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Study identifier: NCT04774536 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

Condition: Sickle Cell Disease  ·  Sponsor: Mark Walters, MD

PhasePhase 1/Phase 2
Planned participants9
Who can joinAll sexes, 12 Years to 35 Years
Healthy volunteersNo

About this study

This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).

This description comes directly from the study's public registry record.

Talk to the study team

Mark Walters, MD  ·  (510) 428-3374  ·  Mark.Walters@ucsf.edu

Christina Chun, MPH  ·  (415) 502-2558  ·  Christina.Chun@ucsf.edu

Always discuss trial participation with your own doctor first.

Locations (2)

University of California, Los AngelesLos Angeles, California, United StatesRecruiting
UCSF Benioff Children's HospitalOakland, California, United StatesRecruiting

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Source record: clinicaltrials.gov/study/NCT04774536