Biotech company Editas to begin human gene editing in 2017

A US biotechnology start-up co-founded by two pioneers of CRISPR technology intends to begin gene editing in humans as part of an experimental treatment to target a rare genetic eye disorder.

Katrine Bosley, CEO of Editas Medicine, announced that research involving the technique, CRISPR/Cas9, will begin to tackle leber congenital amaurosis (LCA), with a clinical trial expected in 2017.

The treatment would involve injecting the retina, the light-sensitive layer of cells at the back of the eye, with modified viruses carrying CRISPR/Cas9. This would be used to delete a portion of DNA in the CEP290 gene, believed to be implicated in the disorder.

People affected with LCA have difficulty seeing anything other than large, bright shapes from infancy. The condition was selected for the first trial as it is easy to target with CRISPR/Cas9, Bosley explained. Additionally, as it affects vision, the success or otherwise will be more obvious afterwards. It is estimated that around 600 people in the USA are affected by the type of LCA that Editas plans to treat.

The use of federal funds for gene editing in human embryos is currently banned in the USA (see BioNews 800) and Editas will also require approval from regulators before embarking upon the trial.

The technology has attracted controversy over safety and ethical concerns about editing the germline in a way that would affect future generations. CRISPR/Cas 9 has so far only been tested in animals and non-viable human embryos.

'It feels fast, but we are going at the pace science allows', Bosley told the EmTech conference in Cambridge, Massachusetts.

Editas was co-founded by Professors Jennifer Doudna and Feng Zhang, who are currently embroiled in a patent dispute over the CRISPR/Cas9 technique (see BioNews 802). In August, it raised US $120 (£80) million from investors to fund research like the kind being done with LCA.

The MIT Technology Review says that Editas is one of a number of start-ups that plan to use CRISPR techniques to target genetic conditions in people, partly because it is so cheap to use but also for its precise application.

The Progress Educational Trust's public conference 'From Three-Person IVF to Genome Editing: The Science and Ethics of Engineering the Embryo' is taking place in central London on Wednesday 9 December 2015.