CRISPR Therapeutics Gene Therapy CTX001 Gets PRIME Status
CRISPR Therapeutics CRSP and its partner Vertex Pharmaceuticals VRTX announced that the European Medicines Agency (EMA) has granted Priority Medicines (“PRIME”) designation to their autologous, ex vivo CRISPR/Cas9 gene-editing therapy candidate, CTX001, for the treatment of severe sickle cell disease (“SCD”).
The decision was based on clinical data from the ongoing phase I/II study — CLIMB-121 — evaluating CTX001 in SCD patients. The EMA grants PRIME status to drugs with major therapeutic advantage over the existing treatments and target an unmet medical need or benefit patients without treatment options.
Shares of CRISPR Therapeutics were up 4.3% on Sep 22 following the announcement. The company’s shares have gained 45.8% so far this year compared with the industry’s increase of 0.5%.
Vertex and CRISPR Therapeutics are evaluating CTX001 in separate clinical studies as a gene edited treatment for SCD and transfusion-dependent beta thalassemia (“TDT”). CTX001 has been developed using CRISPR Therapeutics’ proprietary CRISPR/Cas9 technology. The companies had entered into a strategic research collaboration in 2015 to co-develop and co-commercialize CTX001. Per the agreement, they equally share all R&D costs and profits worldwide. Vertex has rights to license up to six new gene editing treatments (including CTX001), developed using the CRISPR/Cas9 technology from CRISPR that will emerge from the joint research deal.
Another phase I/II study — CLIMB-111 — is evaluating the safety and efficacy of a single dose of CTX001 in TDT patients aged 12 to 35.
We remind investors that CTX001 has previously been granted Regenerative Medicine Advanced Therapy ("RMAT"), Fast Track, and Orphan Drug designations by the FDA for both TDT and SCD. The European Commission has granted Orphan Drug Designation to the gene therapy candidate for both indications.
Other than CRISPR Therapeutics, Editas Medicine, Inc. EDIT is planning to initiate clinical development of CRISPR Cas9 based gene therapy candidate for treating SCD and TDT soon. Other companies involved in developing CRISPR Cas9 therapies include Intellia Therapeutics NTLA and Beam Therapeutics.
Apart from CTX001, CRISPR Therapeutics has other pipeline candidates in early-stage development for immuno-oncological indications. It expects to announce new data from its ongoing hemoglobinopathies and immuno-oncology programs this year.
CRISPR Therapeutics AG Price
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