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Zahara & Kesha Weeks

Our Vision

Accelerate the development of 
gene therapies aimed at curing sickle cell disease.

Our Mission

The Cure Sickle Cell Initiative was created by the 
National Heart, Lung, and Blood Institute to:

  1. Use emerging safe and promising gene therapies to improve the lives of individuals with sickle cell disease.
  2. Actively engage the sickle cell disease community of patients, family members, caregivers and advocates to work together on a path to the cure.
  3. Encourage collaboration among researchers, industry, non-profit organizations, and policy-making agencies who will play a role in curing the disease.

Highlights of the
Cure Sickle Cell Initiative

  • Maintained and enhanced a SCD specific Metadata catalog, and compiled Core Data Elements to facilitate the collection of data on clinical research studies of gene therapies for SCD.

  • Funded developmental work to identify key assays to support gene therapy.

  • Developed a website with information and multiple educational resources related to gene therapies for SCD.

  • Conducted a landscape analysis and published a peer-reviewed paper on cost and cost-effectiveness of gene therapies for SCD.

  • Funded and continue to support a Phase 1/2 trial and Phase 2 trial on gene therapies for SCD

  • Established a cell bank resource for SCD investigators.

  • Established a Patient Readiness and Resilience Working Group to focus on psychosocial considerations related to readiness for gene therapies. A white paper with guidelines was published.

  • Established the Community Input Panel to integrate the patient community voice on a two-way dialogue with the research members of the Initiative.

  • Published 30 peer-reviewed journal articles to date.

  • Collaborated with national sickle cell organizations to develop and implement annual webinars and social media activities.

Collaborators

FEDERAL AGENCIES

Centers for Disease Control and Prevention (CDC)
Centers for Medicare & Medicaid Services (CMS)
Department of Health and Human Services (DHHS)
DHHS Health Resources and Services Administration (HRSA)
DHHS Office of Minority Health (OMH)
Food and Drug Administration (FDA)
National Institute of Standards and Technology (NIST)
National Institutes of Health (NIH)

FEDERAL INITIATIVES

Biologic Specimen and Data Repository (BioLINCC)
Blood and Marrow Transplant Clinical Trials Network (BMT CTN)
Center for International Blood & Marrow Transplant Research (CIBMTR)
Gene Therapy Resource Program
Production Assistance for Cellular Therapies (PACT)
Sickle Cell Disease Implementation Consortium (SCDIC)

FUNDING AWARDEES

Beckman Research Institute/City of Hope
Boston Children’s Hospital
Case Western University
Emmes
Emory University
Fred Hutchinson Cancer Research Center
Harvard University
Medical College of Wisconsin
Research Triangle Institute (RTI)
Rice University
University of California Los Angeles
University of California San Francisco
University of Washington
Vanderbilt University

MEMORANDUM OF UNDERSTANDING (MOU)

American Society of Hematology (ASH)
California Institute for Regenerative Medicine (CIRM)

LEADERSHIP

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The Cure Sickle Cell Initiative is dedicated to accelerating the development of treatments aimed at genetic-based cures.

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