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Clinical Moves for CRISPR

CRISPR Therapeutics, the biotech firm founded by CRISPR researcher Emmanuelle Charpentier, now at the Max Planck Institute for Infection Biology, is submitting paperwork in Europe and the US to embark on clinical trials, Wired reports.

The company has asked European regulators to be able to conduct a trial on treating beta thalassemia and plans to file an investigational new drug application for sickle cell disease with US regulators in early 2018, Wired adds. It notes that both conditions stem from mutations in the HBB gene, which encodes the beta-globin subunit of hemoglobin. The company's treatment approach doesn't focus on fixing that gene but ramping up the expression of another, fetal hemoglobin, which usually ceases production six months after birth. In August, CRISPR Therapeutics' Samarth Kulkarni told Technology Review that they were able to successfully modify 85 percent of the stem cells they isolated from patients to make healthy red blood cells.

Wired notes that other CRISPR-based companies are also moving toward clinical trials. Being the first to do so "isn't just about bragging rights; being first could be a big boon to building out a business, and a proper pipeline," it adds.