By Clinical Informatics News Staff

December 7, 2017 | CRISPR Therapeutics today announced the submission of a Clinical Trial Application (CTA) today for CTX001 in β-thalassemia. CTX001 is an investigational CRISPR gene-edited autologous hematopoietic stem cell therapy for patients suffering from β-thalassemia and sickle cell disease. The clinical trial will begin in 2018 in Europe.

“CRISPR Therapeutics is pioneering a new class of medicines with the CTA submission for CTX001 to conduct the first company-sponsored clinical trial of a CRISPR gene-edited therapy,” said Samarth Kulkarni, Ph.D., CEO of CRISPR Therapeutics in a press release. “We are committed to translating the groundbreaking science of the CRISPR platform into therapies that can fundamentally change the lives of patients suffering from serious diseases such as β-thalassemia and sickle cell disease.”

The Phase 1/2 trial of CTX001 is designed to assess its safety and efficacy in adult transfusion-dependent β-thalassemia patients. CRISPR also plans to file an Investigational New Drug Application for CTX001 to treat sickle cell disease with the United States Food and Drug Administration in 2018.

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.  

“β-thalassemia is a devastating disease that requires serious and chronic medical intervention,” said Tony Ho, M.D., Head of Research and Development at CRISPR. “The efficient and precise editing in a patient’s own blood cells using CRISPR provides the possibility of a one-time treatment for those suffering from β-thalassemia and sickle cell disease.”

CTX001 works in patients suffering from β-thalassemia and sickle cell disease by engineering a patient’s hematopoietic stem cells 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 new HbF could alleviate transfusion requirements for β-thalassemia patients, and painful and debilitating sickle crises for sickle cell patients.