO-Developer Partnership: Solving Complex Challenges in Gene Therapy Development & Manufacturing, Together Moderator: Timothy J. Miller, Ph.D., CEO, President, and Co-Founder, Forge Biologics Panelists: Paul Bresge, Chief Executive Officer, Ray Therapeutics David Dismuke, Ph.D., Chief Technical Officer, Forge Biologics Sadik Kassim, Ph.D., Chief Technical Officer, Danaher Shen Shen, Ph.D., Senior Director, AAV Technologies, Vertex Pharmaceuticals Session: Exhibitor Showcase Date and Time: Wednesday, May 17, 2023, 9:15-9:45 a.m. PT Room: 409 AB Title: Building an Improved Platform Process for AAV Manufacturing Presenter: David Dismuke, Ph.D., Chief Technical Officer, Forge Biologics Session: Tools & Technology Forum Date and Time: Wednesday, May 17, 2023, 3:45-4:00 p.m. PT Room: Presentation Theater in the Exhibit Hall Title: Strategic Collaborations Today for the Gene Therapies of Tomorrow Presenter: Panel hosted by Labcorp featuring John Maslowski, Chief Commercial Officer, Forge Biologics Session: Industry Sponsored Symposium Date and Time: Friday, May 19, 2023, 12:00-1:30 p.m. PT Room: 409 AB Title: RESKUE: First in Human Phase 1/2 Clinical Trial of Intravenous FBX-101 (AAVrh10.hGALC) Administered After Cord Blood Transplantation in Patients with Infantile Krabbe Disease Presenter: Maria Escolar, M.D., Chief Medical Officer, Forge Biologics Session: Gene and Cell Therapy Trials in Progress Date and Time: Saturday, May 20, 2023, 11:30-11:45am PT Room: Concourse Hall 150 and 151 POSTER PRESENTATIONS Title: Development of Chromatography Based Separation of Empty and Full Capsids in rAAV Manufacturing Process Presenter/poster: Corben Davis, Senior Scientist, Downstream Process Development (Poster Board no. 398) Date and Time: Wednesday, May 17, 2023, 12:00 p.m. PT Title: The Impact of the In Vitro Diagnostic Medical Devices Regulation (EU) 2017/746 on Advanced Therapy Medicinal Product Developers Presenter/poster: Claire Marasco, Senior Manager, Regulatory Affairs (Poster Board no. 995) Date and Time: Thursday, May 18, 2023, 12:00 p.m. PT Title: Navigating the EU Clinical Trial Regulation (536/2014): Key Considerations for ATMP Developers Presenter/poster: Claire Marasco, Senior Manager, Regulatory Affairs (Poster Board no. 799) Date and Time: Wednesday, May 17, 2023, 12:00 p.m. PT Title: Advanced Characterization of DNA Molecules in rAAV Vector and Plasmid DNA Manufacturing Preparations by Next Generation Preparations Sequencing Presenter/poster: Esko Kautto, Ph.D., Scientist II, Bioinformatics (Poster Board no. 1345) Date and Time: Friday, May 19, 2023, 12:00 p.m. PT Full abstracts are available on the ASGCT Annual Meeting website, and conference participants can also access posters through the ASGCT website. Forge Biologics will be at booth #540 in the Exibitor Hall. About Forge Biologics Forge Biologics is a hybrid gene therapy contract manufacturing and clinical-stage therapeutics development company. Forge’s mission is to enable access to life-changing gene therapies and help bring them from idea to reality. Forge’s 200,000 square foot facility, the Hearth, utilizes 20 cGMP suites in Columbus, Ohio, to serve as its headquarters. The Hearth is a custom-designed cGMP facility dedicated to AAV manufacturing and hosts scalable, end-to-end manufacturing services. Offerings include process and analytical development, plasmid DNA manufacturing, viral vector manufacturing, final fill, as well as regulatory consulting support to accelerate gene therapy programs from preclinical through clinical and commercial stage manufacturing. By taking a patients-first approach, Forge aims to accelerate the timelines of these transformative medicines for those who need them the most. To learn more, visit .

基因疗法

2023-01-05

·研发客

2022临床研究失败案例启示录：出其不意或早有预见？

2022年的临床研究领域，成功（或取得进展）与失败的情景依旧上演。比如乳腺癌领域，DS-8201的惊艳数据，将改变HER2低表达乳腺癌患者的治疗格局；然而，三阴乳腺癌仍然属于难以攻破的“老大难”。在阿尔茨海默症（AD）领域，虽然卫材/渤健公布了lecanemab的Ⅲ期研究数据证明了Aβ靶点的意义，但突破还需假以时日。还有免疫治疗领域，在联合方案拓展适应症上碰壁。基因治疗领域，则有多个临床研究暂停或终止。研发客总结了2022年里上述领域里较为引人注目的临床失败或暂停的案例。这些案例提醒我们在为成功欢呼的同时，也要注意到这个快速发展领域里的“短板”还将经受更多的时间与实践的捶打。免疫疗法：联合与新机理失利免疫治疗领域中，无论是成熟的PD-1/L1抗体，还是后起之秀，2022年观察到的临床研究失败主要集中在联合疗法上。一年间，O药披露了4项临床失败：1月，其联合5-氟尿嘧啶/亚叶酸/奥沙利铂以及贝伐珠单抗标准疗法（SOC）一线治疗转移性结直肠癌的临床研究CheckMate 9X8未达临床终点。3月，BMS和Nektar宣布，O药+NKTR-214一项一线治疗黑色素瘤的Ⅲ期临床PIVOT IO-001未达无进展生存期（PFS）和客观缓解率（ORR）的主要终点，总生存期（OS）也没有统计学意义的改善，之后公司分析了针对肾细胞癌和膀胱癌的两项临床研究，最终在4月宣布终止联合方案的所有临床研究。5月，O药+伊匹木单抗（ipilimumab）一线治疗尿路上皮癌的Ⅲ期临床CheckMate-901未达改善OS的临床终点；2个月后，这一联合方案又在局限性肾细胞癌的Ⅲ期临床CheckMate-914中失利，未达无病生存期（DFS）的主要临床终点。K药方面，3月，默沙东终止K药联合PARP抑制剂奥拉帕利治疗疾病进展转移性前列腺癌（曾接受化疗和醋酸阿比特龙或恩杂鲁胺治疗）的Ⅲ期临床，因为研究结果没有显示出OS的益处。8月初，K药联用Lenvima（仑伐替尼/乐伐替尼）治疗肝细胞癌的一项Ⅲ期临床未达主要终点。阿斯利康的PD-L1单抗Imfinzi也有一项联合放化疗治疗晚期宫颈癌的Ⅲ期研究未达PFS的主要终点。罗氏在TIGIT上遭遇失利，其研发的TIGIT抗体tiragolumab+PD-L1单抗Tecentriq相继在小细胞肺癌和非小细胞肺癌的两项Ⅲ期临床中宣告失败，两项研究均未达到PFS的主要终点。8月，Innate Pharma宣布，免疫检查点抑制剂NKG2A抗体monalizumab联合西妥昔单抗治疗复发或转移性头颈部鳞状细胞癌的Ⅲ期临床INTERLINK-1失败。这项由阿斯利康赞助的研究已经告停。通过联合治疗拓展适应症，已经是免疫治疗中不言而喻的“战场”。但联合治疗的复杂，也让药企不得不为此“买单”。O药+ipilimumab是个经典组合，已经先后斩获黑色素瘤、非小细胞肺癌、肝细胞癌、肾细胞癌等适应症，但拓展适应症显然不会一帆风顺，2022年两项研究折戟。在这些联合失败案例中，不只有PD-1/L1，联用药中还包括不少近年被看好的新靶点、新机理。NKTR-214是聚乙二醇化IL-2受体激动剂，BMS与Nektar在2018年达成这一产品的交易，总金额高达36亿美元，但现在所有的联合研究均已终止。TIGIT靶点在2014年被发现，它在T细胞和NK细胞表面表达，抑制这些免疫细胞发挥作用。理论上，TIGIT抗体可以同时解除T细胞和NK细胞的免疫抑制，在联合方面的潜力很被业内看好。罗氏是该领域最早的开发者，在2020年的ASCO大会上，罗氏公布了PD-L1+TIGIT在非小细胞肺癌中的积极数据，这也促成该领域的交易变得更为活跃，BMS、GSK，国内的百济神州和君实都有这方面交易，百济神州和君实均是向海外公司授权。在药物研发上，机理可行、早期数据不错，并不意味进一步验证过程中不会遇到问题。先行者罗氏的失利是否让这一新领域笼上阴影，尚未可知。业界大概最不希望看到TIGIT重蹈IDO的覆辙。另外，我们也留意到更早期阶段的新靶点产品临床研究失败案例。由陈列平教授创立的Nextcure，在2022年11月发布的第三季度财报中宣布终止Siglec-15抗体NC318的临床研究，因为其单药在部分Ⅱ期临床中没有观察到响应，但由耶鲁大学开展的联合PD-1抗体的临床试验还会继续。Siglec-15这个靶点由陈列平团队发现，其主要在多种肿瘤细胞和与肿瘤相关的巨噬细胞中表达，可抑制 T 细胞活性。NC318是此前唯一进入临床的Siglec-15抗体。寻找新的有效靶点的征程从来没有停止，且依然任重而道远。AD、部分乳腺癌攻坚战阿尔茨海默病（AD）出现失败案例，我们似乎已经见怪不怪，这个领域的药物开发某种程度上属于“地狱模式”。2022年11月，罗氏宣布其Aβ单抗gantenerumab两项Ⅲ期临床——针对阿尔茨海默病、轻度阿尔茨海默病性痴呆症导致的认知障碍 (MCI)的GRADUATE Ⅰ 和Ⅱ——未达改善认知损伤的主要终点，且在清除β-淀粉样蛋白（Aβ）的水平方面也低于预期。这已经是gantenerumab的第二次失败，2014年，因为Ⅲ期临床的数据不佳，罗氏宣布停止其临床研究。2017年，罗氏重启了gantenerumab的临床。再一次的失败，让gantenerumab前景越发不乐观。乳腺癌领域中也有难啃的骨头。2022年5月，阿斯利康宣布PARP抑制剂奥拉帕利在联合ATR抑制剂ceralasertib或WEE1抑制剂adavosertib二/三线治疗转移性三阴乳腺癌（mTNBC）中的Ⅱ期临床研究（VIOLETTE）失败。数据显示，无论是否存在同源重组修复（HRR）基因突变和BRCA突变，与奥拉帕利单药治疗相比，奥拉帕利+ceralasertib未能在PFS上体现出改善；奥拉帕利+adavosertib组由于发生超出预期的血液学毒性，已提前停药。除了mTNBC，ER+/HER2-乳腺癌也遭遇连环失败，且出于同一类靶点产品。2022年3月，赛诺菲公布雌激素受体降解剂（SERD）amcenestrant治疗ER+/HER2-局部晚期或转移性乳腺癌的Ⅱ期临床研究结果，未达PFS的主要终点。8月，该产品的Ⅲ期临床AMEERA-5也未达主要终点。赛诺菲因此宣布终止该药的全球临床开发计划。4月，罗氏第一季度财报中，公布其SERD药物giredestrant治疗ER+/HER2-局部晚期或转移性乳腺癌的Ⅱ期临床acelERA未达PFS的主要终点。HR+乳腺癌主要的治疗方式是内分泌治疗，但约有30%~50%患者在接受治疗后耐药。SERD药物作为一种新策略，被认为是解决内分泌耐药的重要治疗方式。在SERD药物开发领域，主要的竞争者们依然是跨国药企，布局的包括赛诺菲、罗氏、阿斯利康和礼来。赛诺菲已经暂时退出竞争，罗氏还没有放弃其他的临床研究。阿斯利康和礼来的产品尚在临床中，会在未来一两年内陆续公布临床结果。目前，唯一取得积极临床结果的是Menarini Group和Radius Health共同开发的elacestrant，在二线或三线HR+乳腺癌的治疗中，同时被FDA纳入优先审评。这个领域依然有许多问题有待证实。基因治疗的大考2022年初，我们总结了2021年临床暂停的基因治疗研究和原因（拓展阅读多项基因治疗临床试验因何暂停）。2022年里同样观察到一些类似的暂停或失败的案例，这些临床暂停或失败覆盖了不同的技术领域。在病毒载体领域，6月，安斯泰来治疗晚发性庞贝病的AT845因为一名受试者发生周围感觉神经病变的严重不良事件（SAE），被FDA暂停了Ⅰ/Ⅱ期临床。11月，Lysogene公布了治疗IIIA型粘多糖贮积症的基因疗法LYS-SAF302的Ⅱ/Ⅲ期临床研究数据，结果未达主要和关键次要疗效终点，但在小于30月龄的受试者中，认知有统计学上的显著改善。安全性问题在基因治疗中一直最受关注，之前的总结中可以看到多数的临床暂停都是因为安全性风险，企业多被要求证明这些安全问题与药物的使用无关。AT845采用的是腺相关病毒（AAV）作为载体，较少随机插入的风险，通常被认为比慢病毒更安全。但近年来很多暂停的基因治疗源自AAV疗法，其安全性风险也有很多问题有待解决。安斯泰来此前已经接连遭遇基因疗法的失利，在研的AT132因为肝毒性已经造成4名受试者先后死亡。LYS-SAF302同样采用AAV，面对的是疗效问题，随着越来越多基因疗法进入晚期临床，疗效达不到预期的时有发生。从诞生就被寄予厚望的CRISPR基因编辑，要过的则是体内一关。11月，CRISPR领域领头公司之一的Editas，公布体内基因编辑疗法EDIT-101用于治疗Leber先天性黑蒙症的Ⅰ/Ⅱ期临床试验BRILLIANCE初步结果，14名接受治疗的受试者中3例达到了反应者阈值，在最佳矫正视力 (BCVA) (LogMAR >0.3) 方面具有临床意义的改善，并在三个额外终点中的两个方面表现出持续改善。虽然表现出一定程度疗效，但并未达到预期，且这个领域患者很少，Editas宣布暂停药物的临床入组，并寻找共同开发产品的合作伙伴。EDIT-101是首个体内基因编辑疗法。出于对脱靶和其他潜在风险的考量，基因编辑多是从体外疗法开始，主要针对地贫这类造血干细胞疾病，或者用于修饰T细胞，开发通用型CAR-T。基因编辑向体内进军已经成为业内的一个必然趋势。曾有专家告诉研发客，向体内基因编辑进军可能呈现从遗传病向常见病转化的发展趋势，需要结合特异性的递送工具（拓展阅读地贫治疗小试牛刀，基因编辑未来应用苗头初现）。易于摘取的果实终究是有限的，EDIT-101的失利或许既是技术层面也是市场考量层面的失衡。更新颖的单碱基编辑产品，面临的问题又有所差异，他们在IND阶段就得直面挫折。2022年8月，Beam Therapeutics利用碱基编辑技术开发的CAR-T产品BEAM-201的IND申请被FDA暂停。11月，Verve Therapeutics 针对杂合子家族性高胆固醇血症的碱基编辑疗法Verve-101的IND申请被FDA暂停。对于最新的技术，安全性肯定是最重要的考量。Beam披露了FDA要求公司补充的信息，其中前两条就是涉及安全性的基因组重排和脱靶的研究和数据。Verve还没有公布FDA暂停IND申请的原因。基因治疗领域发展很快，新技术不断涌现，从病毒载体到CRISPR基因编辑，再到最新的单碱基编辑，产品获批上市的也不少，2022年上市的就包括蓝鸟治疗β-地中海贫血的Zynteglo和治疗早期活动性脑肾上腺脑白质营养不良的Skysona。临床研究永远是对技术的大考，这也是基因治疗发展途径中的关键考验。下一篇年度盘点预告：从CDE指导原则看，2022年政策关注什么？总第1796期访问研发客网站可浏览更多文章www.PharmaDJ.com

临床3期免疫疗法引进/卖出临床结果

2022-12-07

·FierceBiotech

Lysogene, looking for saving grace after trial flop, suspends stock trading and seeks partners

Lysogene's current cash runway extends only into February 2023. On the heels of Lysogene’s lead gene therapy candidate flunking a phase 2/3 study, the French biotech has suspended trading of its shares and is seeking potential partnerships and investors in an effort to stay afloat. The gene therapy company focused on central nervous system diseases has requested the opening of a safeguard proceeding, which is a legal option French companies can take in an attempt to stay alive. Safeguard proceedings allow struggling companies to be restructured at a preventive stage under supervision of the court. In anticipation of the safeguard proceedings, in which a decision is expected during the week of Dec. 12, Lysogene has requested that the trading of its shares be suspended until the court rules. The company is also pursuing discussions with potential partners and investors in an effort to strengthen its financial position and extend its cash runway, which currently only lasts into February 2023. Discussions are ongoing and the opening of a safeguard proceeding will provide time for potential partners and investors to come forward, or for the company to find potentially interested parties, according to a Dec. 6 company release. Lysogene has been short on cash since at least mid-November, when its top pipeline candidate, dubbed LYS-SAF302, failed a phase 2/3 trial. The study assessed the gene therapy in patients with mucopolysaccharidosis type IIIA (also known as Sanfilippo syndrome), a neurodegenerative disease that can spur a wide range of bodily dysfunction. Among the six youngest participants in the trial—children younger than 30 months—there was a statistically significant improvement in cognitive function compared to natural history two years after dosing, according to Lysogene. No treatment effect was observed among the remaining 12 participants, dubbed "the main cohort.” Development was measured using one of two different scales that assess neurodevelopmental and cognitive delay. The results come almost a year after Sarepta walked away from a deal that gave it ex-Europe rights to LYS-SAF302, a collaboration that cost $15 million in upfront cash and up to $125 million in biobucks. At the time, Lysogene said the termination had to do in part with “unsuccessful discussions” regarding the transferring of commercial supply of the therapy back to Lysogene. After the disappointing data drop, Lysogene began strategic discussions in the hopes of landing a new licensing deal. The only other clinical asset in Lysogene's pipeline is LYS-GM101, a treatment for the genetic neurological disorder GM1 gangliosidosis. The condition progressively destroys nerve cells in the brain and spinal cord.

引进/卖出基因疗法

100 项与 Olenasufligene relduparvovec(Lysogene SA) 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB16264

研发状态

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
黏多醣贮积症III型	临床3期	荷兰	Lysogene SA	2018-12-17
黏多醣贮积症III型	临床3期	法国	Lysogene SA	2018-12-17
黏多醣贮积症III型	临床3期	美国	Lysogene SA	2018-12-17
黏多醣贮积症III型	临床3期	英国	Lysogene SA	2018-12-17
黏多醣贮积症III型	临床3期	德国	Lysogene SA	2018-12-17

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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


NCT03612869 (AAVance, Corporate Publications) 人工标引	临床2/3期	黏多醣贮积症III型	12	LYS-SAF302 (ancillary cohort)	(衊窪鬱簾憲襯構選夢夢) = 鑰鏇膚糧艱範願齋觸繭繭積顧範艱積鹽憲選簾 (淵鏇醖鏇蓋積艱願醖鏇, 0.5 ) 更多	不佳	2022-11-18
NCT03612869 (AAVance, Corporate Publications) 人工标引	临床2/3期	黏多醣贮积症III型	12	natural history (natural history (NH))	(衊窪鬱簾憲襯構選夢夢) = 鹹選觸廠膚範窪襯觸膚繭積顧範艱積鹽憲選簾 (淵鏇醖鏇蓋積艱願醖鏇, 0.3 ) 更多	不佳	2022-11-18
NCT03612869 (AAVance, Corporate Publications) 人工标引	临床2/3期	黏多醣贮积症III型	-	LYS-SAF-302	鏇醖遞鏇選構窪鬱淵積(醖範夢窪遞築廠廠鹹簾) = 顧獵糧衊襯膚鑰鬱鏇艱夢糧選範鬱夢艱襯獵製 (衊願壓蓋齋鏇壓蓋積鑰 ) 更多	积极	2022-07-07