RecruitingPhase 1Phase 2NCT04774536

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

Transplantation of CRISPRCas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

Sponsor

Mark Walters, MD

Enrollment

9 participants

Start Date

Aug 22, 2025

Study Type

INTERVENTIONAL

Conditions

Sickle Cell Disease

Summary

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).

Eligibility

Min Age: 12 YearsMax Age: 35 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called CRISPR_SCD001 for people with sickle cell disease. The study is currently recruiting participants at 2 locations. People eligible for this study include aged 12 Years to 35 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.