Gene therapy getting more effective and accurate - New startup to shake things up

11/29/2013 - 00:00

A new startup, backed with $43 million in venture investments, aims to develop treatments that could cure inherited diseases with a one-time fix based on a new method of genome editing. The method offers great precision in changing the DNA sequence of a genome and can potentially treat diseases that other forms of gene therapy cannot.

The new method, to be commercialized by Editas Medicine, is based on a genome-editing system that was largely unknown just two years ago. The company’s founders say the method will allow them to go after currently untreatable diseases.

In the last two years, scientists have come to better understand that many microbes use a system of protein and RNAs (molecular cousins of DNA) to defend themselves against invading viruses. Researchers have adapted this bacterial immune system, referred to as CRISPR/Cas, to edit single base pairs of the human genome as well as larger stretches of DNA. “Big ideas like this emerge in the science world infrequently,” says Douglas Cole of Flagship Ventures, one of the three life-science-focused venture-capital firms investing in Editas.