Research
Opportunities

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
ANNOUNCEMENTS

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
ANNOUNCEMENTS

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.

Regulatory
Assistance

Regulatory
Assistance

The Cure Sickle Cell Initiative provides assistance with navigating the regulatory pathway for genetic therapies.

  • 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.

Contact

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View our Researcher-specific FAQs.

FAQs