CRISPR: The Next Step Forward in Eradicating Congenital Diseases

Ben Benoy Economy and MarketsGene editing has been a controversial topic this year! Back in April we learnrned that the Chinese had used a technology called CRISPR to edit non-viable human embryos for the first time.

So basically… by “non-viable,” the embryo wouldn’t have grown into a human either way. But it still meant tampering with some idea of a person.

But even then, it was the first time anyone had successfully edited the human genome.

Imagine the possibilities for treating rare genetic diseases…

If we can modify a person’s DNA, we can wipe out sickle cell anemia… Huntington’s… cystic fibrosis.

This is unchartered territory, folks.

Well now, this technology is taking another step forward… with a company called Editas Medicine. Their goal: to treat a rare form of congenital eye disease.

This event marks yet another milestone for the gene editing technology. This time, CRISPR will be used on a living person’s genome to treat a disease.

And just think – the Chinese got heat for messing with an embryo that was pretty much already gone!

The disease Editas is targeting is called “Leber congenital amaurosis.” That’s LCA for short. It’s an inherited eye condition that causes irregular development of the retina. This hereditary disease starts causing major vision problems as soon as infancy. And it affects roughly 600 people per year in the United States.

That leaves CRISPR with a reasonable sample size to get the job done.

The Editas treatment, which so far has only been run in the lab, is considered gene therapy. The treatment will contain viruses that assemble the CRISPR enzyme complex inside the body… directly affecting the mutated genes.

MIT, who co-developed the technology, offers a simpler explanation: Basically, once the mutated disease genes are modified via CRISPR, they should be able to function normally.

Oorah! (Sorry, a little marine slang for you.)

Editas is one of the first CRISPR-focused biotech companies to start testing gene therapy. In August, the company stated it would focus on LCA treatment after receiving a cash influx of $120 million.

If the tests go well, the CRISPR technology will grab a stronger foothold in the biotech community. It may be seen as a viable path for treatment, possibly exploding the market for this type of therapy.

For BioTech Intel Trader readers, I’ve added the CRISPR technology to my social media collective intelligence system and am tracking its effects throughout the market.

I’m looking forward to seeing what it and other gene editing therapies can offer. We’re one step closer in the push to eradicate inherited diseases.

Ben Benoy

Ben Benoy

Editor, Biotech Intel Trader