Clinical Trial

Disease: Sickle Cell Disease, SCD, (NCT04774536)

Disease info:

Sickle cell disease is a group of disorders that affects haemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical haemoglobin molecules called haemoglobin S, which can distort red blood cells into a sickle or crescent shape.

The production of haemoglobin A, which is the principle type of haemoglobin in humans, is governed by 3 genes: HBA1, HBA2, and HBB. Each haemoglobin A molecule consists of two alpha and two beta chains, and mutations in either of the HBA or the HBB genes may result in abnormal haemoglobin molecules with reduced or diminshed function. Sickle cell diseaase arises from a single point mutation in the 6th codon of the beta-globin gene (HBB), which results in a valine instead of a glutamic acid in the haemoglobin beta-chain.

Abnormal haemoglobin ultimately leads to anaemia as well as other symptoms, depending on the exact mutations present. Diseases caused by defective haemoglobin fall into a larger category of diseases known as the "haemoglobinopathies" which also include the thalassemias, a related group of diseases that are characterised by reduced or deficient rather than abnormal haemoglobin. 

Sickle cell disease affects approximately 100,000 individuals in the USA and more than 3 million worldwide
Official title:
Transplantation of CRISPRCas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease


Name: Mark Walters, MD

Phone: (510) 428-3374



Name: Christina Chun, MPH

Phone: (415) 502-2558



University of California, Los Angeles

University of California, Berkeley


United States, California

University of California, Los Angeles, Los Angeles, California, United States, 90095

UCSF Benioff Children's Hospital, Oakland, California, United States, 94609

Study start:
Jun. 1, 2023
9 participants
Gene editing method:
Type of edit:
Gene knockout
Hemoglobin Subunit Beta, HBB
Delivery method:
Electroporation - Ex-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active not recruiting


The study will evaluate the haematopoietic stem cell transplantation (HSCT) using CRISPR-Cas9-edited red blood cells (known as CRISPR_SCD001 Drug Product). The first six subjects will be adults. If CRISPR_SCD001 is determined to be safe in the first six subjects, the trial will continue to enroll 3 adolescents 12 - 18 years of age to evaluate the safety in younger patients.

Last updated: Dec. 28, 2023
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