BioCentury
ARTICLE | Tools & Techniques

Editing CF

Why CRISPR-Cas9 technology could work in CF where gene therapies haven't

November 23, 2015 8:00 AM UTC

Vertex Pharmaceuticals Inc. and CRISPR Therapeutics AG are collaborating to see whether CRISPR's gene editing technology can succeed where gene therapies have come up short for cystic fibrosis. The technology is expected to produce more consistent efficacy with fewer safety risks, but under a four-year research partnership announced late last month, the companies will still have to solve some of the same delivery challenges that prevented gene therapies from fulfilling their initial promise.

The first human gene therapy trial for CF was conducted in 1993, but more than 20 years later, the technology hasn't produced a viable CF therapy. Reasons range from limited efficacy to severe immune reactions. While gene therapies as a class have overcome many of those hurdles, others remain, including variable transgene expression and targeted delivery to the right tissue or cells. ...