CRISPR_SCD001 / University of California - LARVOL DELTA

Development of CRISPR_SCD001, an Autologous Hematopoietic Stem Cell Gene Therapy for Sickle Cell Disease after CRISPR-Cas9 Mediated Correction. (PubMed, Mol Ther) - "The Drug Product (DP) from Plerixafor-mobilized peripheral-blood CD34+ cells from sickle and healthy donors were used for: 1) pharmacodynamic assessments to interrogate therapeutic potential; 2) evaluation of genotoxicity; and 3) in vivo characterization by a non-GLP toxicology study in NBSGW mice. Our protocol generates a clinical-grade, cryopreserved CD34+ cell product that corrects the sickle mutation, restores anti-sickling hemoglobins, and meets pre-clinical safety criteria, establishing a reproducible manufacturing process suitable for clinical translation. These data support initiation of a first-in-human phase I trial of CRISPR_SCD001 for sickle patients presenting with severe pathology."

First-in-human • Journal • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • CD34

Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease (clinicaltrials.gov) - P1/2 | N=9 | Recruiting | Sponsor: Mark Walters, MD | Trial completion date: Mar 2029 ➔ Mar 2030 | Trial primary completion date: Mar 2027 ➔ Mar 2030

Trial completion date • Trial primary completion date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Transplantation • HP

Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease (clinicaltrials.gov) - P1/2 | N=9 | Not yet recruiting | Sponsor: Mark Walters, MD | Trial completion date: Jun 2028 ➔ Mar 2029 | Trial primary completion date: Jun 2025 ➔ Mar 2027 | Not yet recruiting ➔ Recruiting

Enrollment open • Trial completion date • Trial primary completion date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Transplantation

Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease (clinicaltrials.gov) - P1/2 | N=9 | Not yet recruiting | Sponsor: Mark Walters, MD | Trial completion date: Dec 2027 ➔ Jun 2028 | Trial primary completion date: Dec 2024 ➔ Jun 2025

Trial completion date • Trial primary completion date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Transplantation • HP

Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease (clinicaltrials.gov) - P1/2 | N=9 | Not yet recruiting | Sponsor: Mark Walters, MD | Trial completion date: Dec 2026 ➔ Dec 2027 | Trial primary completion date: Dec 2023 ➔ Dec 2024

Trial completion date • Trial primary completion date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease • Transplantation • HP