CRISPR Therapeutics CRSP and its partner Vertex Pharmaceuticals VRTX announced the dosing of the first patient in a phase I/II study evaluating the CRISPR/Cas9 gene-editing therapy, CTX001, in patients with beta thalassemia, a form of anemia. This is the first in-human use of CTX001 in any clinical study.
The companies also enrolled first patients in another phase I/II study evaluating CTX001 in patients with severe sickle cell disease (“SCD”), a severe hereditary form of anemia. Dosing in the study is expected to start in mid-2019.
Shares of CRISPR Therapeutics surged more than 25% following the announcement of the progress made by the company in studies on CTX001. However, the stock has declined 14.3% in the past year.
We remind investors that last month, the FDA assigned Fast Track designation CTX001 for the treatment of SCD. With this designation, the drug is expected to be granted a priority review once the company files a new drug application.
In December 2018, Vertex and CRISPR Therapeutics selected CTX001 to move into clinical development as a gene edited treatment for sickle cell disease and beta -thalassemia. 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 will 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.
Other than CRISPR Therapeutics, Intellia Therapeutics NTLA and Editas Medicine, Inc EDIT plan to carry out clinical studies using CRISPR Cas9 to cure diseases.
CRISPR Therapeutics also announced its fourth-quarter results in a separate press release. The company reported revenues of $0.1 million, which came from collaborations, compared to $32.3 million in year-ago period. Reported loss was 92 cents per share in the fourth quarter. The company achieved breakeven results in the year-ago quarter.
The company remains on track to initiate an immuno-oncology study in the first half of 2019 on its CAR-T cell therapy candidate, CTX110, for treating CD19+ malignancies. The company is the sole owner of the candidate. Since September, the company has inked or modified several collaboration agreements with other pharma companies for pre-clinical development of its new CRISPR/Cas9 gene editing candidates.
CRISPR Therapeutics currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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