The Cure Sickle Cell Initiative is a collaborative, patient-focused research effort designed to accelerate promising gene therapies to cure sickle cell disease (SCD). The Initiative allows us to rethink the traditional ways that we advance research and has brought together the SCD community—including patients, advocates, caregivers, researchers, providers, industry, and others. Launched in September 2018 by the National Heart, Lung, and Blood Institute (NHLBI), the Initiative aims to transform the lives of individuals living with SCD by moving the most promising therapies safely into clinical trials.

The NHLBI-led Cure Sickle Cell Initiative has identified and supported the most promising and innovative gene therapies to cure sickle cell disease. To that end, NHLBI has engaged patients, families, advocates, and researchers from academia and the pharmaceutical industry, to leverage resources. The Initiative’s support is in addition to NHLBI’s broader sickle cell disease research investment that aims to improve the lives of people living with sickle cell disease.

The Initiative builds on the foundation of advancements in basic science consistently supported by NHLBI and others. That foundational work has brought us to this point where a focused and coordinated approach can help us find sickle cell disease cures. With the growing number of tools available to correct the genetic mutation associated with sickle cell disease, researchers now have an opportunity to develop therapies for a far broader group of people.

An analysis of the cost effectiveness of gene therapy for SCD supported by CureSCi has found that gene therapies will likely be a cost-effective option compared to conventional treatments given during a patient’s lifetime. NHLBI has partnered across the Federal government with the agencies who are tasked to consider questions of cost and access to address these aspects of curative therapies. CureSCi has worked closely with Centers for Medicare & Medicaid Service (CMS), for example, to increase the accessibility of gene therapies for Medicaid enrollees living with SCD. NHLBI is also working with CMS to identify and reduce barriers to uptake, as well as to support readiness of patients and clinicians for gene therapies.

The U.S. Food and Drug Administration’s approval of Casgevy and Lyfgenia, the first cell-based gene therapies for the treatment of sickle cell disease, has the potential to provide a life-changing and disease-modifying outcome for individuals living with sickle cell disease. Both of these therapies, which use different ways to change the expression of an individual’s genes to make more fetal hemoglobin, show the importance of providing patients with options and not relying on one single curative option. NHLBI and CureSCi continue to advance gene therapy clinical trials for sickle cell disease to ensure that they are safe, effective, and last over time.

CureSCi activities extend beyond the implementation of clinical trials, and leadership is working to address related areas that impact patients. For example, efforts are focused on safety and risks, addressing long-term follow up related to SCD gene therapies, best practices to evaluate patient readiness and resilience for gene therapy trials, mental health needs of the community, and cost effectiveness of gene therapies, among others.