MyoSyntax Corporation — Department of Health and Human Services SBIR Phase I: DESCRIPTION (provided by applicant): Sickle cell disease is a major clinical problem in th
MyoSyntax Corporation — SBIR Phase I award from Department of Health and Human Services.
- Amount
- $225,000
- Agency
- Department of Health and Human Services · National Institutes of Health
- Program / Phase
- SBIR · Phase I
- Solicitation
- PA13-234
- NAICS
- —
- Place of performance
- MA
- Period
- —
Description
DESCRIPTION (provided by applicant): Sickle cell disease is a major clinical problem in the US, France, the United Kingdom, Brazil, and in Africa, India and the Middle East. A recent study shows that the annual cost of medical care in the US for people whosuffer from sickle cell disease exceeds 1.1 billion. Sickle hemoglobin (HbS) polymerization drives the pathophysiology of this disease. Fetal hemoglobin (HbF) is present throughout fetal development and is replaced by adult hemoglobin (HbA). A major therapeutic goal in sickle cell disease is to induce high HbF levels in sickle erythrocytes. We contend that the amount of HbF per F-cell (HbF/F-cell) and the distribution of concentrations of HbF/F-cells is a critical determinant of disease severity. Existingmeasurement of hemolysate HbF concentration or the number of F-cells in blood does not measure HbF/F-cell or the distribution of HbF/F- cell in a patient blood sample. This unmet need is critical because even patients with high HbF can have severe di