Editas Medicine just announced it plans to deploy CRISPR gene editing technology in humans as early as 2017.
Editas CEO Katrine Bosley told an audience at the MIT Technology Reviews’ EmTech conference this week that it’ll kick off clinical trials in 2017 to treat a rare eye disorder called Leber congenital amaurosis (LCA). See Bosley speak here.
The Cambridge startup received an impressive $120 million funding round in August, which should keep it flush with cash through the launch and early stages of LCA clinical trials.
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Business Insider writes:
Bosley didn’t specify the age of people with LCA in the trial, but it’ll likely be done in adults using what she described as a gene-therapy-like treatment. To date, no gene therapies have yet been approved in the US, though one to treat another form of LCA has had promising results in late-stage trials.
CRISPR/Cas 9 has thus far not been used in humans – just non-viable human embryos. Editas’ target, a rare eye disease, is caused by a minor flaw in the genetic coding that leads to severely impaired vision. The CRISPR treatment will be injected directly into the retina, so it’s site specific – allowing researchers to evaluate its efficacy with some level of ease. Editas will use this technology to delete a portion of the CEP290 gene that causes LCA. However, as Business Insider points out:
But like any experimental treatment, this one comes with some caveats. First, LCA affects only about 3,000 people in the US, and just 20% of those have the type that Editas’ treatment addresses, Jean Bennet, the Director of Advanced Retinal and Ocular Therapeutics at the University of Pennsylvania, told MIT Technology Review. That means that even if it proves successful, it’ll likely be very expensive to treat. And launching studies to treat more widespread diseases could still be more than a few years off.
