临床试验结果 - 公司公布BB-301 1b/2a期临床试验的积极中期结果 所有6名Cohort 1患者均达到正式统计学应答标准 应答率为100% [1] - 患者在接受BB-301治疗后表现出显著且持续的改善 包括吞咽困难症状负担减轻 吞咽后残留物积累减少 饮用固定体积液体所需时间缩短 以及吞咽时咽部闭合改善 [1] - 具体改善数据为：悉尼吞咽问卷得分下降328点 液体咽部最大收缩面积减少40% 所有稠度食物咽部最大收缩面积减少42% 液体总咽部残留减少45% 所有稠度食物总咽部残留减少49% 液体标准化残留比率量表得分下降69% 所有稠度食物标准化残留比率量表得分下降63% [6] 监管进展与公司计划 - 基于积极的中期数据 美国FDA已授予BB-301用于治疗伴有吞咽困难的眼咽肌营养不良症的快速通道资格 该疗法此前已获得FDA和欧洲药品管理局的孤儿药资格认定 [1][4] - 2025年第四季度 Cohort 2的首位患者已成功接受BB-301治疗 公司计划在2026年与FDA会面 以确认BB-301的关键研究设计 [4] - 公司任命曾担任Intra-Cellular Therapies Inc董事长、首席执行官兼联合创始人的Sharon Mates博士为董事会成员 [4] 产品与平台技术 - BB-301是一种基于公司专有“沉默与替换”DNA导向RNA干扰平台的新型基因疗法 其机制是沉默致病的突变型PABPN1基因表达 同时提供功能性的替代蛋白 [8][9] - 该疗法针对影响97%眼咽肌营养不良症患者的进行性吞咽困难这一严重威胁生命的并发症 [2]

-Dr. Mates served as the Chairman, Chief Executive Officer, and Co-founder of Intra-Cellular Therapies, a mental health company, from June 2002 until it was acquired by Johnson & Johnson in 2025-HAYWARD, Calif., Nov. 03, 2025 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (“ddRNAi”) platform, today a ...

Interim Clinical Study Results for Cohort 1 of the BB-301 Phase 1b/2a Treatment Study Anticipated in Q4 2025 Enrollment of the First Subject into Cohort 2 of the BB-301 Phase 1b/2a Treatment Study Expected in Q4 2025 HAYWARD, Calif., Sept. 22, 2025 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) (“Benitec” or “Company”), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary “Silence and Replace” DNA-directed RNA interference (“ddR ...

HAYWARD, Calif., May 14, 2025 (GLOBE NEWSWIRE) -- Benitec Biopharma Inc. (NASDAQ: BNTC) ("Benitec" or "Company"), a clinical-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on its proprietary "Silence and Replace" DNA-directed RNA interference ("ddRNAi") platform, today announced financial results for its third fiscal quarter ended March 31, 2025. The Company has filed its quarterly report on Form 10-Q with the U.S. Securities and Exchange Commission. "We are prof ...