OTQ923

BIOLOGICAL2 trials

Sponsors

Novartis Pharmaceuticals

Conditions

Sickle Cell Disease

Phase 1

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

TerminatedNCT04443907

Novartis PharmaceuticalsSickle Cell Disease

Start: 2020-08-25End: 2025-01-06Updated: 2026-01-08

Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC)

Active, not recruitingNCT06155500

Novartis PharmaceuticalsSickle Cell Disease

Start: 2024-04-16End: 2039-01-11Updated: 2026-07-28

Related Papers

Rheological Comparison of Red Cells from Individuals with SCD Treated with Gene Therapy, Haploidentical Transplantation or Hydroxyurea

Blood2024-11-052 citations

Cerebral Hemodynamics Improve in Adults with Sickle Cell Disease after Gene Therapy

Blood2024-11-05

Incremental eligibility criteria for the BMT CTN 1507 haploidentical trial for children with sickle cell disease

Blood Advances2024-10-071 citations

CRISPR-Cas9 Editing of the <i>HBG1</i> and <i>HBG2</i> Promoters to Treat Sickle Cell Disease

New England Journal of Medicine2023-08-30134 citations

Treatment of Individuals with Severe Sickle Cell Disease with OTQ923, an Autologous, <i>Ex Vivo</i>, CRISPR/Cas9-Edited, CD34+ Hematopoietic Stem and Progenitor Cell Product, Leads to Durable Engraftment and Fetal Hemoglobin Induction

Blood2022-11-155 citations

Optimization of Autologous Hematopoietic Progenitor Stem Cell Apheresis Collection from Plerixafor-Mobilized Patients with Sickle Cell Disease

Blood2021-11-05