Roth Capital Partners initiated coverage of CRISPR Therapeutics PLC (NASDAQ:CRSP) with a “buy” rating and $50 price target. The stock closed at $44.38, up $1.50, on June 10.
CRISPR Therapeutics is a gene-editing company developing CRISPR/Cas9-based therapeutics. Its most advanced program is for genetically defined diseases of transfusion-dependent beta-thalassemia and severe sickle cell disease. “These are hemoglobinopathies with high unmet need, and the opportunity could be significant for a potentially curative therapy,” writes analyst Tony Butler.
For its lead product candidate, CTX001, the company is taking an “ex-vivo” approach, in which a patient’s cells are edited outside of the body using CRISPR/Cas9 before being re-infused, he added.
CRISPR and its partner, Vertex, are investigating CTX001 in a Phase 1/2 open-label clinical trial, with 45 patients, designed to assess the safety and efficacy of a single dose of CTX001 in patients aged 18-to-35 with beta-thalassemia. The study is being conducted at trial sites in Europe and Canada, with U.S. sites expected to open this year.
CRISPR and Vertex also are investigating CTX001 in a Phase 1/2 open-label clinical trial, with 45 patients, designed to assess the safety and efficacy of a single dose of CTX001 in patients aged 18-to-35 with severe sickle cell anemia. An FDA clinical hold placed on the study in May 2018 was lifted in October 2018, and sites in Canada and Europe are also set to open later in the year.
