The importance of the FDA granting Fast Track Designation to Vertex’s and CRISPR’s Product CTX-001

Earlier this month the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for CTX001 for sickle cell disease (SCD).

CTX001 is in clinical trials conducted by CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX). It is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies.

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.  A drug granted Fast Track Designation may be eligible for frequent meetings and communications with the FDA and if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA).

In October 2018, the FDA had accepted the Investigational New Drug application (IND) for CTX001 for the treatment of sickle cell disease (SCD). Currently the drug is in a Phase 1/2 trial in SCD in the U.S.

Vertex and Crispr are also evaluating CTX001 for β-thalassemia, which is also enrolling in a Phase 1/2 trial and is currently open at multiple clinical trial sites in Europe.

The Product 

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

About the CRISPR-Vertex Collaboration 

CRISPR Therapeutics 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 come from the joint research program. For CTX001, CRISPR and Vertex will equally share all research and development costs and profits worldwide.

Prohost Observations

This news confirms that investors’ skepticism of the CRISPR gene editing successes was based on a mistaken interpretation of many studies conducted by researchers interested in the breakthrough gene editing. The researchers meant to caution with regard to problems that would take place in editing genes with the CRISPR editing techniques, but did not mean to condemn the use of the technology in cases where its usage is safe and effective. The cases safe usage with CRISPR are numerous and the results could solve the problems of currently untreatable diseases


For Subscribers: Please read in the article posted earlier today under Today’s Highlights about the two firms whose stocks have rallied today and the reasons why.

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