Disease: Thalassemia Genetic Diseases, Inborn Hematologic Diseases Hemoglobinopathies

Disease info:

Beta thalassemia is a blood disorder that reduces the production of hemoglobin. Hemoglobin is the iron-containing protein in red blood cells that carries oxygen to cells throughout the body.

In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body.

People with beta thalassemia are at an increased risk of developing abnormal blood clots.

The disorder affects all genders but is more prevalent in certain ethnicities and age groups. In the United States, thalassemia’s prevalence is approximately 1 in 272,000 or 1,000 people.
Official title:
A Phase 1/2 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-Thalassemia

No information


United States, California

United States, New York

United States, Tennessee

Canada, Toronto

Canada, Vancouver

Germany, Regensburg

Germany, Tuebingen

United Kingdom, London


Study start:
Sep. 14, 2018
45 participants
Gene editing method:
No information
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Active recruiting


This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in up to 12 subjects 18 to 35 years of age with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.