Developed jointly by Vertex Pharmaceuticals Incorporated (VRTX) and CRISPR Therapeutics AG (CRSP), CTX001 has received Priority Medicines (PRIME) status from the European Medicines Agency (EMA).
It is now clear that the European Medicines Agency will assign PRIME status to CTX001 to treat transfusion-dependent beta-thalassemia. Before this treatment receiving priority status, it was used to treat sickle cell anaemia.
Assigned PRIME status, promising drug developers receive support from the FDA on a timely basis. Finally, with the support of the regulator, therapies can enter the market quickly and reach patients. This was a Phase I/II clinical trial by CRISPR Therapeutics and Vertex that led to the designation of CTX001 for beta-thalassemia.
CRISPR / Cas9 gene-editing therapy CTX001 is an experimental therapy. Based on this study, it is thought that hematopoietic stem cells from the patient’s blood are edited to produce high haemoglobin levels and implanted back. By increasing HbF with CTX001, patients with beta-thalassemia can reduce or eliminate their need for blood transfusions, and patients with sickle cell disease can reduce painful and sometimes debilitating crises.
CTX001 is now being investigated by CRISPR Therapeutics and Vertex in patients aged 12 to 35 years. The companies intend to test CTX001 15 years after the first dose in addition to the short-term effects of the therapy.
Despite significant advancements in CRISPR Therapeutics’ therapy, investing in this company still carries a high level of risk. Additionally, if approved, the commercial potential of a company’s developments can be great, which increases interest in the company from investors.
CRISPR Therapeutics AG (CRSP) closed the last trading day at $135.54, up 1.88%. There were 1,27 million shares traded, which was lower than the average volume of 2.00 million in three months. During the trading session, the price fluctuated between $128.25 and $136.24. There were a -1.26 earnings per share ratio for the company. The shares of CRSP have gained 8.49% of their value in the past five sessions and moved 22.42% in the past month. However, they have lost -11.48% on an annual basis. Its 50-day moving average is $125.54, while its 200-day moving average is $120.46. Moreover, the stock is currently trading at an RSI of 60.55.