Sickle cell disease is a group of disorders that affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. People with this disorder have atypical hemoglobin molecules called hemoglobin S, which can distort red blood cells into a sickle, or crescent, shape.
Sickle cell disease is the most common inherited blood disorder in the United States, affecting 70,000 to 80,000 Americans. The disease is estimated to occur in 1 in 500 African Americans and 1 in 1,000 to 1,400 Hispanic Americans.
A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease
OTQ923 is a Sickle Cell Disease treatment based on genome editing of Hematopoietic Stem Cells, using CRISPR-Cas9 RNA guides identified through Intellia’s cell therapy research collaboration with Novartis.
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy
Status: Active recruiting
This study will evaluate two genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) products - OTQ923 and HIX763 - each reducing the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.
Experimental: OTQ923 or HIX763
Single intravenous infusion of either OTQ923 or HIX763, Part A - Adults treated with OTQ923; Part B - Adults treated with HIX763 Part C - Children age 2-17 - either OTQ923 or HIX763 based on review of data from Part A and/or Part B by Health agency after a formal interim analysis.