Grant Award Details
- The objective of this award is to complete a Phase 1 clinical trial to test the safety and efficacy of treating patients with sickle cell disease with autologous, gene-edited hematopoietic stem cells, in which the genetic defect is repaired using CRISPR/Cas9 gene editing. The trial will first test safety and efficacy in two three-patient cohorts of adult patients, then a three-patient cohort of adolescent (12-18 yrs) patients. The manufacturing of the product for each patient will be done at the UCLA GMP facility.
Grant Application Details
- Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease
Therapeutic Candidate or Device
CRISPR corrected blood stem cells are manufactured from persons with severe sickle cell disease and returned by transplant to the same person
Persons with sickle cell disease (adults and adolescents) with repeated, severe painful and lung events are eligible for the clinical trial
Blood stem cells are harvested from blood in persons with severe sickle cell disease. The cells are treated with DNA editing tools (CRISPR) to convert the sickle-producing cells to healthy blood stem cells. If this sickle correction occurs in enough of the blood stem cells, it could help or even stop complications of sickle cell disease after the CRISPR treated stem cells are returned to the bloodstream. Persons receive chemotherapy before the re-infused, corrected cells set up in the marrow.
Unmet Medical Need
Sickle Cell Disease is inherited mostly by persons in under-represented minorities. It has not received enough attention and funding. CIRM is supporting research to discover new cures for sickle cell disease. This proposal could give many more persons with sickle cell disease a chance of cure.
Complete an early phase trial in up to 9 persons.
Major Proposed Activities
- Prepare to enroll patients at 2 clinical sites (UCSF and UCLA) by completing the protocol and readying the clinical and manufacturing teams.
- Enroll 3 adults with severe sickle cell disease, staggering the study schedule for a safety assessment before each new patient is treated.
- Enroll 3 more adults; if safety and clinical benefit are shown, enroll 3 adolescents on a staggered schedule. If promising, prepare for next study.
The economic and health burdens of sickle cell disease are significant. As many as 13,000 persons in California are affected. The benefit of a curative therapy such as CRISPR_SCD001 is that it has the potential to be available in all affected persons if its safety and effectiveness is proved. This benefit will be magnified if it can be delivered safely early in life, before the chronic life-long problems of sickle cell disease have occurred.