CRISPR Therapeutics (NASDAQ:CRSP) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) have announced that the European Medicines Agency has granted their experimental, autologous, ex vivo CRISPR/Cas9 gene-edited therapy, CTX001 Priority Medicines designation. The therapy has been granted PRIME designation for severe sickle cell disease treatment.

CTX001 receives PRIME designation from EMA In sickle cell disease

The PRIME designation is a regulatory mechanism that offers early and proactive support to promising medicines developers to enhance development plans. Also, it accelerates the evaluation of medicines under development to reach patients as soon as possible. PRIME’s objective is to ensure patients benefit fasters from new proprietary therapies that have shown the potential of addressing a significant unmet medical need. The companies received the PRIME designation based on data from their on-going phase 1/2 study of CTX001 in treating severe sickle cell disease patients.

CTX001 is currently being investigated to treat patients with severe SCD or transfusion-dependent beta-thalassemia, whereby hematopoietic stem cells are purposed to produce high fetal hemoglobin levels in red cells. HbF is an oxygen-carrying hemoglobin form present naturally at birth, which later switches to adult hemoglobin form. HbF elevation by CTX001 can potentially alleviate transfusion requirements for TDT patients and minimize painful and devastating sickle crises in patients with SCD.

CTX001 co-developed and co-commercialized by the two companies 

So far, CTX001 has received Regenerati9ve Medicine Advanced Therapy (RMAT), Orphan Drug Designation, and Fast Track designation from the FDA. Also, CTX001 has Orphan Drug Designation from the European Commission for SCD and TDT.

CRISPR Therapeutics and Vertex are developing CTX001 under a co-development and co-commercialization agreement. So far, CTX001 is the most progressive gene editing therapy under development for SCD and TDT. The companies entered a strategic collaboration agreement in 2015 intending to use CRISPR/Cas9 to develop and discover novel therapies for the treatment of underlying genetic causes of diseases. CTX001 is the first joint treatment to emerge from the collaboration, and all development costs and profits will be shared.



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