Large biotech companies are getting more and more interested in the relatively new CRISPR-Cas9 gene editing treatment technology. The potential for this technology to treat eye diseases is certainly piquing the interest of one of biotech's leading companies: Allergan.
Recently, Allergan announced its intention to partner with the company Editas Medicine. Headquartered in Cambridge, Massachusetts, Editas is mainly concerned with treating genetic eye disorders using CRISPR-Cas9. Allergan paid Editas nearly £73 million for the ability to license five of Editas' experimental gene editing treatments.
Editas is currently working hard on a program to counter the rare genetic eye disease known as . Leber Congenital Amaurosis type 10 (LCA10). Although this disease is rare, it can lead to blindness. Investors expect Editas will apply to the FDA for approval to test their LCA10 therapy on humans before the end of 2017.
A few other genetic treatments Editas is working on include treatments for Usher Syndrome and Herpes Simplex Virus type 1. Editas hopes LCA10 will be the very first CRISPER-Cas9 genetic treatment to go into human trials.
People who follow the biotech world closely shouldn't see Allergan's deal as any real surprise. Indeed, Allergan has had great success in recent years with their experimental eye drugs. Estimates show that Allergan made over £500 million from various eye therapies in 2016 alone. These therapies mainly deal with helping patients with glaucoma or dry eye.
The main importance of this deal for the medical community is that it legitimizes CRISPR-Cas9 gene editing treatments. Geneticists believe the CRISPR-Cas9 has great potential in the future of genetic treatment, but this technology has yet to find big backers. Everyone has their fingers crossed that the first human tests using CRISPR-Cas9 will be a success.
Users of the CRISPER-Cas9 system use a pair of molecular scissors to literally cut a patient's DNA. There's also a programmable guide in the CRISPER-Cas9 system to help geneticists achieve optimal accuracy. People involved in CRISPER-Cas9 technology believe this therapy has the potential to cure hundreds of genetic eye diseases.
Of course, it's not all smooth sailing for CRISPR-Cas9 treatment. Some scientists have questioned this therapy's safety in the past few years. The only real way to find out if CRISPR-Cas9 will work for humans is to examine the success rate in human clinical trials.
Editas isn't the only company on the biotech market involved in CRISPR-Cas9 treatment strategies. The company Intellia Therapeutics also works with CRISPR-Cas9 and has entered into a deal with both Novartis and Regeneron Pharmaceuticals. Also, CRISPR Therapeutics as partnered with powerful companies like Vertex Pharmaceuticals and Bayer.
Although the LCA10 treatment is Editas' best-known treatment, the company has numerous other therapies in preclinical trails. Just a few other diseases Editas hopes to cure using CRISPER-Cas9 therapy include cystic fibrosis, alpha-1 antitrypsin deficiency, and sickle cell disease.
The reason some people might be familiar with the name Editas has to do with a major court victory the company won in February. Editas' founders from both MIT and Harvard were embroiled in a patent struggle over the CRISPR-Cas9 with a few researchers at the University of California, Berkeley. The court ruled in Editas' favor, which gave the company a huge boost in the stock market.
Editas is traded on the NASDAQ under the ticker symbol EDIT.