华人科学家刘如谦将获早老症研究基金会科学与医学奖 其参与创办的基因编辑疗法完成首例患者给药
早老症研究基金会（Progeria Research Foundation, PRF）10月7日经 PR Newswire 宣布，将在2026年11月14日晚于 波士顿（Boston） 的 Westin Boston Seaport Hotel 举行的 Night of Wonder Gala 上，向华人科学家刘如谦（David R. Liu）颁发 S.A.M. 科学与医学奖（S.A.M. Award for Science & Medicine）。该奖以启发基金会创立的 Sam Berns 命名，表彰推进早老症科学与医学、指向治愈的研究者。
刘如谦是碱基编辑（base editing）技术的发明人，也是 PRF「早老症基因团队」（Progeria Gene Team）成员。PRF 表示，碱基编辑如同一套 DNA「拼写检查」系统，能够纠正导致 Hutchinson-Gilford 早老症的单个基因突变；他参与开发的基因疗法 SamPro-2 正在向人体试验推进。早老症是一种罕见的致死性遗传病，会使儿童迅速衰老。
「当你见到一个患早老症的孩子，你立刻明白这项工作为什么不能等。」刘如谦在基金会新闻稿中说，「一个单一的基因突变正在偷走他们的时间，而直到不久前，我们还没有办法从源头纠正它。」PRF 执行主任 Audrey Gordon 表示，刘如谦的开创性工作让基因编辑走上了治愈早老症的道路。
刘如谦现任 Broad 研究所 Richard Merkin 教授兼 Merkin 转化医疗技术研究所所长，同时任 Harvard 大学教授与 Howard Hughes 医学研究所研究员，已发表论文 300 余篇、持有 115 项以上美国专利，当选美国国家科学院与国家医学院院士，并获 2025 年生命科学突破奖与 2026 年 Harvey 奖。
与此相邻的另一项进展来自他参与创办的基因编辑公司 Prime Medicine。该公司 10 月 5 日经 GlobeNewswire 宣布，其体内 Prime Editor 疗法 PM577a 已在全球 1/2 期临床试验中完成首例患者给药，受试对象为携带 ATP7B 基因 H1069Q 突变的 Wilson 病患者。PM577a 设计为一次性静脉输注，旨在直接纠正肝细胞中的致病突变；这也是该公司首个进入人体试验的体内 Prime Editing 疗法。美国食品药品监管局 FDA 已授予 PM577 罕见儿科疾病认定，初步临床数据预计 2027 年公布。Prime Medicine 首席执行官 Allan Reine 表示，首例患者给药是向「为患者提供变革性的一次性治疗」目标迈出的一步。
Chinese American Scientist David Liu to Receive Progeria Research Foundation Science & Medicine Award; Prime Editing Therapy From Company He Co-Founded Doses First Patient
The Progeria Research Foundation (PRF) announced on October 7, via PR Newswire, that it will present its S.A.M. Award for Science & Medicine to Chinese American scientist David R. Liu at the 2026 Night of Wonder Gala, to be held on the evening of November 14 at the Westin Boston Seaport Hotel in Boston. The award, named for Sam Berns, who inspired PRF's founding, honors those who advance the science and medicine that will one day cure Progeria.
Liu is the inventor of base editing and a member of PRF's Progeria Gene Team. According to PRF, base editing functions like a "spell check" for DNA and is capable of correcting the single genetic mutation that causes Hutchinson-Gilford Progeria Syndrome; the gene therapy SamPro-2, which he helped develop, is now advancing toward human trials. Progeria is a rare and fatal genetic disease that causes rapid aging in children.
"When you meet a child with Progeria, you understand immediately why this work can't wait," Liu said in the foundation's release. "A single genetic mutation is stealing their time, and until recently we had no way to correct that at its source." PRF Executive Director Audrey Gordon said Liu's groundbreaking work has put gene editing on the path to a cure for Progeria.
Liu is the Richard Merkin Professor and director of the Merkin Institute for Transformative Technologies in Healthcare at the Broad Institute of MIT and Harvard, a professor at Harvard University, and a Howard Hughes Medical Institute investigator. He has published more than 300 papers, holds more than 115 U.S. patents, is an elected member of the National Academy of Sciences and the National Academy of Medicine, and received the 2025 Breakthrough Prize in Life Sciences and the 2026 Harvey Prize.
In a related development, Prime Medicine, a gene-editing company Liu co-founded, announced on October 5, via GlobeNewswire, that the first patient had been dosed in the global Phase 1/2 trial of PM577a, its in vivo Prime Editor therapy for Wilson disease patients carrying the H1069Q mutation in the ATP7B gene. PM577a is designed as a one-time intravenous infusion to correct the disease-causing mutation in liver cells, and the trial is the company's first clinical study of an in vivo Prime Editing therapy. The U.S. Food and Drug Administration has granted Rare Pediatric Disease designation to PM577, and initial clinical data are expected in 2027. Prime Medicine Chief Executive Officer Allan Reine said dosing the first patient was a meaningful step toward delivering a transformative, one-time treatment for patients.
发布：GSM·美国（发布链接）
图片来源：Broad研究所Merkin大楼（刘如谦任所长的Merkin Institute所在地，资料图） / The Broad Institute's Merkin Building in Cambridge, home to the Merkin Institute directed by David Liu (file photo). Photo: Kenneth C. Zirkel via Wikimedia Commons, CC BY-SA 4.0.
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