Date: 2017-12-10
Type of
information: Presentation of results at a congress
phase: preclinical
Announcement: presentation of results at the 59th American Society of Hematology (ASH) Annual Meeting in Orlando
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, sickle cell disease
Therapeutic
area: Rare diseases - Genetic diseases - Hematological diseases
Country:
Trial
details:
Latest
news:
- • On December 10, 2017, Crispr Therapeutics announced the presentation of new data on CTX001, an investigational CRISPR gene-edited therapy for patients suffering from Beta-thalassemia and sickle cell disease. The data were discussed in an oral presentation at the American Society of Hematology (ASH) Annual Meeting. Data presented at ASH demonstrate that Crispr Therapeutics’ proprietary CRISPR gene-editing approach results in high editing efficiency, with >90% of the hematopoietic stem cells edited at the target site. A vast majority of these cells are edited on both copies of the gene, which leads to expression levels of fetal hemoglobin of 40%, well above the level believed to be sufficient to ameliorate symptoms in patients with Beta-thalassemia and sickle cell disease.
- Crispr conducted extensive genome-wide off-target assessment including detailed analyses at over 6,000 sites, which showed no off-target editing. Full toxicology analysis also demonstrated that the CRISPR gene-editing had no adverse impact on engraftment of the hematopoietic stem cells and no other safety signals.
- These results were presented in an oral session by Dr. Bill Lundberg, Chief Scientific Officer of Crispr in a session entitled CRISPR/Cas9 Genome Editing to Treat Sickle Cell Disease and ?-Thalassemia: Re-Creating Genetic Variants to Upregulate Fetal Hemoglobin Appear Well-Tolerated, Effective and Durable.
- Crispr Therapeutics has filed a Clinical Trial Application for CTX001, and is planning to start a Phase 1/2 trial in Beta-thalassemia in Europe in 2018.
Is
general: Yes
