Isaralgagene civaparvovec

所有患者停止标准治疗！FDA受理创新基因疗法滚动递交上市申请 Sangamo Therapeutics今日宣布，美国FDA已同意受理该公司针对在研疗法isaralgagene civaparvovec（ST-920）滚动递交生物制品许可申请（BLA）。ST-920是Sangamo自主研发、拟用于治疗成人法布里病患者的在研基因疗法。法布里病是一种溶酶体贮积症，由于编码α-半乳糖苷酶A的基因发生突变，导致α-半乳糖苷酶A酶活性不足，而α-半乳糖苷酶A是代谢酰基鞘氨醇三己糖（Gb3）所必需的代谢酶。Isaralgagene civaparvovec使用重组AAV2/6载体，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，减轻患者接受酶替代疗法（ERT）的输注负担。 今年2月所公布的试验结果显示，治疗时间最长的患者其α-半乳糖苷酶A的活性持续升高并维持近四年（47个月），显示该疗法具有持久益处。在已达到至少一年随访的23名患者中，观察到正向的平均估算肾小球滤过率（eGFR）斜率，数值达3.061 mL/min/1.73m2/年（95% CI：0.863，5.258），显示患者的肾功能有显著改善。此外，所有18名在研究启动时接受标准治疗ERT的患者均已停止ERT的治疗，并且目前仍未恢复使用ERT。 FDA批准Keytruda又一项适应症！ 美国FDA今日批准默沙东（MSD）的PD-1靶向抗体Keytruda（pembrolizumab）静脉制剂或Keytruda Qlex（pembrolizumab与berahyaluronidase alfa）皮下制剂，联合辉瑞（Pfizer）与安斯泰来（Astellas Pharma）联合开发的Nectin-4靶向抗体偶联药物Padcev（enfortumab vedotin）用于肌层浸润性膀胱癌（MIBC）成年患者的术前新辅助治疗，并在膀胱切除术后继续给予辅助治疗。适用人群为不适合接受顺铂化疗的患者。 这次批准主要基于开放标签、随机、多中心的3期研究KEYNOTE-905/EV-303的结果，该试验纳入344例既往未接受治疗且可接受根治性膀胱切除（含盆腔淋巴结清扫）的MIBC患者。研究主要终点为无事件生存期（EFS），总生存期（OS）亦为疗效终点。 分析显示，与单纯手术相比，术前及术后序贯使用pembrolizumab联合enfortumab vedotin显著改善患者的EFS和OS。联合治疗组的中位EFS尚未达到（NR；95% CI：37.3-NR），而手术组为15.7个月（95% CI：10.3-20.5），风险比为0.40（95% CI：0.28-0.57；p＜0.0001）。总生存期方面，联合组中位OS未达到，明显优于手术组的41.7个月（95% CI：31.8-NR），风险比为0.50（95% CI：0.33-0.74；p＝0.0002）。研究中联合疗法的安全性与其此前在晚期尿路上皮癌试验中观察到的情况一致。 新锐获1.15亿美元助力推动帕金森病细胞疗法开发 Aspen Neuroscience近日宣布完成1.15亿美元C轮融资，所获资金将用于推动其帕金森病细胞疗法的进一步临床开发。本轮融资由OrbiMed、ARCH Venture Partners、Frazier Life Sciences和Revelation Partners共同领投，Medical Excellence Capital、S32、Axon Ventures、LYFE Capital、LifeForce Capital等现有投资方继续跟投，吉利德科学（Gilead Sciences）旗下Kite、Balyasny Asset Management、Cormorant Asset Management、Prebys Ventures等新投资方加入。这使Aspen迄今累计融资总额超过3.4亿美元，其中包括来自加州再生医学研究院（CIRM）的800万美元资助，进一步巩固了公司在细胞治疗领域的基础。 此次C轮融资将重点用于支持其主打细胞疗法ANPD001正在开展的临床试验，以推动这一自体细胞疗法候选药物在帕金森病领域的临床验证和未来商业化，并推进其基于自体诱导多能干细胞（iPSC）来源细胞的其他神经系统疾病治疗管线。此次融资紧随ANPD001在1/2a期试验中完成第3队列给药，以及公布第1队列术后6个月的积极安全性和初步疗效数据之后进行。 参考资料： [1] Aspen Neuroscience Announces $115 Million Series C Financing to Accelerate Personalized Cell Therapy Programs. Retrieved November 21, 2025 from https://aspenneuroscience.com/aspen-neuroscience-announces-115-million-series-c-financing-to-accelerate-personalized-cell-therapy-programs/ [2] FDA approves pembrolizumab with enfortumab vedotin-ejfv for muscle invasive bladder cancer. Retrieved November 21, 2025 from https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-pembrolizumab-enfortumab-vedotin-ejfv-muscle-invasive-bladder-cancer [3] Sangamo Therapeutics Announces FDA Acceptance of BLA Rolling Submission Request for ST-920 in Fabry Disease. Retrieved November 21, 2025 from https://www.globenewswire.com/news-release/2025/11/21/3192774/33816/en/Sangamo-Therapeutics-Announces-FDA-Acceptance-of-BLA-Rolling-Submission-Request-for-ST-920-in-Fabry-Disease.html [4] Sangamo Therapeutics Announces Updated Phase 1/2 STAAR Study Data in Fabry Disease Showing Sustained Benefit, Improvements in Kidney Function and Favorable Safety Profile. Retrieved February 6, 2025 from https://www.businesswire.com/news/home/20250206513390/en/Sangamo-Therapeutics-Announces-Updated-Phase-12-STAAR-Study-Data-in-Fabry-Disease-Showing-Sustained-Benefit-Improvements-in-Kidney-Function-and-Favorable-Safety-Profile 免责声明：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：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

Sangamo Therapeutics said it plans to file its Fabry disease gene therapy isaralgagene civaparvovec (isa-vec; ST-920) ahead of schedule after the FDA agreed to accept a rolling submission of its biologics license application (BLA).The company's filing timeline has now been moved forward to the fourth quarter of 2025, up from a previous target of early 2026. The news sent Sangamo's shares up about 40% at Friday's market open before most of those gains were pared back to about a 7% bump by mid-afternoon.Sangamo said the FDA's acceptance followed a meeting last month in which the agency reiterated its 2024 position to allow use of estimated glomerular filtration rate (eGFR) slope as an endpoint to support potential accelerated approval. Detailed findings from the pivotal Phase I/II STAAR trial presented at the International Congress of Inborn Errors of Metabolism (ICIEM) in September showed that a single dose of isa-vec was associated with positive mean annualised eGFR slope of 1.965 mL/min/1.73m2/year at 52-weeks across all 32 dosed patients, with a sustained mean slope of 1.747 mL/min/1.73m² among the 19 who had reached 104 weeks of follow-up.Various cardiac measures also remained stable for at least a year, while α-Gal A activity stayed elevated for up to 4.5 years in the longest-treated patient. Moreover, all 18 participants who had entered the STAAR study on enzyme replacement therapy (ERT) discontinued therapy and remained off it at the data cutoff.In a recent note, Jefferies analysts caught up with Sangamo management who said the FDA was focused on consistency and durability of positive eGFR slope, as well as the totality of clinical data in the open-label Phase I/II dataset, including safety and cardiac stabilisation, with "meta-analyses of approved Fabry ERTs serving as supportive evidence and not as an external comparator."The agency accepting the BLA rolling submission request comes as other developers — including uniQure, whose accelerated approval path for its Huntington's gene therapy programme appears to be in jeopardy, and Biohaven, whose spinocerebellar ataxia filing was rejected — have run into greater scrutiny over external control datasets. Jefferies analysts said those cases have "muddied the waters" more broadly from a regulatory standpoint, and while the FDA affirming the accelerated pathway using the 52-week eGFR endpoint "is encouraging…there is still uncertainty."The analysts also continued to flag Sangamo's need for a partnership to support isa-vec's eventual commercialisation. "While the strength of the Fabry gene therapy dataset and regulatory clarity de-risk the path forward, there is risk with securing [a business development] deal that will be key to resolve cash overhang (runway extended into 1Q26 vs prior 4Q25)," they said.As of Sept. 30, Sangamo held $29.6 million in cash and equivalents, down from $41.9 million at the end of 2024.