基本信息
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Bio
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
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
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Research Interests
20072024
Papers共 58 篇Author StatisticsCo-AuthorSimilar Experts
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All2025202120202019201820172016201520142013201220112010200820072005
CYTOTHERAPYno. 1 (2025): 78-84
Cited0Views36Bibtex
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H. Frangoul,D. Altshuler,M. D. Cappellini, Y-S Chen,J. Domm,B. K. Eustace,J. Foell,J. de la Fuente,S. Grupp,R. Handgretinger,T. W. Ho,A. Kattamis,A. Kernytsky,J. Lekstrom-Himes,A. M. Li,F. Locatelli,M. Y. Mapara,M. de Montalembert,D. Rondelli,A. Sharma,S. Sheth,S. Soni,M. H. Steinberg,D. Wall, A. Yen,S. Corbacioglu
Agnieszka Czechowicz, Maria Grazia Roncarolo,Brian C. Beard,Ken Law,Eileen Nicoletti,Paula Rio,Juan A. Bueren,Jonathan D. Schwartz,Sandeep Soni
Premanjali Lahiri,Daniel P. Dever,Annalisa Lattanzi,Narae Talbott,Jason Skowronski, Peter Llontop, Nara Vu, Thomas Cole,Fariba Fazeli,Helen Segal,Sandeep Soni,Matthew H. Porteus,Neehar Bhatia
Cited0Views26Bibtex
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26
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Author Statistics
#Papers: 58
#Citation: 1413
H-Index: 18
G-Index: 37
Sociability: 6
Diversity: 3
Activity: 6
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