In September 2018, the National Heart, Lung, and Blood Institute (NHLBI) launched the Cure Sickle Cell Initiative (CureSCi), a national initiative that supports technologies and treatments that accelerate the implementation of accessible cures for sickle cell disease (SCD).
This effort will bring together the necessary stakeholders to seize opportunities and address challenges faced by the patient community to access clinical trials and potential curative treatments. Likewise, the initiative intends to address challenges faced by the medical research community and biomedical industry to bring therapies and treatments to market. NHLBI’s initiative will play a critical role in bringing together government agencies and the private sector to improve on processes that will help accelerate the implementation of curative therapies.
The vision of CureSCi is to accelerate the development of treatments aimed at a genetic-based cure for sickle cell disease. To this end, CureSCi intends to assist investigators as they move meritorious, peer-reviewed projects through IND enabling studies and into clinical trials. CureSCi will not replace NHLBI funding of sickle cell disease-focused science through traditional mechanisms. NHLBI will continue to fund meritorious investigator-initiated grants related to sickle cell disease. CureSCi also intends to support meritorious, peer-reviewed projects intended to support the clinical evaluation of novel gene therapy and gene editing.
OTA-19-005: Preclinical and Clinical Studies for Curative Therapies for Sickle Cell Disease
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).
OTA-19-007: Analytical and/or Clinical Validation of Candidate Biomarkers for Curative Therapies in Sickle Cell Disease
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 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 deployed in clinical laboratories at the treatment sites.
The Cure Sickle Cell Initiative collaborates with The Production Assistance for Cellular Therapies (PACT) program, a NIH wide resource, to provide regulatory assistance to investigators who may require support with the following services:
- Guidance for pre-pre-IND INTERACT discussions with FDA
- INTERACT meeting package preparation
- Guidance for pre-IND discussions with FDA
- Pre-IND meeting package preparation
- Pre-clinical study design
- Chemistry, Manufacturing and Controls (CMC) development
- Initial Investigational New Drug (IND) preparation
Additionally, investigators may request support in the form of a Gap Analysis to shed light on what may be needed to support a successful IND application. The goal of this analysis is to improve understanding of how the data generated from the proof of concept, non-clinical studies, and the CMC section will map to the requirements of an IND application. The Gap Analysis may cover the following areas, as needed:
- Nonclinical studies to support the proposed clinical development plan
- Manufacturing process for both nonclinical and clinical trial materials
- Process development and scale up
- Process validation
- SOP development and batch production records
- Product cryopreservation / labeling / release / shipment
- Technology transfer
- Regulatory finalization and IND submission assistance
- Phase I clinical development plan
- FDA communications and commitments
Click on the link below if you would like to ask us about any of the regulatory services provided through the Initiative.