Anh Nguyen, M.D., M.B.A.

I am a Harvard and NIH trained biopharma executive and clinician-scientist with a 25-year proven track record of driving innovation in rare diseases, neuroscience, oncology, immunology, and diabetes. Former FDA and CMS and NIH Medical Officer. My expertise spans from regulatory submissions to clinical strategy and development, medical affairs, and commercial launches. I have led the successful approval of multiple therapeutics – including small molecules and peptides, biologics. My focus is in rare disease and oncology, using cutting-edge platforms like RNA, AAV, and Cell and Gene Therapies (CGT), and Machine Learning AI applied to drug development. Passionate about delivering transformative therapies, I excel in cross-functional leadership, risk mitigation, and executing clinical trials to improve patient outcomes.

Key Accomplishments:

Innovator in Translational Medicine: Spearheaded IND submissions and Phase 1/2 trials for groundbreaking therapies, including circular RNA (circRNA), AAV, CAR-T. Focus in oncology, immuno oncology, and rare disease neuroscience.

Directly responsible for NDA/MAA/BLA filings and drug approvals (US and EU) and commercial launch, multiple INDs and CTAs cleared, including FIH clinical studies, and first-in-class technologies.

Leadership in Global Biopharma: Directed multidisciplinary teams in end-to-end R&D for ultra-rare diseases, achieving key milestones within strict timelines.

FDA & NIH Expertise: Award-winning contributions in clinical trial innovation and regulatory strategy at FDA and NIH, in oncology and pediatric rare orphan disease.

Skills and Expertise:

Therapeutic Areas: Rare Diseases, Oncology, Neuroscience, Neuromuscular, Endocrinology, Diabetes, and Immunology.

Modalities: Adeno-associated virus (AAV), cell therapy (allogeneic and autologous CAR-T, HSCT), RNA, ligand lipid nanoparticle (LNP), bi-specific monoclonal antibody (mAb), gene editing, base editing, gene silencing, gene replacement, gene augmentation.

Strategic Leadership: Risk assessment, designed clinical-regulatory strategy and led program execution, and cross-functional team management.

Commercial Readiness: Built and operationalized pharmacovigilance systems and medical affairs strategies for global drug launches.