ATSN-101

2026年7月24日   美国讯            Atsena Therapeutics 是一家临床阶段的基因治疗公司。 今日宣布，欧洲药品管理局（EMA）已授予其两款在研基因治疗候选产品孤儿药资格，用于治疗遗传性视网膜疾病。这两款产品分别是用于治疗莱伯氏先天性黑蒙1型（LCA1）的 ATSN-101，以及用于治疗X连锁视网膜劈裂症（XLRS）的 ATSN-201。     Atsena 首席执行官 Patrick Ritschel 表示：“ATSN-101 和 ATSN-201 获得 EMA 孤儿药资格认定，凸显了这些项目能够满足目前尚无治疗选择的患者群体的巨大未满足需求。目前，ATSN-201 的关键性试验（3期）正在快速招募患者，而 ATSN-101 的全球关键性试验（3期）也计划于今年晚些时候启动。我们比以往任何时候都更接近于为美国、欧洲及其他地区的患者提供能够改变这些遗传性视网膜疾病疾病结局的基因疗法。”      EMA 授予的孤儿药资格认定，主要针对用于治疗、诊断或预防罕见病、危及生命或慢性衰弱性疾病的药物和生物制剂。获得该认定可为企业带来诸多益处，包括降低监管费用、获得临床试验方案协助、研发资助，以及在欧盟市场长达10年的独占权。     在美国，美国食品药品监督管理局（FDA）此前已授予 ATSN-101 和 ATSN-201 孤儿药、罕见儿科疾病、快速通道和再生医学先进疗法（RMAT）资格。      2026年6月，Atsena 为 LIGHTHOUSE 试验关键性3期队列的首位患者注射了 ATSN-201，目前患者招募工作进展迅速。今年晚些时候，Atsena 预计将启动一项全球性的关键性3期临床试验，评估与日本新药株式会社（Nippon Shinyaku）合作开发的 ATSN-101。 关于X连锁视网膜劈裂症（XLRS）      X连锁视网膜劈裂症（XLRS）是一种由 RS1 基因突变引起的单基因X连锁疾病。RS1 基因编码视网膜劈裂蛋白，这是一种主要由感光细胞分泌的蛋白质，定位于视杆细胞、视锥细胞和双极细胞的细胞外表面。XLRS 的特征是视网膜层异常分裂（即视网膜劈裂），导致视力下降且无法通过眼镜矫正，最终引发进行性视力丧失直至失明。该病主要影响男性，通常在幼儿时期确诊。目前北美和欧洲约有3万名男性患有 XLRS，且尚无获批的特异性治疗方法。 关于 ATSN-201       ATSN-201 是 Atsena 正在研发的一款基因疗法，它利用了 AAV.SPR——一种新型的横向扩散型衣壳。该衣壳旨在高效靶向中央视网膜的光感受器，同时避免黄斑区脱离的手术风险。 该疗法正在 LIGHTHOUSE 试验中进行评估。该试验分为 A、B、C 三个部分，共包含六个队列： ●       A部分和B部分（I/II期研究）：测试了不同的剂量浓度和注射容量，包括在儿童患者中的应用。 ●       C部分（关键性III期研究）：这是一项随机对照试验，纳入约76名6岁及以上患者，以微视野检查为主要终点，并计划进行为期52周的主要数据解读。      关键队列已于2026年6月开始给药。目前美国试验中心的招募人数超出预期，预计今年晚些时候将在欧洲和英国开设更多试验中心。更多信息请访问 ClinicalTrials.gov（标识符：NCT05878860）。      在已完成的 I/II 期研究中，ATSN-201 是首个被证实有效且安全的 X 连锁视网膜病变基因疗法。大多数患者的视网膜结构（如黄斑裂孔闭合）得到改善，微视野检查、最佳矫正视力和低照度视力评估均显示，患者的视网膜和视觉功能有显著改善。ATSN-201 安全性良好，耐受性佳，部分患者的随访数据已长达两年。      ATSN-201 已获得美国 FDA 授予的再生医学先进疗法（RMAT）、快速通道、罕见儿科疾病和孤儿药资格，以及欧洲 EMA 授予的孤儿药资格。 关于 AAV.SPR       Atsena 研发的新型衣壳 AAV.SPR 可在视网膜下注射部位以外横向扩散，从而在黄斑区以外的区域注射后，实现对中央视网膜的安全高效转导。一项在非人灵长类动物中进行的临床前研究表明，与仅限于原始注射泡边缘的现有 AAV 载体不同，AAV.SPR 能够促进转基因表达，且表达范围远超视网膜下注射泡边缘。在临床相关剂量下，AAV.SPR 无需手术脱离即可有效转导至中央凹视锥细胞，且与现有衣壳相比具有良好的安全性。欲了解更多关于该临床前研究及 AAV.SPR 作用机制的信息，请访问：https://atsenatx.com/our-approach/laterally-spreading-aav/。 关于 GUCY2D 相关的莱伯氏先天性黑蒙症（LCA1）    LCA1 是一种破坏视网膜功能的单基因眼病，由 GUCY2D 基因突变引起，会导致患者早期严重的视力障碍或失明。LCA1 是 LCA 最常见的类型之一，目前尚无获批的治疗方法。 关于 ATSN-101     ATSN-101 是一种在研基因疗法，已完成针对 LCA1 的 I/II 期临床试验。在接受高剂量治疗36个月后，患者视力表现出持久且具有临床意义的改善，且 ATSN-101 耐受性良好，未报告任何与药物相关的严重不良事件。     Atsena 预计将于今年晚些时候启动 ATSN-101 的全球关键性 III 期临床试验，试验中心计划遍布美国、欧洲和日本。ATSN-101 是 Atsena 与日本新药株式会社（Nippon Shinyaku Co., Ltd.）战略合作的成果，日本新药株式会社拥有该药物在美国和日本的独家权益；Atsena 保留该药物在世界其他地区的权益。      ATSN-101 已获得美国 FDA 的再生医学先进疗法、快速通道、儿科罕见病和孤儿药认定，以及欧洲 EMA 的孤儿药认定。 关于 Atsena Therapeutics       Atsena 是一家临床阶段的基因治疗公司，致力于开发一流的疗法，以逆转或预防遗传性视网膜疾病导致的失明。该公司的主要项目正在一项关键性临床试验中评估 ATSN-201 治疗 X 连锁视网膜劈裂症（XLRS）的疗效。XLRS 是一种通常在儿童时期确诊的遗传性疾病，会导致成年后失明。       此外，Atsena 还与日本新药株式会社（Nippon Shinyaku Co., Ltd.）开展独家战略合作，开发 ATSN-101（一种用于治疗莱伯氏先天性黑蒙1型（LCA1）的在研基因疗法）。ATSN-101 已完成一项 I/II 期临床试验并取得积极结果（https://doi.org/10.1016/s0140-6736(24)01447-8），Atsena 预计将于2026年下半年启动一项全球关键 声明：本文旨在传递遗传性视网膜疾病研究和治疗前沿信息。文章内容仅代表作者观点，并不代表RP之光爱心家园立场，亦不构成医疗指导，如有需求请咨询专业人士或前往正规医院就诊。

Orphan designation provides market exclusivity and development benefits in the European Union as both programs advance in global pivotal clinical trials DURHAM, N.C., July 23, 2026 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on using the life-changing power of genetic medicine to reverse or prevent blindness, today announced that the European Medicines Agency (EMA) has granted orphan designation to its two clinical-stage gene therapy product candidates being developed for inherited retinal diseases: ATSN-101 for Leber congenital amaurosis 1 (LCA1), and ATSN-201 for X-linked retinoschisis (XLRS). "Receiving orphan designation from the EMA for both ATSN-101 and ATSN-201 underscores that these programs address significant unmet needs for patients who currently have no treatment options," said Patrick Ritschel, Chief Executive Officer of Atsena. "With our pivotal trial for ATSN-201 enrolling rapidly and our global pivotal trial for ATSN-101 on track to begin later this year, we are closer than ever to delivering gene therapies that can change the trajectory of these inherited retinal diseases for patients in the U.S., Europe and beyond." The EMA grants orphan designation to drugs and biologics intended for the treatment, diagnosis, or prevention of rare, life-threatening, or chronically debilitating diseases or conditions. Orphan designation provides companies with certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants, and up to 10 years of market exclusivity in the EU. In the United States, the U.S. Food and Drug Administration (FDA) has previously granted Orphan Drug, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations to both ATSN-101 and ATSN-201. Atsena dosed the first patient in the pivotal Phase 3 cohort of the LIGHTHOUSE trial with ATSN-201 in June 2026 and enrollment is progressing rapidly. Later this year, Atsena expects to initiate a global, pivotal Phase 3 clinical trial evaluating ATSN-101, which is being developed in collaboration with Nippon Shinyaku Co., Ltd. About X-linked Retinoschisis (XLRS) XLRS is a monogenic X-linked disease caused by mutations in the RS1 gene, which encodes retinoschisin, a protein secreted primarily by photoreceptors. RS1 is localized to the extracellular surface of rods, cones and bipolar cells. XLRS is characterized by schisis, or abnormal splitting of retinal layers, which causes impaired visual acuity that is not correctable with glasses and leads to progressive vision loss and ultimately blindness. XLRS primarily affects males and is typically diagnosed in early childhood. Approximately 30,000 males in North America and Europe have XLRS, for which there are currently no approved, disease-specific treatments. About ATSN-201 ATSN-201 is Atsena's investigational gene therapy leveraging AAV.SPR, a novel, laterally spreading capsid designed to efficiently target photoreceptors in the central retina while avoiding the surgical risks of foveal detachment. It is being evaluated in the LIGHTHOUSE trial, which consists of three parts (A, B and C) across six cohorts. Parts A and B comprised the Phase 1/2 portion of the study, testing different dose concentrations and injection volumes, including in pediatric patients. Part C is the pivotal Phase 3 portion of the study, a randomized, controlled trial of approximately 76 patients ages 6 and older, with microperimetry as the primary endpoint and a 52-week primary readout. The pivotal cohort began dosing in June 2026 and is enrolling ahead of expectations at sites in the U.S., with additional sites in Europe and the United Kingdom (U.K.) expected to open later this year. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860). In the completed Phase 1/2 portion of the study, ATSN-201 was the first XLRS gene therapy to demonstrate evidence of efficacy and safety, with the majority of patients showing improvements in retinal structure (foveal schisis closure) and meaningful improvements in retinal and visual function as assessed by microperimetry, best-corrected visual acuity and low-luminance visual acuity. ATSN-201 has demonstrated a favorable safety profile and has been well-tolerated, with data now extending out to two years in some patients. ATSN-201 has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration and Orphan Designation from the European Medicines Agency. About AAV.SPR AAV.SPR, one of Atsena's novel capsids, spreads laterally beyond the subretinal injection site to enable safe and efficient transduction of the central retina when injected into areas outside the macula. A preclinical study in non-human primates demonstrated that AAV.SPR promotes transgene expression well beyond subretinal injection bleb margins, in contrast to benchmark AAV vectors, which remain confined to the original bleb margins. At clinically relevant doses, AAV.SPR efficiently transduces foveal cones without the need for surgical detachment and has a favorable safety profile relative to benchmark capsids. For more information about the preclinical study and how AAV.SPR works, visit https://atsenatx.com/our-approach/laterally-spreading-aav/. About GUCY2D-associated Leber Congenital Amaurosis (LCA1) LCA1 is a monogenic eye disease that disrupts retinal function. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. LCA1, one of the most common forms of LCA, has no approved treatments. About ATSN-101 ATSN-101 is an investigational gene therapy that has completed a Phase 1/2 clinical trial for LCA1. At 36 months post-treatment with the high dose, patients demonstrated durable, clinically significant improvements in vision, and ATSN-101 was well tolerated with no drug-related serious adverse events reported. Atsena expects to initiate a global pivotal Phase 3 clinical trial for ATSN-101 later this year, with sites planned across the U.S., Europe and Japan. ATSN-101 is being developed as part of a strategic collaboration with Nippon Shinyaku Co., Ltd., which holds exclusive rights in the United States and Japan; Atsena retains rights for the rest of the world. ATSN-101 has received Regenerative Medicine Advanced Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration and Orphan Designation from the European Medicines Agency. About Atsena Therapeutics Atsena is a clinical-stage gene therapy company developing best-in-class treatments for the reversal or prevention of blindness from inherited retinal diseases. The company's lead program is evaluating ATSN-201 in a pivotal clinical trial for X-linked retinoschisis (XLRS), a genetic condition typically diagnosed in childhood that leads to blindness later in life. Atsena is also developing ATSN-101, an investigational gene therapy for Leber congenital amaurosis type 1 (LCA1), as part of its exclusive strategic collaboration with Nippon Shinyaku Co., Ltd.; ATSN-101 has completed a Phase 1/2 trial with positive results (https://doi.org/10.1016/s0140-6736(24)01447-8), and Atsena expects to initiate a global pivotal Phase 3 clinical trial evaluating ATSN-101 in the second half of 2026. The company's proprietary pipeline also includes gene therapies in development for Usher syndrome type 1B and Stargardt disease. Founded by pioneers in ocular gene therapy, Atsena is led by an experienced team dedicated to addressing the needs of patients with vision loss. For more information, please visit https://atsenatx.com/. Investor and Media Contact: Argot Partnersatsena@argotpartners.com Business Contact:info@atsenatx.com