CRISPR Therapeutics Submits First Clinical Trial Application for a CRISPR Gene-Edited Therapy, CTX001 in β-thalassemia

-Phase 1/2 trial in β-thalassemia expected to begin in 2018-

ZUG, Switzerland and CAMBRIDGE, MA, USA I December 07, 2017 I CRISPR Therapeutics (NASDAQ:CRSP) today announced the submission of a Clinical Trial Application (CTA) 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.

“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.

“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.”

About CTX001

CTX001 is an investigational CRISPR gene-edited therapy for patients suffering from β-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 β-thalassemia patients and painful and debilitating sickle crises for sickle cell patients.

About the CRISPR-Vertex Collaboration 

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.  

 About CRISPR Therapeutics

CRISPR Therapeutics is a leading gene-editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary technology that allows for precise, directed changes to genomic DNA. The company's multi-disciplinary team of world-class researchers and drug developers is working to translate this technology into breakthrough human therapeutics in a number of serious diseases. Additionally, CRISPR Therapeutics has established strategic collaborations with Bayer AG and Vertex Pharmaceuticals to develop CRISPR-based therapeutics in diseases with high unmet need. The foundational CRISPR/Cas9 patent estate for human therapeutic use was licensed from the company's scientific founder Emmanuelle Charpentier, Ph.D. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts. For more information, please visit http://www.crisprtx.com

SOURCE: CRISPR Therapeutics

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