23/10/2026
#香港青年科學院 曾任院士、 #香港中文大學 生命科學學院 #陳浩然 教授與中大衍生企業罕動力有限公司共同研發的多肽候選藥物，近日獲美國食品及藥物管理局（FDA）授予「孤兒藥」資格，擬用於治療罕見神經退行性疾病亨廷頓舞蹈症。該候選藥物以創新方式靶向有毒擴增CAG重複RNA，為目前仍缺乏根治方法的疾病帶來新希望。研究團隊正積極推進臨床前開發，目標於一至兩年內開展人體臨床試驗。
Professor Edwin CHAN Ho-yin, Alumni Member of the Hong Kong Young Academy of Sciences ( ) and Professor and Director of the School of Life Sciences at The Chinese University of Hong Kong ( ), co-developed a novel peptide drug candidate with CUHK spin-off Rare Power Limited that has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of Huntington’s disease. The candidate targets toxic expanded CAG-repeat RNA, offering a potential disease-modifying approach for this currently incurable rare neurodegenerative disorder. The team aims to initiate first-in-human clinical trials within one to two years.
