Amit C. Nathwani

Amit C. Nathwani, M.D., Ph.D., is Professor of Haemophilia at University College London (UCL) Cancer Institute, Honorary Consultant in Haematology, and Director of the Katharine Dormandy Haemophilia Centre and Thrombosis Unit at Royal Free London NHS Trust, where he also serves as Senior NIHR Investigator. He is Founder and Chief Scientific Officer of Freeline Therapeutics and founder of NovalGen. Dr. Nathwani is internationally recognized as a global leader in clinical translation of AAV gene therapy for hemophilia B. In 1997, he moved to St. Jude Children's Research Hospital in Memphis, USA, to work with Dr. Arthur Nienhuis on adeno-associated virus-mediated gene transfer. In 2001, he returned to UCL as Senior Lecturer in Haematology and Consultant to National Blood Services, subsequently becoming Professor of Haemophilia in 2011. Nathwani led the landmark St. Jude/UCL phase 1/2 trial that provided the first clear evidence of stable, dose-dependent increase in factor IX levels in severe hemophilia B patients following single administration of AAV vectors. His 2011 New England Journal of Medicine publication demonstrated that peripheral-vein infusion of scAAV2/8-LP1-hFIXco resulted in therapeutic FIX transgene expression sufficient to improve bleeding phenotype with few side effects. The 2014 NEJM follow-up reported long-term safety and efficacy over three years, with patients achieving a median 9.7-fold reduction in bleeding rate and 12.4-fold decrease in factor IX concentrate usage. Most remarkably, Nathwani's June 2025 NEJM publication reported 13-year follow-up data—one of the longest follow-up studies in gene therapy—demonstrating durable factor IX expression, sustained clinical benefit, and no late-onset safety concerns. This work established the clinical evidence base for FDA-approved hemophilia gene therapies including Hemgenix (2022). In 2015, Nathwani co-founded Freeline Therapeutics with UCLB and Syncona LLP to commercialize his hemophilia B gene therapy research, serving as Founder, CSO, and board member. Freeline completed its NASDAQ IPO in August 2020. He subsequently founded NovalGen to further advance gene therapy commercialization. Nathwani received the 2016 European Society for Gene and Cell Therapy Outstanding Achievement Award.

Amit C. Nathwani医学博士、哲学博士，是伦敦大学学院(UCL)癌症研究所血友病教授、荣誉血液学顾问、Royal Free London NHS Trust的Katharine Dormandy血友病中心和血栓形成部门主任，同时担任高级NIHR研究员。他是Freeline Therapeutics的创始人兼首席科学官和NovalGen的创始人。 Nathwani博士国际公认为血友病B AAV基因治疗临床转化的全球领导者。1997年，他前往美国孟菲斯圣犹大儿童研究医院与Arthur Nienhuis博士合作研究腺相关病毒介导的基因转移。2001年，他回到UCL担任血液学高级讲师和国家血液服务顾问，随后于2011年成为血友病教授。 Nathwani领导了里程碑式的圣犹大/UCL I/II期试验，首次提供了严重血友病B患者单次给予AAV载体后凝血因子IX水平稳定、剂量依赖性增加的明确证据。他2011年发表在《新英格兰医学杂志》上的论文证明，外周静脉输注scAAV2/8-LP1-hFIXco导致治疗性FIX转基因表达，足以改善出血表型，副作用少。2014年NEJM后续研究报告了三年以上的长期安全性和有效性，患者出血率中位数降低9.7倍，凝血因子IX浓缩物使用减少12.4倍。 最引人注目的是，Nathwani 2025年6月的NEJM出版物报告了13年随访数据——基因治疗领域最长的随访研究之一——证明持久的凝血因子IX表达、持续的临床获益且无迟发安全性问题。这项工作为FDA批准的血友病基因治疗（包括2022年的Hemgenix）建立了临床证据基础。 2015年，Nathwani与UCLB和Syncona LLP共同创建Freeline Therapeutics，以商业化他的血友病B基因治疗研究，担任创始人、首席科学官和董事会成员。Freeline于2020年8月完成纳斯达克IPO。随后他创建了NovalGen以进一步推进基因治疗商业化。Nathwani获得了2016年欧洲基因与细胞治疗学会杰出成就奖。