- The company recently submitted a CTA for CTX-001 (the potential treatment for the blood disorder Beta-thalassemia). It’s the first application for the CRISPR/Cas9 gene-editing platform.
- CRISPR/Cas9 is a potentially revolutionary platform to launch a vast number of individualized treatments for genetic diseases and cancers like sickle cell disease.
- With extensive resources/expertise, Vertex can help CRISPR to unlock substantial value for the CRISPR/Cas9 platform to capture lucrative orphan-disease markets.
- The stellar CAR-T platform is highly likely to work, and serves as a cushion for CRISPR to fall back on if CRISPR/Cas9 does not generate positive data.
Based in Basel, Switzerland, CRISPR Therapeutics (NASDAQ:CRSP) is focused on the innovation and development of the gene-editing technology platform (known as CRISPR/Cas9) and CAR-T for the potential treatment of a vast number of genetic diseases and cancers. Lead therapeutic CTX-001 is an investigational CRISPR/Cas9-based gene editing therapy that can potentially benefit patients afflicted by the blood disorders Beta-thalassemia (B-thalassemia) and sickle cell disease. The therapeutic works by the engineering of the patient’s blood stem cells with the genes to make more fetal hemoglobin (HbF) that, in and of itself, carries oxygen better than normal hemoglobin. By raising the HbF level in patients, the need for transfusions for B-thalassemia are significantly reduced.
On Dec. 7, 2017, Crispr Therapeutics announced the submission of a clinical trial application for CTX-001 as the potential treatment for the blood disorder B-thalassemia. Aiming to assess the efficacy and safety of CTX-001, the company is planning to commence the Phase I/II trial in Europe (beginning in 2018). Moreover, the firm is expected to file an investigational new drug application with the U.S. FDA this year. In response, the stock appreciated by $0.61 at $19.14 for 3.29% profits during the Dec. 8 trading session. For the past six months, shares also traded $4.36 higher for 30% overall profits.