3 Things About CRISPR Therapeutics That Smart Investors Know
Many biotech companies have been formed hoping to be first to market with CRISPR gene-editing therapies. CRISPR Therapeutics (NASDAQ: CRSP) and its development partner Vertex Pharmaceuticals (NASDAQ: VRTX) are leading the way with the CTX001 therapy for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
These blood disorders are particularly good targets for CRISPR techniques, as blood stem cells can be readily harvested, edited, and reintroduced into patients. On June 11, CRISPR Therapeutics and Vertex held a joint press conference highlighting CTX001 results, which showed that patients in a 22-person phase 1/2 clinical trial are having a "consistent and sustained response to treatment."
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