Gene Therapy Research Seeks to Cure Sickle Cell Disease

Promising new research is underway to develop a safe, affordable gene-based cure for sickle cell disease (SCD), a major cause of death and disability in many developing countries. About 1,000 children are born with SCD each day in sub-Saharan Africa, and about half of those will die before the age of five.

Existing treatments for SCD, which is caused by a single, inherited genetic mutation, are expensive and resource intensive. The new research, funded by the Gates Foundation and in collaboration with Novartis Institutes for BioMedical Research (NIBR), seeks to develop a single-dose shot to treat the disease, making it more accessible to the people who need it most.

Initially, the NIBR research team will focus on discovering and assessing new gene therapy candidates, to be followed by clinical trials, if successful. The scientists believe the advances will also lead to new gene-based treatments for HIV, and have committed to ensuring any resulting cures are made available to low- and middle-income countries.