Medical professional with academic and industry experience in rare pediatric disorders with focused interest in early phase clinical development in orphan/rare disease space.

Expertise in bone marrow transplantation (HSCT), cellular therapies and gene therapy for malignant and non-malignant blood disorders and clinical development of programs. Expertise in lentiviral vectors, Crispr- Cas9 gene editing, stem cell transplant, T-cell therapies and regulatory/safety aspects of gene-therapies.

1. Industry experience- in cellular/gene therapy development, pharmacovigilance, regulatory science as applicable to rare diseases and design and management of clinical trials. 3 years industry experience at bluebird bio and Crispr Tx (Medical Lead of hemoglobinopathies programs).

2. Academic experience- Proficient in FACT-JACIE standards, cellular/gene therapy development and translational research. Experienced PI for design and conduct of multiple Phase I to III trials related to cellular/gene therapy using GCP guidelines, gene editing and T-cell therapies, especially for thalassemia, sickle cell disease, neuroblastoma and rare pediatric disorders.

3. Administrative- Clinical Trials and Translational Research; Consultant/Advisory experience
Medical Director, Cell and Gene Therapy Clinical Trials Office, Stanford University