UCLA Advances CRISPR Sickle Cell Therapy to Manufacturing

Researchers have established a reproducible process for producing an experimental gene-edited stem cell therapy for sickle cell disease.

Updated on Nov. 5, 2026 in Biotech

Clear culture flasks of pale cell suspension on a steel laboratory bench beside an electroporation rack in a clean biomanufacturing facility. — UCLA researchers have established a reproducible manufacturing process for an experimental CRISPR gene-edited stem cell therapy for sickle cell disease, now used in an ongoing phase 1 trial. AI Illustration. Upload story photo >

A UCLA-led team has advanced an experimental CRISPR-Cas9 therapy that corrects the sickle cell mutation in a patient's own blood-forming stem cells into clinical-scale manufacturing. The process has been implemented at UCLA to produce cell products for two trial participants in an ongoing phase 1 study.

Why it matters

Sickle cell disease disproportionately affects Black Americans, and a one-time corrective therapy could address its underlying genetic cause rather than only managing symptoms. Establishing reproducible manufacturing is a necessary step before any gene-editing therapy can reach broader patient access.

Mouse studies showed a stable proportion of corrected gene copies over 16 weeks and reduced sickle hemoglobin in red blood cells derived from edited stem cells, while all four full-scale manufacturing runs met quality requirements. Long-term persistence and safety in human patients have not yet been established.

The players

UCLA

The University of California, Los Angeles is a public research university where the therapy was developed and its manufacturing process was established.

UCSF Benioff Children's Hospital Oakland

A pediatric hospital in Oakland, California, and the site of the ongoing phase 1 clinical trial of the therapy.

Dr. Mark Walters

A clinician at UCSF Benioff Children's Hospital Oakland who leads the phase 1 clinical trial of the CRISPR sickle cell therapy.

The details

Stem cells are maintained in culture for two days, then exposed to electrical pulses that temporarily open pores so CRISPR-Cas9 and a nonviral DNA template can enter and correct the single-point mutation. The therapy is being evaluated in a phase 1 trial led by Dr. Mark Walters at UCSF Benioff Children's Hospital Oakland, and no noteworthy treatment-related toxicity or abnormal tissue changes were found in mouse studies.

Stem cells are cultured for two days before electrical-pulse editing.
Mouse engraftment data covered a 16-week period.
Research describing the advance was reported on November 4, 2026.

Big Picture

The advance extends the CRISPR-Cas9 platform for editing a patient's own blood-forming stem cells from laboratory work into clinical-scale production for sickle cell disease. It positions gene-edited cell therapy as a potential template for other inherited blood disorders that may later be treated the same way.

For people living with sickle cell disease, a successful one-time therapy would replace lifelong symptom management with a potential cure, though access and cost remain undetermined. Patients and families should expect the therapy to remain investigational until phase 1 results and later-stage trials are reported.

The takeaway

Establishing a reproducible manufacturing process is a critical milestone that moves gene-edited stem cell therapy closer to clinical use. Whether the corrected cells persist and provide lasting benefit in patients will determine if this becomes a durable treatment.