What is the goal of the Cure Sickle Cell Initiative?

The Cure Sickle Cell Initiative is an NHLBI-led collaborative research effort that is accelerating the development of gene therapies to cure sickle cell disease. The Initiative identifies and supports promising gene therapies that are currently being tested in multicenter clinical research trials. One of these studies is co-funded by the California Institute of Regenerative Medicine (CIRM). Preliminary results of these clinical trials are expected within three to five years. The Initiative aims to transform the lives of people who have sickle cell disease by creating a collaborative, patient-focused research environment. With new advancements in gene therapy, the time is right to push toward cures that can be offered to many of the approximately 100,000 Americans, and more than 20 million people worldwide, who have sickle cell disease.

Other goals of the NHLBI’s Cure Sickle Cell Initiative:

  • Create a collaborative, patient-focused research environment.
  • Engage academic researchers, private-sector researchers, and patients and their caregivers to develop strategies for cures.
  • Determine the safest, most effective, and most readily and widely adoptable gene therapies.
  • Continue to search for newly developed gene therapies, including the latest gene-editing approaches, and move them into clinical research.


  • Sickle cell disease affects about 100,000 Americans and more than 20 million people worldwide.
  • The NHLBI-led Initiative is funding clinical research trials that use gene therapy to cure sickle cell disease.
  • Preliminary results are expected within three to five years.
  • The Initiative builds on NHLBI’s broader sickle cell disease research investment that aims to improve the lives of patients.
  • People are the most important part of NHLBI research; clinical trial participation is critical to the Initiative’s success.
  • The Initiative is building partnerships, as well as supporting educational and community engagement activities.

What cures are being explored in the Cure Sickle Cell Disease Initiative?

Currently, bone marrow transplants can cure sickle cell disease, but they are most effective in children who have well-matched donors. The Cure Sickle Cell Initiative aims to develop curative strategies by initially focusing on gene therapies that modify hematopoietic stem cells (HSCs), which make red and other blood cells. These modified HSCs can then be used in gene therapy, making a cure available to more patients.

The following are possible ways to use gene therapy to modify patients’ HSCs so that their red blood cells are no longer sickle-shaped:

  • Introduce the normal hemoglobin gene into the HSCs.
  • Correct the mutation in the abnormal hemoglobin gene and transfer that corrected gene into the HSCs.
  • Modify the HSCs so they make less abnormal hemoglobin and start making other hemoglobin.

Some of these gene therapy approaches are in the early stages of testing. Two have been brought to the U.S. Food and Drug Administration (FDA) for review and approval. 

How is the Cure Sickle Cell Initiative contributing to scientific discoveries?

The Cure Sickle Cell Initiative builds on the legacy of NHLBI-supported research that has contributed to improving clinical care for people who have sickle cell disease. It also complements the Institute’s broader sickle cell disease research investment, which includes basic, clinical, translational, and implementation science research.

The following list contains some of the projects supported by the Initiative:

  • Two gene therapy clinical trials, one currently recruiting participants, and the other expected to begin recruitment in the spring of 2024.
  • Enhanced clinical trial safety monitoring to quickly address any adverse events that may occur during the clinical trials.
  • A Clonal Hematopoiesis (CH) study to determine the clinical impact of CH in individuals with sickle cell disease. Clonal hematopoiesis is when HSCs change into different subtypes of blood cells, which most often happens as people age. This study can help determine if people with sickle cell disease are at greater risk to develop CH.
  • Dedicated centers to safely manufacture cellular therapy products to be used in the ongoing clinical trials.
  • A Natural History Data Resource (NHDR) that will provide a data repository of contemporaneous controls to compare outcomes of the clinical trials.
  • Clinical and Economic Impact Analysis (CEIA) group to compare the costs of the new gene therapies with the standard of care for patients with sickle cell disease. The group published several articles on the economic impact of the disease, treatments, and post-curative quality of life.
  • The Patient Readiness and Resilience Working Group (PRRWG) to study complex psychosocial factors faced by people living with sickle cell disease who are considering gene therapy. The PRRWG published a white paper summarizing their recommendations in August of 2023.
  • The Community Input Panel (CIP), diverse in representation, is formed by people with sickle cell disease, caregivers, and people who have undergone a curative therapy. They provide a forum for continuous communication between NHLBI, researchers, and other representatives of the sickle cell community. 
  • CureSCi is a comprehensive website developed by the Initiative that includes current information and resources relevant to gene therapy, clinical trials, patient education, publications, webinars, videos, patient stories, and much more. 

How does the Cure Sickle Cell Initiative work?

People with sickle cell disease have always been at the heart of NHLBI’s research progress. They are recruited from the sickle cell community to serve on the executive committee and other subcommittees. Participation from people with the disease has been critical to the Initiative’s success.

Initiative activities have empowered the research community and accelerated promising gene therapies. Initiative activities include:

  • Research funding within academia and the private sector to identify the most promising cellular and gene therapies. 
  • Fostering collaboration between professional societies, academic institutions, patient and advocacy groups, pharmaceutical companies, federal agencies, and biotechnology companies.
  • Partnering with federal agencies, academic institutions, pharmaceutical companies, professional societies, community advocacy organizations, patient representatives, and foundations.
  • Educating and engaging the sickle cell community with the latest information and resources available from trusted sources; informing patients, caregivers, providers, and stakeholders about its work; promoting trust and collaboration; and motivating people to learn more and support clinical trials.