CRISPR Therapeutics plans to begin industry-sponsored, clinical human trials with a CRISPR therapy in 2018. Officially submitted to European regulatory authorities, the application outlines a test of CTX001, a CRISPR treatment designed for patients with sickle cell disease (SCD) and beta-thalassemia.
Samarth Kulkarni, CEO of CRISPR Therapeutics, told Wired, “I think it’s a momentous occasion for us, but also for the field in general. Just three years ago we were talking about CRISPR-based treatments as sci-fi fantasy, but here we are.”
According to data collected through cell and animal experiments, CTX001 is highly efficient in editing the gene that produces fetal hemoglobin, which is mutated in patients with SCD or beta-thalassemia, and so far, the technique shows no signs of affecting other genes. These data were presented by CRISPR Therapeutics at the 2017 ASH Annual Meeting in Atlanta.
Stuart Orkin, MD, from Boston Children’s Hospital, told Chemical & Engineering News, “It is important that they do this very carefully. Because if there is a mistake or bad effect [from CRISPR], it will have repercussions beyond a single patient.”
However, if this clinical trial succeeds, it could lead to more innovative and boundary-pushing CRISPR treatments from antibiotic resistance to disease reversal and eradication. For an in-depth look at what CRISPR is and the concerns about unintended side effects of gene editing, see the ASH Clinical News feature, “Demystifying Gene Editing With CRISPR.”
Source: Futurism, December 17, 2017.