Revakinagene taroretcel-lwey

来源：药渡 撰文：Pharmadeep     编辑：维他命 近日，Neurotech Pharmaceuticals宣布FDA已批准Encelto（revakinagene taroretcel），用于治疗2型黄斑毛细血管扩张症（Macular Telangiectasia Type 2，简称MacTel）。该药是首个且唯一获得FDA批准的MacTel治疗药物。这一创新疗法的获批，不仅为MacTel患者带来了新的希望，也为眼科疾病的治疗开辟了新的道路。 来源：FDA官网 1 MacTel:威胁视力的罕见眼病 2型黄斑毛细血管扩张症（MacTel）是一种罕见的神经退行性眼病，主要影响成年人的视网膜。其特征为局部视网膜变性，并伴有视网膜血管的继发性改变，导致视网膜神经细胞的渐进性退化和不可逆的视力丧失。MacTel的发病机制尚未完全明确，但研究表明，它可能与遗传、代谢紊乱以及视网膜血管异常等多种因素相关。 MacTel的病程通常进展缓慢，早期症状可能不明显，但随着病情的加重，患者会逐渐出现中央视力下降、阅读困难、色觉异常以及视野缩小等症状。由于该疾病主要影响黄斑区，即视网膜中负责中央视力和精细视觉的部分，因此对患者的日常生活质量造成了极大的影响。 在Encelto获批之前，MacTel的治疗选择极为有限。传统的治疗策略主要集中在缓解症状和减缓病情进展上，例如通过激光治疗、抗VEGF药物注射或玻璃体手术等方式来控制视网膜水肿和出血。然而，这些方法并不能从根本上阻止疾病的进展，且治疗效果因人而异。 2 Encelto:创新的细胞基因疗法 Encelto的获批，标志着MacTel治疗领域迈入了一个新的时代。Encelto是一种基于封装细胞疗法（Encapsulated Cell Therapy，ECT）的创新药物，其核心机制是通过持续向视网膜输送睫状神经营养因子（Ciliary Neurotrophic Factor，CNTF），以减缓疾病的进展。 封装细胞疗法是一种独特的细胞基因治疗技术。Encelto的治疗系统由一个小型的半透膜胶囊组成，内部含有经过基因工程改造的异体视网膜色素上皮细胞（RPE细胞），这些细胞能够稳定地分泌重组人睫状神经营养因子（rhCNTF）。当胶囊被植入眼内后，其半透膜允许营养物质进入，同时让CNTF释放到眼内，直接作用于视网膜。 CNTF是一种神经营养因子，能够支持视网膜神经细胞的存活和功能，从而对抗MacTel引起的神经退行性变化。通过持续释放CNTF，Encelto能够为视网膜提供长期的保护，减缓光感受器细胞的丢失，进而延缓视力的进一步下降。 3 临床试验数据 此次批准基于两项III期临床研究，研究旨在评估Encelto在减缓黄斑光感受器细胞丢失方面的疗效和安全性。 试验结果显示，与对照组相比，接受Encelto治疗的患者在植入后24个月内，黄斑光感受器细胞的丢失速度显著减缓。具体而言，在24个月时，对照组的椭圆体带面积增加了0.213平方毫米，而治疗眼仅增加了0.148平方毫米。MacTel病变面积的增加差异（0.065平方毫米）具有统计学显著性（p=0.030）。与基线相比，椭圆体带面积增加35%及以上的眼在治疗组中的比例显著降低（p=0.045），并且与对照组相比，治疗组的黄斑厚度显著增加（p=0.007）。次要临床终点显示，治疗眼的阅读速度得以维持，而对照组的阅读速度则有所下降（p=0.016）。 此外，Encelto总体上耐受性良好，与既往在视网膜色素变性和年龄相关性黄斑变性患者中对Encelto的研究结果一致。在研究过程中，没有受试者因任何原因移除植入物。大多数不良事件与手术操作有关，且在所有病例中，这些与手术相关的事件均在3个月内得到解决。 4 未来探索与展望 Encelto的获批不仅是MacTel治疗领域的一个里程碑，也为其他慢性眼病的治疗提供了新的思路。Neurotech Pharmaceuticals的封装细胞疗法平台具有广泛的适用性，未来有望用于治疗更多的眼科疾病，如年龄相关性黄斑变性（AMD）、视网膜色素变性（RP）以及其他神经退行性眼病。 此外，Encelto的上市也预示着细胞基因疗法在眼科领域的巨大潜力。与传统的药物治疗相比，细胞基因疗法能够提供更持久、更精准的治疗效果，为患者带来更显著的临床获益。 结 语 Encelto获FDA批准，为MacTel患者带来了新的希望。这一创新疗法通过持续输送睫状神经营养因子，显著减缓了疾病的进展，为患者保留了更多的功能性视力。随着获批Encelto将正式进入美国市场，我们期待它能够为更多MacTel患者带来光明。同时，我们也期待未来有更多的创新疗法问世，为眼科疾病的治疗带来更多突破。 参考资料： 1.Charbel Issa P, Gillies MC, Chew EY, et al. Macular Telangiectasia Type 2. Progress in Retinal and Eye Research, 2013. 2.Neurotech Pharmaceuticals, Inc. FDA Approves Encelto (revakinagene taroretcel-lwey) for the Treatment of Macular Telangiectasia Type 2 (MacTel). Business Wire, 2025. 3.Neurotech Pharmaceuticals, Inc. Highlights of Prescribing Information for Encelto. Neurotech Pharmaceuticals, 2025. 4.Neurotech Pharmaceuticals, Inc. About Encapsulated Cell Therapy (ECT). Neurotech Pharmaceuticals, 2025. *声明：本文仅是介绍医药疾病领域研究进展或简述研究概况或分享医药相关讯息，并非也不会进行治疗或诊断方案推荐，也不对相关投资构成任何建议。内容如有疏漏，欢迎沟通指出！

In macular telangiectasia type 2, small blood vessels at the center of the retina dilate and, in some cases, new blood vessels form under the retina, damaging patients' central vision and potentially leading to loss of detail vision. With a U.S. green light for its encapsulated cell therapy technology in the bag, privately held biotech Neurotech Pharmaceuticals is on the verge of making its commercial debut.The FDA last week approved (PDF) Neurotech’s cell therapy implant revakinagene taroretcel to treat the vision-robbing neurodegenerative disease macular telangiectasia (MacTel) type 2.The drug, which will adopt the brand name Encelto, is the first to win the FDA’s blessing in the rare vision disorder, Neurotech said in a release.In MacTel type 2, small blood vessels at the center of the retina dilate and, in some cases, new blood vessels form under the retina in a process known as neovascularization, according to the American Academy of Ophthalmology. Those blood vessels subsequently bleed or leak fluid, causing the light-sensitive portion of the retina, the macula, to swell or thicken, affecting patients’ central vision.MacTel type 2, which primarily occurs in middle-aged adults, can also lead to loss of detail vision. The type 2 version of the disease affects both eyes, though not always with the same severity. Encelto works by utilizing Neurotech’s proprietary encapsulated cell therapy (ECT) platform to continually deliver therapeutic doses of the protein ciliary neurotrophic factor straight to a patient’s retina, which is in turn expected to slow the progression of MacTel.Neurotech describes its ECT platform as a cell-based gene therapy delivery system designed to provide therapeutic proteins to patients with chronic eye diseases.The platform itself is made up of a small, semipermeable capsule loaded with allogeneic retinal pigment epithelium (RPE) cells that have been genetically engineered to produce therapeutic proteins.After the capsule is implanted via a surgical procedure, its semipermeable exterior allows essential nutrients to enter while therapeutic proteins can escape and travel to the retina at the back of the eye, Neurotech explained in its Thursday approval announcement.While Encelto currently stands alone against MacTel type 2, the drug boasts a clear convenience edge over other eye disorder drugs, which are often given via repeat injections or refillable delivery system implants. The FDA based its approval decision on the readouts of two late-stage trials that found Encelto significantly slowed the loss of macular photoreceptors in MacTel patients over a 24-month span, Neurotech said.In results from a pair of replicative phase 3 trials issued in late 2022, Neurotech reported that Encelto—then coded as NT-501—curbed the rate of MacTel progression by 56.4% and 29.2% compared to a simulated treatment through 24 months."This is a historic moment for the MacTel community, as Encelto becomes the first-ever FDA approved treatment for this vision-threatening disease,” Neurotech’s chief medical officer, Thomas Aaberg Jr., M.D., said in a statement. “For those who have been affected by MacTel and for all who have supported this journey, today we look forward to a future where vision loss from MacTel may be slowed."Encelto is slated to become Neurotech’s first commercial product. The company plans to launch the product in the U.S. starting in June.