About this study
Sickle cell disease (SCD) is a group of blood disorders that affects the hemoglobin in red blood cells. Treatments include blood transfusions and medicines to prevent or manage complications. These are usually tried first to control the symptoms of SCD. They do not cure the disease.
A stem cell (bone marrow) transplant is one of the established curative treatments for SCD. For this treatment, patients must have a suitable stem cell donor. But not everyone has a donor. At the same time, a stem cell transplant can have severe immune complications, which could be life threatening.
Two types of gene therapy treatments were approved by the FDA in 2023. Only a handful of people have received these treatments. Most have benefited from them. Several more genetic treatments are in development. It is not known yet which approaches are the best.
The SAGES1 study will test a new gene therapy treatment for SCD. Patients will not need a stem cell donor. Patients will donate their own stem cells.
In the lab, we will edit (change) the genes in the patient’s donated cells using a ‘molecular scissor’ called CRISPR/Cas9. This technique will edit the stem cells. The patient will be given chemotherapy that removes their bone marrow cells. This will create “room” for incoming genetically modified cells. We will then infuse the gene-edited cellular product into their vein. This procedure is called an autologous (self) bone marrow transplant.
We will closely monitor the patient for about 3 years after the infusion. After that, we will continue to check their progress for 12 more years in a long-term follow-up study.
Our goal is to find out if a single dose of this gene-edited drug product is safe and effective to reduce the symptoms of SCD.
Eligibility overview
- Sickle cell disease
- 18–25 years old
- Severe disease symptoms
- Willing to take part in a long-term follow-up study after the clinical trial ends