CRISPR Therapeutics announced the submission of a clinical trial application for CTX001 in β-thalassemia.
CRISPR Therapeutics (NASDAQ:CRSP) announced the submission of a clinical trial application (CTA) for CTX001 in β-thalassemia.
As quoted in the press release:
CTX001 is an investigational CRISPR gene-edited autologous hematopoietic stem cell therapy for patients suffering from β-thalassemia and sickle cell disease.
“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,” commented Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “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 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 United States Food and Drug Administration in 2018.