Crispr Therapeutics AG (NASDAQ: CRSP) stock enhanced over 8% on December 22nd, 2017 leading to a total rally of over 31% in the last six months. Analysts from Piper Jaffray issued an overweight rating on the stock with an $30 price target. Analysts forecasts that the group would file an Investigational New Drug (IND) application with the Food and Drug Administration before the end of 2018. This move would drive the stock to a new heights.
The group finished the preclinical activities during the third quarter for CTX001, aiming two hemoglobinopathies, Sickle Cell Disease and β-Thalassemia. The group is also planning to submit an IND application for CTX001 in Sickle Cell Disease in the first half of 2018. The group is also advancing in developing their proprietary CRISPR-based allogeneic CAR-T candidates and looking forward to reporting a new preclinical data on their lead CAR-T candidate, CTX101 for CD19+ malignancies.
The group teamed up with Vertex Pharmaceuticals for co-developing and co-commercializing CTX001, a gene editing treatment for β-thalassemia and sickle cell disease (SCD). A Phase 1/2 study in Europe should launch next year. CTX001, licensed to Vertex from CRISPR, is an ex vivo (out of the body) therapy in which a patient’s hematopoietic stems are engineered to produce high levels of fetal hemoglobin (HbF) in red blood cells. Elevated HbF might cut the need for transfusions in β-thalassemia patients and decrease episodes of painful vaso-occlusive crises in SCD sufferers. The group reported that the Phase 1/2 trial in β-thalassemia is forecasted to start in 2018.
The companies are collaborating on discovering and developing gene-editing treatments based on CRISPR/Cas9 under a 2015 agreement.
On the other side, the group formed Casebia Therapeutics, along with Bayer AG for developing CRISPR-based therapeutics in select disease areas, while signed a collaboration agreement with CureVac AG, a biopharmaceutical firm for mRNA-based drugs. CureVac would develop novel Cas9 mRNA constructs with better properties for gene editing applications, like rising potency, decreased the duration of expression, and reduced potential for immunogenicity. The group intends to leverage the CureVac mRNA manufacturing through clinical development and commercialization of the three programs. For this, CureVac would get an upfront payment and research funding, and would be eligible to get a development and commercial milestones and royalties on commercialized products arising from the collaboration.