Learn more →
Back to Expert Scholars
Gene Therapy / 基因治疗AAV Vectors

R. Jude Samulski

Ph.D.

🏢AskBio / University of North Carolina at Chapel Hill(AskBio公司 / 北卡罗来纳大学教堂山分校)🌐USA

Co-founder of AskBio, Distinguished Professor Emeritus (UNC)AskBio联合创始人、杰出荣誉退休教授(UNC)

3
Key Papers
13
Awards
2
Key Contributions

👥Biography 个人简介

Richard Jude Samulski, Ph.D. (born 1954), is co-founder of AskBio (Asklepios BioPharmaceutical Inc.) and former Director of the University of North Carolina at Chapel Hill Gene Therapy Center (1993-2016), where he now serves as Distinguished Professor Emeritus. Dr. Samulski is recognized as one of the founding fathers of AAV gene therapy. As a graduate student at the University of Florida in 1982, he achieved the groundbreaking feat of cloning adeno-associated virus into a bacterial plasmid (pBR322) and rescuing intact virus from recombinant DNA—a discovery that made AAV manipulable in the laboratory and established the foundation for recombinant AAV (rAAV) vector technology. His 1982 PNAS and 1983 Cell publications are considered seminal works that enabled the widespread use of AAV as a mechanism to deliver therapeutic genes to cells. Samulski's pioneering research and discoveries have played critical roles in developing all currently FDA-approved AAV gene therapies—including Luxturna, Zolgensma, and Hemgenix—and most AAV therapies in clinical trials. He holds over 20 patents in AAV vector technology, with his innovations forming key components of modern genetic medicines. Beyond academic leadership at UNC's Gene Therapy Center, Samulski successfully translated basic research into commercial and clinical impact through multiple biotechnology ventures. In 2001, he co-founded AskBio, which was acquired by Bayer in October 2020 for $4 billion, validating the commercial value of AAV therapeutics. He also founded Bamboo Therapeutics (acquired by Pfizer, 2016), Chatham Therapeutics (acquired by Baxter International, 2014), and NanoCor Therapeutics. Recognized in 2025 as the world's most cited and co-cited expert on AAV gene therapy, Samulski has authored 208 academic publications with 17,140 scholarly citations and 3,158 patent citations. As the 2025-2026 inaugural recipient of the Edward T. Award, his career exemplifies the rare trajectory where breakthrough graduate research sets the course for decades of transformative contributions bridging academia and industry, ultimately bringing AAV gene therapies from laboratory concept to life-saving treatments for millions of patients.

Richard Jude Samulski哲学博士(生于1954年),是AskBio(Asklepios BioPharmaceutical Inc.)联合创始人和北卡罗来纳大学教堂山分校基因治疗中心前主任(1993-2016),现为杰出荣誉退休教授。 Samulski博士被公认为AAV基因治疗的奠基人之一。1982年作为佛罗里达大学研究生,他完成了将腺相关病毒克隆到细菌质粒(pBR322)并从重组DNA中拯救完整病毒的开创性壮举——这一发现使AAV在实验室中可操作,并为重组AAV (rAAV)载体技术奠定了基础。他1982年的PNAS和1983年的Cell出版物被认为是促成AAV广泛用作向细胞递送治疗基因机制的开创性工作。 Samulski的开创性研究和发现在开发所有目前FDA批准的AAV基因治疗——包括Luxturna、Zolgensma和Hemgenix——以及大多数处于临床试验阶段的AAV疗法中发挥了关键作用。他拥有20多项AAV载体技术专利,其创新构成了现代遗传药物的关键组成部分。 除了在UNC基因治疗中心的学术领导外,Samulski通过多个生物技术企业成功地将基础研究转化为商业和临床影响。2001年,他联合创建了AskBio,该公司于2020年10月被Bayer以40亿美元收购,验证了AAV治疗的商业价值。他还创建了Bamboo Therapeutics(2016年被辉瑞收购)、Chatham Therapeutics(2014年被Baxter International收购)和NanoCor Therapeutics。 2025年被认定为AAV基因治疗领域被引用和共引用最多的专家,Samulski发表了208篇学术出版物,拥有17,140次学术引用和3,158次专利引用。作为2025-2026年度Edward T. Award首届获得者,他的职业生涯体现了罕见的轨迹:突破性研究生研究为数十年的变革性贡献奠定了基础,连接学术界和工业界,最终将AAV基因治疗从实验室概念带到为数百万患者提供拯救生命的治疗。

Share:

🧪Research Fields 研究领域

AAV CloningAAV克隆
rAAV Development重组AAV开发
AAV CommercializationAAV商业化
Gene Therapy Entrepreneurship基因治疗创业

🎓Key Contributions 主要贡献

AAV Cloning and rAAV Vector Development Pioneer

One of the most important pioneers in AAV gene therapy. In 1982, as a graduate student, first successfully cloned AAV into bacterial plasmids—a breakthrough that made AAV a manipulable gene therapy tool. Samulski's early discoveries (how to clone AAV) enabled its widespread use in delivering healthy genes to cells, recognized as the foundation of AAV gene medicine. His pioneering research and discoveries in AAV biology, recombinant AAV development, and underlying AAV vector technology validated their critical roles in all currently approved AAV gene therapies and most clinical trials. Holds over 20 AAV vector patents; technology is a key component of all FDA-approved AAV gene therapy products including Luxturna, Zolgensma, and Hemgenix.

AAV Gene Therapy Commercialization and Industrialization

Not only an outstanding scientist but also a successful entrepreneur. Co-founded multiple gene therapy biotechnology companies: AskBio (founded 2001, acquired by Bayer for $4 billion in 2020, validating AAV gene therapy commercial value); Bamboo Therapeutics (acquired by Pfizer in 2016); Chatham Therapeutics (acquired by Baxter International in 2014); and NanoCor Therapeutics. As UNC Gene Therapy Center Director (1993-2016), built the center into a leading institution for AAV research and clinical translation. In 2025, recognized as the most cited and co-cited expert in AAV and gene therapy, with 208 academic publications, 3,158 patent citations, and 17,140 scholarly citations. Successfully translated basic research into clinical and commercial success, advancing AAV gene therapy from laboratory to millions of patients.

Representative Works 代表性著作

[1]

Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells

Proceedings of the National Academy of Sciences USA (1982)

As a University of Florida graduate student, first successfully cloned AAV into bacterial plasmid pBR322 and rescued intact virus from recombinant plasmid. This pioneering work in AAV gene therapy history made AAV manipulable and modifiable in the laboratory, laying the foundation for subsequent recombinant AAV (rAAV) vector development. This graduate breakthrough determined Samulski's entire career direction.

[2]

Rescue of adeno-associated virus from recombinant plasmids: gene correction within the terminal repeats of AAV

Cell (1983)

Subsequent research further refined the AAV recombinant plasmid system, demonstrating gene correction mechanisms within AAV terminal repeats (ITR). Provided key insights into AAV replication and packaging mechanisms, establishing theoretical foundations for developing rAAV vector systems. These two early papers (1982 PNAS + 1983 Cell) constitute the technological basis for modern AAV gene therapy.

[3]

Adeno-Associated Virus at 50: A Golden Anniversary of Discovery, Research, and Gene Therapy Success—A Personal Perspective

Human Gene Therapy (2015)

Samulski's 50th anniversary review of AAV discovery from a personal perspective, chronicling AAV's journey from discovery to gene therapy success. Summarizes AAV biological research progress and clinical translation milestones, providing firsthand insights into AAV gene therapy development history.

🏆Awards & Recognition 奖项与荣誉

🏆Former Director, UNC Gene Therapy Center (1993-2016)
🏆Distinguished Professor Emeritus, UNC
🏆2025-2026 Edward T. Award inaugural recipient
🏆World's most cited and co-cited expert on AAV gene therapy (2025)
🏆208 academic publications, 17,140 scholarly citations, 3,158 patent citations
🏆Over 20 AAV vector patents
🏆AskBio co-founder (acquired by Bayer for $4 billion in 2020)
🏆Bamboo Therapeutics founder (acquired by Pfizer, 2016)
🏆Chatham Therapeutics founder (acquired by Baxter, 2014)
🏆NanoCor Therapeutics co-founder
🏆First AAV cloning (1982, graduate work)
🏆rAAV technology pioneer
🏆Technological basis for all FDA-approved AAV therapies

📄Data Sources 数据来源

Last updated: 2026-03-08 | All information from publicly available academic sources

关注 R. Jude Samulski 的研究动态

Follow R. Jude Samulski's research updates

留下邮箱,当我们发布与 R. Jude Samulski(AskBio / University of North Carolina at Chapel Hill)相关的新研究或访谈时,我们会通知你。

我们不会泄露你的信息,也不会发送无关内容。随时可以退订。

Explore More Experts

Discover the researchers shaping the future of cancer treatment