Stoke Therapeutics发布了FALCON常染色体显性视神经萎缩自然史研究的两年数据Stoke Therapeutics Releases 2-Year Data from FALCON Natural History Study in Autosomal Dominant Optic Atrophy

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Stoke Therapeutics Releases 2-Year Data from FALCON Natural History Study in Autosomal Dominant Optic AtrophyStoke Therapeutics发布FALCON常染色体显性视神经萎缩自然史研究的两年数据October 21, 20252025年10月21日Stoke ...

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Patients underwent comprehensive assessments at baseline, 6, 12, 18, and 24 months.FALCON 是一项为期 24 个月、多中心、前瞻性的自然史研究，在美国、英国、意大利和丹麦的 10 个研究中心招募了 47 名年龄在 8 至 60 岁之间的参与者。患者在基线、6 个月、12 个月、18 个月和 24 个月时接受了全面评估。The study’s primary goal was to observe how ADOA-related disease parameters evolve over time to provide a clinical framework for future treatment strategies.该研究的主要目标是观察与ADOA相关的疾病参数如何随着时间的推移而演变，以为未来的治疗策略提供临床框架。Key Findings主要发现The findings confirmed that OPA1-associated ADOA progresses slowly; however, there are notable signs of functional decline:研究结果证实，OPA1相关的ADOA进展缓慢，但存在显著的功能下降迹象：• 24% of patients experienced a ≥5-letter loss in low-contrast visual acuity (LCVA) over the two-year period• 在两年期间，24%的患者在低对比度视力（LCVA）上经历了≥5个字母的损失• Increased mitochondrial dysfunction was observed in patients with ADOA compared to healthy individuals与健康个体相比，ADOA患者的线粒体功能障碍有所增加。• No significant anatomical changes in the retina were detected, suggesting that retinal dysfunction may be reversible if treated early• 视网膜未检测到显著的解剖学变化，表明如果早期治疗，视网膜功能障碍可能是可逆的。“These findings suggest that impaired function in the retina and the optic nerve occurs before permanent cell loss,” said Patrick Yu-Wai-Man, MD, PhD, Professor of Ophthalmology at the University of Cambridge and Principal Investigator of the Phase 1 OSPREY study. “By increasing levels of naturally occurring OPA1 protein to improve mitochondrial function, it may be possible to stabilize and even restore vision in people with ADOA.”.“这些研究结果表明，视网膜和视神经的功能障碍发生在细胞永久性丧失之前，”剑桥大学眼科教授、1期OSPREY研究首席研究员Patrick Yu-Wai-Man博士表示。“通过增加天然存在的OPA1蛋白水平以改善线粒体功能，可能可以稳定甚至恢复ADOA患者的视力。”About STK-002关于STK-002The FALCON results are expected to support the development of STK-002, Stoke’s proprietary antisense oligonucleotide (ASO) therapy currently being evaluated in the Phase 1 OSPREY trial.FALCON 研究结果预计将支持 STK-002 的开发，STK-002 是 Stoke 公司专有的反义寡核苷酸 (ASO) 疗法，目前正在进行 1 期 OSPREY 试验评估。STK-002 is designed to upregulate OPA1 protein expression by leveraging the wild-type (non-mutant) copy of the OPA1 gene, with the goal of maintaining or improving visual function in patients with ADOA.STK-002 旨在通过利用 OPA1 基因的野生型（非突变）拷贝来上调 OPA1 蛋白表达，以期在 ADOA 患者中维持或改善视觉功能。STK-002 has received Orphan Drug Designation from the FDA, underscoring its potential as the first disease-modifying treatment for ADOA.STK-002 已获得 FDA 的孤儿药资格认定，凸显了其作为 ADOA 首个疾病修饰治疗方案的潜力。“The FALCON study is the largest prospective natural history study to evaluate the effects of ADOA,” said Barry Ticho, MD, PhD, Chief Medical Officer of Stoke Therapeutics. “These data will provide important context as we initiate our Phase 1 study of STK-002.”“FALCON 研究是评估 ADOA 影响的最大规模前瞻性自然史研究，”Stoke Therapeutics 首席医学官 Barry Ticho 医学博士表示，“这些数据将为我们启动 STK-002 的 1 期研究提供重要的背景信息。”Understanding ADOA: A Rare Disease with High Vision Loss Burden了解ADOA：一种具有高视力丧失负担的罕见病ADOA is a rare genetic optic neuropathy caused by mutations in the OPA1 gene, leading to mitochondrial dysfunction and progressive degeneration of the optic nerve. Symptoms typically begin in the first decade of life, and up to 46% of affected individuals are registered as legally blind.ADOA是一种由OPA1基因突变引起的罕见遗传性视神经病变，会导致线粒体功能障碍和视神经的进行性退化。症状通常在生命的第一十年开始出现，高达46%的受影响个体被登记为法定盲人。","kind":"news","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"0","news_tag":"dataReport","news_rank":0,"symbols":[],"gpt_button":1,"need_auth":false,"code":"91000000","status":"200"}}}