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CRISPR Therapeutics and Vertex Announce FDA Has Lifted the Clinical Hold on the Investigational New Drug Application for CTX001 for the Treatment of Sickle Cell Disease

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Friday, October 12, 2018

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ZUG, Switzerland and BOSTON and CAMBRIDGE, Mass. - CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold and accepted the Investigational New Drug application (IND) for CTX001 for the treatment of sickle cell disease (SCD).  CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. 

In addition to the acceptance of the IND announced, CRISPR and Vertex previously announced that they had obtained approvals of Clinical Trial Applications for CTX001 in multiple countries outside the U.S. for both beta-thalassemia and SCD. The companies remain on track to initiate a Phase 1/2 clinical study in SCD by the end of 2018 and are currently enrolling patients with transfusion dependent β-thalassemia in a Phase 1/2 trial in beta-thalassemia in Europe.

About CTX001
CTX001 is an investigational ex vivo CRISPR gene-edited therapy for patients suffering from β-thalassemia or 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.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. The companies have obtained approvals of Clinical Trial Applications (CTA) in multiple countries for both β-thalassemia and sickle cell disease (SCD).

About the CRISPR-Vertex Collaboration
CRISPR and Vertex entered into a strategic research collaboration in 2015 aimed at the discovery and development of gene editing treatments using the CRISPR/Cas9 technology to correct defects in specific gene targets known to cause or contribute to particular diseases. Vertex has exclusive rights to license up to six new CRISPR/Cas9-based treatments that emerge from the collaboration, and CTX001 represents the first treatment to emerge from the joint research program. For CTX001, CRISPR and Vertex will equally share all research and development costs and profits worldwide.

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 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer AG, Vertex Pharmaceuticals and ViaCyte, Inc. 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, and business offices in London, United Kingdom. For more information, please visit www.crisprtx.com.

About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada and Australia. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for eight years in a row.

For additional information and the latest updates from the company, please visit www.vrtx.com.

Contacts:
CRISPR Investor Contact:
Susan Kim
susan.kim@crisprtx.com

CRISPR Media Contact:
Jennifer Paganelli
WCG on behalf of CRISPR
347-658-8290
jpaganelli@wcgworld.com

Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, +1 617-341-6108
or
Eric Rojas, +1 617-961-7205
or
Zach Barber, +1 617-341-6470
or

Media:
Heather Nichols, 617-341-6992
mediainfo@vrtx.com

Source: CRISPR Therapeutics
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