FLT-201

▎药明康德内容团队编辑 近期，全球细胞和基因疗法（CGT）领域迎来系列进展。用于治疗法布里病的在研基因疗法isaralgagene civaparvovec有望提前3年在美国获批上市。REGENXBIO公司与艾伯维（AbbVie）联合开发的ABBV-RGX-314有望成为首个治疗慢性视网膜疾病的基因疗法，在一项2期临床试验中使78%接受治疗的湿性年龄相关性黄斑变性（AMD）患者在治疗9个月后仍无需补充注射抗血管内皮生长因子（VEGF）抗体。基因编辑疗法NTLA-2002治疗遗传性血管水肿（HAE）患者的1/2期临床试验结果亮眼，单次给药使患者平均每月HAE发作率与安慰剂相比减少达81%。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。 图片来源：123RF ———✦研发进展✦——— ◇ Sangamo Therapeutics宣布已与美国FDA就其在研基因疗法isaralgagene civaparvovec（ST-920）用于治疗法布里病的加速批准监管途径达成共识。FDA同意正在进行的1/2期STAAR研究的数据可以作为该疗法获得加速批准的主要依据，并将所有患者52周的估算肾小球滤过率（eGFR）斜率作为中间临床终点。Sangamo预计，支持加速批准的完整数据集将在2025年上半年完成，并计划于2025年下半年提交生物制品许可申请（BLA）。如果获批，将会比预期的批准时间提前三年，且无需进行额外的注册性研究。Sangamo已开始为isaralgagene civaparvovec的上市申请进行准备工作。 Isaralgagene civaparvovec是一种以重组腺相关病毒2/6（AAV2/6）为载体的基因疗法，将表达α-半乳糖苷酶A的GLA基因递送到肝脏，旨在通过一次性治疗获得持久疗效，减轻法布里病的标准治疗酶替代疗法（ERT）的输注负担。接受该疗法的患者不需要预处理。在STAAR研究中，在18名接受isaralgagene civaparvovec治疗并有超过一年随访数据的男女患者中，观察到平均和中位eGFR水平的显著改善，年化eGFR斜率呈现积极趋势。不仅如此，截至目前，所有18名患者均不再使用ERT。 ◇ REGENXBIO公司公布了其与艾伯维联合开发的在研基因疗法ABBV-RGX-314在双眼湿性年龄相关性黄斑变性患者中进行的2期研究的积极数据。数据显示，在接受ABBV-RGX-314治疗9个月后，患者的治疗负担减少了97%。100%的患者只需零次或一次补充抗VEGF抗体注射，78%的患者完全无需注射。根据公司网站，ABBV-RGX-314有望成为首个治疗慢性视网膜疾病的基因疗法。 ABBV-RGX-314使用AAV8载体携带表达抗VEGF抗体片段的转基因，在体内表达的蛋白通过阻断VEGF信号传导，抑制心血生成和视网膜液体聚集。在治疗湿性AMD之外，两家公司也在探索ABBV-RGX-314治疗糖尿病视网膜病变和其他慢性视网膜疾病的潜力。 ◇ Intellia Therapeutics公司公布了其在研体内CRISPR基因编辑疗法NTLA-2002用于治疗遗传性血管水肿患者的1/2期临床试验的2期部分积极数据。结果显示，在第5至16周期间，单次输注50 mg NTLA-2002可使患者平均每月HAE发作率与安慰剂相比减少达81%。目前该疗法的全球关键性3期HAELO研究正在进行中。 NTLA-2002通过脂质纳米颗粒（LNP）以mRNA形式递送CRISPR-Cas9基因编辑系统，靶向血浆前激肽释放酶（KLKB1）基因，使该蛋白失活以永久性降低血浆中激肽释放酶活性，从而防止遗传性血管水肿的发作。根据新闻稿，NTLA-2002有望成为首个针对HAE的一次性治疗方案。 ◇ Spur Therapeutics公司公布了其用于治疗1型戈谢病的候选基因疗法FLT201的1/2期GALILEO-1试验的最新积极数据。结果显示，单次输注FLT201治疗的患者体内葡萄糖基神经酰胺（lyso-Gb1，戈谢病临床缓解的最佳预测指标之一）和骨髓负担持续降低，血细胞计数和器官容积得到改善或维持。在这项研究中接受治疗的所有患者中，FLT201继续表现出良好的安全性和耐受性。 FLT201是一种AAV基因疗法，用于治疗1型戈谢病成年患者。该疗法旨在提供一次性治疗，以持续增加患者体内GCase表达并减少有害底物的积累，阻止疾病进展，让患者不必终生接受治疗。FLT201采用Freeline的AAVS3衣壳，将新型基因引入肝细胞，生产经过理性设计的GCase变体。临床前和临床数据显示，FLT201的表达稳健而持久，并且由于酶缺乏而导致的底物毒性积累显著减少。 ◇ BridgeBio Pharma公司公布了其用于治疗卡纳万病（Canavan disease）的在研AAV9基因疗法BBP-812在CANaspire1/2期临床试验中的初步阳性数据。与该公司正在进行的CANinform自然史比较研究中观察到的疾病进程相比，接受BBP-812治疗后12个月时，患者运动功能的持续、渐进改善和运动里程碑的实现代表了重要的、具有统计学意义的变化。所有接受低剂量治疗患者的尿液、脑脊液和大脑中的N-乙酰天冬氨酸（NAA）水平显著、持续降低，接受高剂量BBP-812治疗患者尿液中的NAA水平呈进一步下降趋势。 卡纳万病是一种由编码天冬氨酸酶的ASPA基因突变引起的超罕见、可致残或致命的疾病。BBP-812能够将ASPA基因的功能拷贝递送到全身并进入大脑，从而可能根治疾病。新闻稿指出，如果获得批准，BBP-812有望成为患有卡纳万病这种先天性致命神经发育障碍的患儿的首个治疗方案。 ◇ Interius BioTherapeutics公司宣布已在一项1期临床试验中为首例B细胞恶性肿瘤患者提供其在研基因疗法INT2104。INT2104是一种慢病毒载体基因疗法，设计靶向CD7阳性的T细胞和NK细胞，并递送嵌合抗原受体（CAR）转基因，以在体内生成效应CAR-T和CAR-NK细胞。这些CAR细胞靶向CD20阳性B细胞，用于治疗B细胞恶性肿瘤。与体外CAR-T疗法不同，INT2104是一种现货型的单剂量疗法，可通过静脉输注系统性给药，患者无需进行淋巴清除，治疗时不需要任何特殊设备或人员培训。根据行业媒体STAT报道，INT2104是首个进入人体临床试验的体内CAR-T疗法。 ◇ Precision BioSciences公司宣布，其主打在研体内基因编辑疗法PBGENE-HBV的临床试验申请（CTA）已获得摩尔多瓦（Moldova）的监管单位批准。PBGENE-HBV旨在通过消除乙型肝炎病毒（HBV）的共价闭合环状DNA（cccDNA），同时灭活整合在肝细胞中的HBV DNA，以达到功能性治愈慢性乙型肝炎的目的。该公司已启动1期临床项目，并准备进行患者给药。Precision公司预计向全球监管单位提交更多的CTA和IND申请。根据新闻稿，PBGENE-HBV是进入临床阶段的首个靶向HBV的体内基因编辑疗法。 ◇ 艾尔普再生医学宣布FDA批准了其自主研发的新一代细胞疗法“人诱导多能干细胞（iPSC）来源心肌细胞注射液”（HiCM-188）用于治疗终末期心力衰竭的IND申请。新闻稿指出，HiCM-188是首个在美国接受用于治疗终末期心力衰竭临床评估的iPSC衍生细胞创新药。该候选疗法此前已在中国获准临床试验，并取得安全性及有效性结果。 ◇ 中国国家药监局药品审评中心（CDE）官网公示，贝斯昂科生物1类新药NK510细胞注射液获批临床，拟开发用于经标准治疗失败的晚期恶性实体瘤。新闻稿指出，在产品研发过程中，NK510采用了零脱靶碱基编辑器AccuBase，对NK细胞中关键基因进行了精确修改，编辑效率超过90%。研究表明，NK510既能克服癌细胞的免疫逃逸，又能特异性地识别和杀伤癌细胞，在多项体外、体内实验中展现出强大的抗肿瘤能力。 ◇ CDE官网公示，原启生物自主研发的双靶点Claudin 18.2/MSLN自体CAR-T细胞疗法OriC613注射液获批临床，拟开发治疗晚期实体瘤。根据原启生物新闻稿介绍，OriC613采用“AND”逻辑门控设计，通过拆分二代CAR结构元件，使OriC613 CAR-T细胞表面并联表达针对MSLN和Claudin 18.2的嵌合抗原受体，且仅对同时表达两个抗原的肿瘤细胞产生应答，从而能在增加肿瘤靶向杀伤活性的同时降低非肿瘤靶向毒性（OTOT），提高临床安全性。 ———✦融资、合作、M&A✦——— 图片来源：123RF ◇ AvenCell Therapeutics公司宣布完成1.12亿美元的B轮融资，此次融资所获得的资金将用于继续验证AvenCell的专有可控通用CAR-T细胞疗法平台，该平台产生的CAR-T细胞即使在患者接受治疗后也可以快速“关闭”和“打开”。与传统细胞疗法相比，该通用平台旨在更安全、更有效地治疗多种血液系统恶性肿瘤。 目前，AvenCell的临床管线包括一种高度差异化的自体CAR-T细胞疗法AVC-101，和一种通过CRISPR改造的异体CAR-T细胞疗法AVC-201，这两种管线均靶向急性髓系白血病（AML）细胞上常见的CD123抗原。这两款候选疗法已在早期临床试验中显示出令人鼓舞的安全性和疗效。此外，AvenCell有多个管线候选药物将在未来两年进入临床阶段。 ◇ 致力于开发B细胞疗法的Be Biopharma公司宣布完成8200万美元的融资，获得的资金将用于实现其用于治疗血友病B的主打项目BE-101的临床概念验证，并推进其用于治疗低磷酸酶症（HPP）的候选药物BE-102的开发。这两个项目均基于该公司强大且高效的B细胞药物（BCM）平台，该平台利用基因编辑技术改造B细胞，使其能够持续产生治疗性蛋白质，最终开发出具有持久性、可滴定且可重复给药的候选药物，无需预处理，具有成为潜在“best-in-class”基因疗法的潜力。 ◇ March Biosciences公司宣布成功完成超额认购的2840万美元A轮融资，获得资金将用于支持其主打在研自体CAR-T细胞疗法MB-105的2期临床开发，并进一步优化生产工艺。MB-105旨在治疗复发性和难治性CD5阳性T细胞淋巴瘤。MB-105专门设计用于克服与T细胞靶向相关的主要障碍，在解决T细胞同类相杀问题同时，保持对CD5阳性肿瘤细胞的高效杀伤力。MB-105在贝勒医学院（Baylor College of Medicine）进行的一项针对复发性T细胞淋巴瘤患者的1期临床试验中显示出良好的安全性和持久的缓解效果。该公司计划于2025年初开始2期临床试验。 ◇ 士泽生物宣布完成逾亿元B1轮市场化融资，至此，该公司近2年内已完成逾3亿元A轮/A+轮/B1轮融资。新闻稿指出，本轮融资将用于士泽生物临床级iPS衍生细胞药治疗神经系统疾病管线的完善、进一步开展临床研究以及推进多项注册临床试验。 该公司自研设计并建立了底层技术平台，覆盖iPSC重编程与建库、基因编辑iPSC、iPSC分化为亚型特化的神经细胞及临床前疾病动物模型的构建及评价等关键技术平台，并与多家知名研究机构及医院开展技术研究及临床开发合作。目前，士泽生物的自研临床级iPS衍生细胞药已在渐冻症和帕金森病患者中取得进展。其中，用于治疗帕金森病的iPS衍生多巴胺能神经前体细胞在多个临床案例中显示出了良好的安全性和有效性，患者症状有显著改善趋势，且未出现细胞疗法相关不良事件。 ◇ 利用人工智能（AI）推动体内基因递送的生物技术公司Dyno Therapeutics宣布与罗氏（Roche）达成第二项研究合作，开发针对神经疾病的下一代AAV载体基因疗法。Dyno和罗氏此前于2020年10月宣布了一项针对神经疾病和肝脏定向疗法的研究合作与许可协议。 根据此次新合作协议，Dyno将负责设计和发现具有改进功能特性的全新AAV衣壳。罗氏则负责进行衣壳验证研究，并推进多个神经基因疗法候选产品的临床前、临床期及商业化活动，这些产品均利用了Dyno开发的创新衣壳。罗氏将向Dyno支付5000万美元的预付款，此外在研究阶段还会进行额外付款，并有望根据临床前、临床期和销售里程碑支付总计超过10亿美元的款项。 ◇ Lyell Immunopharma公司宣布，已达成收购ImmPACT Bio公司的最终协议。ImmPACT公司的主打项目IMPT-314是一种CD19/20靶向CAR－T细胞候选产品，Lyell公司将继续开发该产品用于治疗血液恶性肿瘤，包括大B细胞淋巴瘤。新闻稿指出，收购ImmPACT公司预计将大大加强Lyell公司处于临床阶段的下一代CAR－T细胞疗法产品线，并补充其专有技术套件，这些技术旨在生成更持久的功能性T细胞，为实体瘤和血液系统恶性肿瘤患者带来更持久的疗效。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料（可上下滑动查看） [1] Sangamo Therapeutics Announces Alignment With FDA on Accelerated Approval Pathway for ST-920 in Fabry Disease With BLA Submission Expected in 2025. Retrieved October 23, 2024, from https://investor.sangamo.com/news-releases/news-release-details/sangamo-therapeutics-announces-alignment-fda-accelerated [2] REGENXBIO Presents Positive Data from the Phase II Study of Subretinal ABBV-RGX-314 in Patients with Bilateral Wet AMD at AAO 2024. Retrieved October 21, 2024, from https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-presents-positive-data-phase-ii-study-subretinal-abbv [3] 世界首款碱基编辑NK细胞产品临床试验申请获得受理. Retrieved July 30, 2024, from https://mp.weixin.qq.com/s/_jnzmCZrRSi7pbSDHlUWSA [4] Spur Therapeutics Showcases Positive New Data from Phase 1/2 GALILEO-1 Trial of FLT201, Its Novel Gene Therapy Candidate for Gaucher Disease, in Oral Presentation at ESGCT 31st Annual Congress. Retrieved October 25, 2024, from https://www.globenewswire.com/news-release/2024/10/23/2967469/0/en/Spur-Therapeutics-Showcases-Positive-New-Data-from-Phase-1-2-GALILEO-1-Trial-of-FLT201-Its-Novel-Gene-Therapy-Candidate-for-Gaucher-Disease-in-Oral-Presentation-at-ESGCT-31st-Annua.html [5] Interius BioTherapeutics Doses First Patient with in vivo Chimeric Antigen Receptor (CAR) Gene Therapy for B-cell Malignancies. Retrieved October 25, 2024, from https://interiusbio.com/2024/10/interius-biotherapeutics-doses-first-patient-with-in-vivo-chimeric-antigen-receptor-car-gene-therapy-for-b-cell-malignancies/ [6] 重大突破！艾尔普再生医学新一代细胞创新药临床IND获美国FDA批准. Retrieved October 25, 2024, from https://mp.weixin.qq.com/s/sNs5TT0emP4ns_CysiWNQw [7] 原启生物宣布双靶点MSLN/Claudin 18.2 OriC613注射液新药临床试验（IND）申请获中国NMPA批准. Retrieved October 17, 2024, from https://www.oricell.com/newsdetail/id/40.html [8] AvenCell Raises $112 Million in Series B; Funding Led by Novo Holdings. Retrieved October 22, 2024 from https://avencell.com/avencell-raises-112-million-in-series-b-funding-led-by-novo-holdings/ [9] Be Bio Announces $82 Million Financing and Transition to Clinical Stage Company. Retrieved October 22, 2024, from https://www.businesswire.com/news/home/20241022283327/en [10] March Biosciences Closes Oversubscribed $28.4 Million Series A Financing. Retrieved October 23, 2024, from https://www.globenewswire.com/news-release/2024/10/23/2967726/0/en/March-Biosciences-Closes-Oversubscribed-28-4-Million-Series-A-Financing.html [11] 士泽生物完成超亿元B1轮融资，推进临床级iPS衍生细胞药治疗神经系统疾病管线. Retrieved October 25, 2024, from https://mp.weixin.qq.com/s/R4wXpGtrxhwkVnRDbwV4ZA [12] BridgeBio Shares Positive Data from High Dose Cohort of Phase 1/2 CANaspire Study of Gene Therapy BBP-812 for Canavan Disease at ESGCT 2024. Retrieved October 25, 2024, from https://www.globenewswire.com/news-release/2024/10/24/2968601/0/en/BridgeBio-Shares-Positive-Data-from-High-Dose-Cohort-of-Phase-1-2-CANaspire-Study-of-Gene-Therapy-BBP-812-for-Canavan-Disease-at-ESGCT-2024.html [13] Precision BioSciences Receives First Approval of Clinical Trial Application to Initiate PBGENE-HBV First-In-Human Study for the Treatment of Chronic Hepatitis B. Retrieved October 24, 2024, from https://investor.precisionbiosciences.com/news-releases/news-release-details/precision-biosciences-receives-first-approval-clinical-trial [14] Intellia Presents Positive Results from the Phase 2 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary Angioedema (HAE). Retrieved October 24, 2024 from https://ir.intelliatx.com/news-releases/news-release-details/intellia-presents-positive-results-phase-2-study-ntla-2002 [15] Dyno Therapeutics Forms New Strategic Partnership With Roche To Advance AAV Gene Therapy Vectors For Neurological Diseases. Retrieved October 24, 2024, from https://www.dynotx.com/dyno-therapeutics-forms-new-strategic-partnership-with-roche-to-advance-aav-gene-therapy-vectors-for-neurological-diseases/ [16] Lyell Immunopharma to Acquire ImmPACT Bio and Prioritizes its Pipeline to Focus on Next-Generation CAR T-cell Therapies. Retrieved October 24, 2024, from https://www.globenewswire.com/news-release/2024/10/24/2969054/0/en/Lyell-Immunopharma-to-Acquire-ImmPACT-Bio-and-Prioritizes-its-Pipeline-to-Focus-on-Next-Generation-CAR-T-cell-Therapies.html a-Therapeutics-and-Factor-Bioscience-Announce-Exclusive-License-and-Collaboration-Agreement-to-Accelerate-Cell-Therapy-Development-for-Oncology-Autoimmune-and-Rare-Diseases.html [12] 因诺惟康IVB103注射液获得CDE临床许可. Retrieved October 18, 2024, from https://mp.weixin.qq.com/s/OU_bxfT9u4a_-Cs6WreoDQ 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

Data highlight potential of FLT201 to set new standard of care for Gaucher disease Dramatic and durable reductions observed in lyso-Gb1, an established biomarker of clinical response, in patients with persistently high levels despite years of prior treatment on currently approved therapies Substantial improvements in bone marrow burden demonstrate FLT201 penetrating deeper tissues that currently approved therapies poorly address Favorable safety and tolerability continue to be seen in all patients dosed On track to initiate Phase 3 trial of FLT201 in 2025 Oct. 23, 2024 -- Spur Therapeutics today announced positive new data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, an adeno-associated virus (AAV) gene therapy candidate for Gaucher disease type 1, showing durable reductions in glucosylsphingosine (lyso-Gb1), one of the best predictors of clinical response in Gaucher disease, and bone marrow burden, as well as improvements or maintenance of blood counts and organ volume in patients treated with a single infusion of FLT201. FLT201 continues to demonstrate a favorable safety and tolerability profile in all patients treated in the study. These data are being showcased in an oral presentation at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress taking place in Rome, Italy. “Gaucher disease is a debilitating chronic disorder, and despite treatment with currently approved therapies, many patients continue to have serious symptoms,” said Pamela Foulds, M.D., Spur’s Chief Medical Officer. “Consistent with what is seen in real-world practice, patients entered our trial with different unmet needs. All of them had bone disease, which current therapies poorly address. Most had high levels of harmful substrate accumulation. Some also had an enlarged spleen, low platelets, pain or fatigue. What is exciting is that after a single infusion of FLT201, patients improved in areas their disease was poorly controlled and remained well-controlled in areas their disease was controlled. These data underscore FLT201’s potential to set a new standard of care for Gaucher disease type 1.” Today’s oral presentation at ESGCT during the session titled “AAV Vectors as Tools in Gene Therapy of Rare Diseases - Recent Development to Improve Efficacy and Safety” will include updated data on safety and tolerability, as well as biomarker and efficacy endpoints from the ongoing Phase 1/2 GALILEO-1 study, a first-in-human, international, multicenter dose-finding study in adults with Gaucher disease Type 1. Dosing has been completed in the trial. A total of six patients were treated with a single infusion of FLT201 at a dose of 4.5e11 vg/kg and have been followed for between seven and 15 months after dosing. All six patients are included in the safety analysis; one patient with detectable pre-existing neutralizing antibodies (NAbs) to the AAVS3 capsid below the protocol cut-off has been excluded from the efficacy analysis. The data as of September 27, 2024 demonstrated: Favorable safety and tolerability, with no infusion reactions or dose limiting toxicities. All treatment-related adverse events were mild to moderate in nature Durable reductions, ranging from 42% to 96%, in lyso-Gb1 levels in patients who entered the trial with persistently high levels despite years of treatment with enzyme replacement therapy (ERT) or substrate reduction therapy (SRT). Low lyso-Gb1 levels maintained for more than a year as of the cut-off date after the withdrawal of prior therapy in the one patient who entered the trial with well-controlled levels. Lyso-Gb1 levels in the blood are highly correlated with substrate levels in disease-affected tissues and one of the best predictors of clinical response in Gaucher disease. Substantial improvements in bone marrow burden (BMB) seen in all five patients, including those who entered the trial with severe bone involvement. Notably, BMB is correlated with increased fractures, necrosis, bone pain and joint replacements. Improvement or maintenance of spleen and liver volume. Maintenance of hemoglobin levels, an established endpoint for Gaucher disease clinical trials, was observed beyond a year post withdrawal of treatment with ERT or SRT. Improvement or maintenance of platelet counts was also seen post withdrawal of treatment with ERT or SRT. “FLT201 exemplifies our focus on developing the next generation of gene therapies, optimizing each component of our gene therapy candidates with the aim of changing the course of disease and changing people’s lives,” said Michael Parini, Spur’s Chief Executive Officer. “Our more stable GCase85 enzyme, combined with our potent and proprietary capsid, is leading to improvements in bone, pain and fatigue, all of which are key areas of ongoing need in Gaucher disease. These data highlight FLT201’s potential to provide better outcomes than current standard of care while dramatically reducing the treatment burden for patients, and we are actively engaging with regulators to advance FLT201 into a Phase 3 trial.” Spur expects to report additional data from the Phase 1/2 GALILEO-1 trial in the first half of 2025 and initiate a Phase 3 trial for FLT201 in adults with Gaucher disease type 1 in 2025. FLT201 is an adeno-associated virus (AAV) gene therapy candidate that is currently being investigated in the Phase 1/2 GALILEO-1 clinical trial in adults with Gaucher disease. FLT201 leverages Spur’s proprietary and potent AAVS3 capsid to deliver GCase85, a rationally engineered longer-acting version of the enzyme deficient in people with Gaucher disease, with the goal of stopping disease progression, reducing or eliminating symptoms, and allowing patients to come off current lifelong treatments. Preclinical and clinical data for FLT201 have shown robust and durable expression and a substantial reduction in the toxic buildup of substrate that results from the enzyme deficiency. For more information about the GALILEO-1 trial, please visit clinicaltrials.gov (NCT05324943). Gaucher disease is caused by a mutation in the GBA1 gene that results in abnormally low levels of glucocerebrosidase (GCase), an enzyme needed to metabolize a certain type of lipid. As a result, harmful substrates glucosylceramide (Gb-1) and glucosylsphingosine (lyso-Gb1) build up in cells, which then accumulate in tissues and organs throughout the body, causing inflammation and dysfunction. Despite treatment with currently approved therapies, many people with Gaucher disease continue to experience debilitating symptoms, including enlarged organs, fatigue, bone pain and reduced lung function. Gaucher disease affects approximately 18,000 people in the United States, United Kingdom, France, Germany, Spain, Italy and Israel. Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions. By optimizing every component of its product candidates, Spur aims to unlock the true potential of gene therapy to realize outsized clinical results. Spur is advancing a breakthrough gene therapy candidate for Gaucher disease and a potential first-in-class gene therapy candidate for adrenomyeloneuropathy, as well as a research strategy to move gene therapy into more prevalent diseases, including forms of Parkinson’s, dementia, and cardiovascular disease. Expanding our impact, and advancing the practice of genetic medicine. The content above comes from the network. if any infringement, please contact us to modify.