Anh Nguyen, MD, MBA
Dr. Anh Nguyen serves as Chief Medical Officer, leading clinical development, regulatory strategy, and medical affairs to advance innovative therapies for patients with rare diseases and complex conditions. With a strong focus on translational medicine and patient-centered drug development, Dr. Nguyen drives cross-functional collaboration to bring next-generation therapies from discovery to commercialization.
Dr. Nguyen has more than 20 years of experience spanning biopharmaceutical leadership across small molecules, biologics, RNA therapeutics, and cell and gene therapies. His experience spans early through late-stage drug development and commercial launch - including drug discovery, translation medicine, CMC, regulatory affairs, clinical development, medical affairs, pharmacovigilance, and market access. He led multiple IND submissions for first-in-class therapies, including circular RNA treatments for oncology, immuno-oncology vaccines, gene-edited solid organ xenotransplants, and allogeneic CAR-T therapies. Prior he was Vice President, Therapeutic Sector Lead at Asklepios Biopharmaceutical (AskBio), where he guided the IND clearance and early-phase clinical trials for AAV gene therapies targeting heart failure and neuromuscular rare disease.
Dr. Nguyen also held key leadership roles at Xeris Pharmaceuticals, where he served as Program Medical Lead for Gvoke®’s NDA and MAA approvals and multiple IND clearances in endocrine and rare disease indications. Earlier in his career, he served as Global Medical Director at Baxter Healthcare, where he oversaw clinical trials and regulatory submissions for drugs, devices, and biologics across immunology, nephrology, pulmonology, cardiovascular, and digital health.
Dr. Nguyen has deep expertise in federal regulatory and policy development, having served as a Medical Officer and clinical reviewer at the U.S. Food and Drug Administration (FDA) and the Centers for Medicare and Medicaid Services (CMS). At the FDA, he contributed to regulatory strategy, combination product approvals, and policy guidance for digital health and combination products. While at CMS, he conducted coverage analyses for novel therapeutics and helped shape Medicare policy. As a Robert Wood Johnson Foundation Health Policy Fellow and Senate Committee Staffer, Dr. Nguyen played a pivotal role in drafting components of the landmark "21st Century Cures Act", which strengthened U.S. biomedical innovation and became law in 2016.
Dr. Nguyen’s commitment to advancing medical science was further recognized during his tenure at the National Institutes of Health (NIH), where he led the establishment of an ambulatory surgical service to expedite pediatric orphan disease and oncology trials. His contributions in developing and executing early-stage pediatric trials in oncology, neurology, and rare metabolic disorders earned him the prestigious NIH Director’s Award.
Dr. Nguyen holds an MD from Rutgers New Jersey Medical School and an MBA with a concentration in finance from the University of Chicago Booth School of Business. He completed his residency and fellowship training in anesthesiology and cardiovascular anesthesia at Massachusetts General Hospital, Harvard Medical School.