Date: 2017-12-07
Type of
information: Submission of a clinical trial application
phase: 1-2
Announcement: submission of a clinical trial application
Company: Crispr Therapeutics (Switzerland - UK)
Product: CTX001
Action
mechanism:
- gene therapy/cell therapy. CTX001 is an investigational CRISPR gene-edited autologous hematopoietic stem cell therapy for patients suffering from ?-thalassemia and sickle cell disease in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen carrying hemoglobin that is naturally present at birth, and is then replaced by the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion-requirements for ?-thalassemia patients and painful and debilitating sickle crises for sickle cell patients.
- CTX001 is the first CRISPR/Cas9-based treatment to advance from a research program jointly conducted by CRISPR Therapeutics and Vertex Pharmaceuticals under the companies’ collaboration aimed at the discovery and development of new gene editing treatments that use the CRISPR/Cas9 technology. Under the agreement, Vertex has exclusive rights to license up to six new CRISPR/Cas9-based treatments that emerge from the collaboration.
Disease: Beta-thalassemia
Therapeutic
area: Rare diseases - Genetic diseases - Hematological diseases
Country: USA
Trial
details:
Latest
news:
- • On December 7, 2017, Crispr Therapeutics announced the submission of a clinical trial application (CTA) for CTX001 in Beta-thalassemia. The Phase 1/2 trial of CTX001 is designed to assess its safety and efficacy in adult transfusion dependent Beta-thalassemia patients and is expected to begin in Europe in 2018. CRISPR also plans to file an Investigational New Drug Application for CTX001 to treat sickle cell disease with the FDA in 2018.
Is
general: Yes
