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674 : Gene Therapy for Fanconi Anemia, Complementation Group a: Updated Results from Ongoing Global Clinical Studies of RP-L102

Researchers

Presenter

  • Agnieszka Czechowicz

Principal Investigators

  • Rajni Agarwal

  • Julin Sevilla

  • Paula Ro

  • Susana Navarro

  • Brian C Beard

  • Kenneth M Law

  • Grace Choi

  • Miriam Zeini

  • Eileen Nicoletti

  • John E Wagner

  • Claire Booth

  • Jonathan D Schwartz

  • Juan A Bueren

  • Maria Grazia Roncarolo

Medical Centers

  • Center for Definitive and Curative Medicine, Stanford University, Stanford, CA

  • Stanford Childrens Health, Palo Alto, CA

  • Hospital Infantil Universitario Nino Jesus, Madrid, Spain

  • Division of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain

  • Centro de Investigaciones Biomedicas en Red de Enfermedades Raras, Ciberer, Madrid, Spain

  • Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez. (IIS-FJD, UAM), Madrid, Spain

  • Progenics Pharmaceuticals, Inc., New York, NY

  • Division of Blood and Marrow Transplantation, Department of Pediatrics, University of Minnesota, Minneapolis, MN

  • Great Ormond Street Institute of Child Health, UCL, London, United Kingdom

Locations

  • United States

  • Spain

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Blood/haematological Diseases

Disease

  • Fanconi anemia

  • Fanconi anemia, complementation group A

Biomarkers

  • Cluster of Differentiation 34

  • Granulocyte colony-stimulating factor

Drug/Treatment

  • Filgrastim

  • Mitomycin-C

  • Plerixafor

  • mitomycin

Outcome

  • N/A

Study Design

  • N/A

Phase

  • II

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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