Crispr Therapeutics Explodes 15% After New Blood Disease Drug Gets Fast-Track

Shares of Crispr Therapeutics were on the rise since Monday, shooting up almost 15% after the company announced that it and Vertex Pharmaceuticals have gained a key designation for a new gene-editing drug.

It was on Tuesday that the IBD Relative Strength (RS) for Crispr rose to 93. The scale runs from 1-99 with 99 being the best.

The Food & Drug Administration has granted the drug CTX001 a regenerative medicine advanced therapy designation or a RMAT.

RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including genetic therapies.

“RMAT designation is another important regulatory milestone for CTX001 and underscores the transformative potential of a CRISPR-based therapy for patients with severe hemoglobinopathies,” said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. “We expect to share additional clinical data on CTX001 in medical and scientific forums this year as we continue to work closely with global regulatory agencies to expedite the clinical development of CTX001.”

“The first clinical data announced for CTX001 late last year represented a key advancement in our efforts to bring CRISPR-based therapies to people with beta thalassemia and sickle cell disease and demonstrate the curative potential of this therapy,” said Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. “We are encouraged by these recent regulatory designations from the FDA and EMA, which speak to the potential impact this therapy could have for patients.”

CTX001 has additionally received Orphan Drug Designation from the U.S. FDA for TDT and from the European Commission for TDT and SCD. CTX001 also has Fast Track Designation from the U.S. FDA for both TDT and SCD.

Disclaimer: We have no position in any of the companies mentioned and have not been compensated for this article.