The vision of the Cure Sickle Cell Initiative is to accelerate the development of treatments aimed at genetic cures for sickle cell disease.
To this end, the Initiative intends to assist investigators as they move meritorious, peer-reviewed projects through Investigational New Drug (IND) enabling studies and into clinical trials. The Initiative also intends to fund projects that support the clinical evaluation of novel genetic therapy and gene editing. The Cure Sickle Cell Initiative does not replace NHLBI funding of sickle cell disease-focused science through traditional mechanisms. NHLBI continues to fund those investigator-initiated research on sickle cell disease.
ACTIVE RESEARCH OPPORTUNITY
The NHLBI is soliciting applications for preclinical and clinical projects focused on curative strategies for sickle cell disease in the areas of gene therapy (replacement) and gene editing. Proposed projects may also be focused on developing or refining activities that improve the safety or efficacy of the clinical protocol for gene therapy or gene edited autologous hematopoietic transplantation (e.g. improved bone marrow conditioning regimens, maximizing stem cell mobilization and harvesting).
INACTIVE RESEARCH OPPORTUNITY
The NHLBI is soliciting applications to develop and validate assays necessary for the assessment of hematological outcomes and other measures of disease modification following transplantation of an autologous, gene modified (gene therapy or gene edited) hematopoietic cell graft. This graft would be conducted with the intent to provide a single treatment resulting in alleviation, or cure, of sickle cell disease. Priority is given to assays that can be validated in both manufacturing processes and in clinical trials conducted in clinical laboratories at the treatment sites.