HIX763 / Novartis - LARVOL DELTA

CRISPR/Cas9 Genome-Edited Autologous Hematopoietic Stem Cell Transplantation Therapies for Sickle Cell Disease (ASH 2022) - "We have developed 2 genome editing strategies targeting BCL11A for the induction of HbF levels and report the preclinical development of two potentially curative CRISPR/Cas9 ex-vivo genome-edited autologous hematopoietic stem cell therapies: HIX763 and OTQ923. Finally, a comparability assessment of OTQ923 produced using the research process and the clinical manufacturing process showed comparability in the quality of cells, as measured by off-target editing, genomic rearrangement, cell viability, editing efficiency, HbF induction, and in vivo engraftment. Together these studies demonstrate that the preclinical data adequately reflects the behavior of the cells intended for clinical use and supports advancing these drug products into clinical trials for the treatment of sickle cell disease."

Bone Marrow Transplantation • Genetic Disorders • Hematological Disorders • Oncology • Sickle Cell Disease • Transplantation • BCL11A

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) (clinicaltrials.gov) - P1/2 | N=30 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial primary completion date: Dec 2023 ➔ Aug 2025

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

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) (clinicaltrials.gov) - P1/2 | N=30 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial completion date: Dec 2024 ➔ Aug 2025

Trial completion date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) (clinicaltrials.gov) - P1/2; N=30; Recruiting; Sponsor: Novartis Pharmaceuticals; Trial completion date: Dec 2023 ➔ Dec 2024

Clinical • Trial completion date • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) (clinicaltrials.gov) - P1/2; N=30; Recruiting; Sponsor: Novartis Pharmaceuticals; N=20 ➔ 30

Clinical • Enrollment change • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) (clinicaltrials.gov) - P1/2; N=20; Recruiting; Sponsor: Novartis Pharmaceuticals; Not yet recruiting ➔ Recruiting; N=30 ➔ 20

Clinical • Enrollment change • Enrollment open • Genetic Disorders • Hematological Disorders • Sickle Cell Disease

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD) (clinicaltrials.gov) - P1/2; N=30; Not yet recruiting; Sponsor: Novartis Pharmaceuticals

Clinical • New P1/2 trial • Gene Therapies • Genetic Disorders • Hematological Disorders • Sickle Cell Disease