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CRISPR Takes a Leap Forward With Huntington's Disease

- By Matt Winkler

The CRISPR-cas9 gene editing technique is one of the most exciting advances in genomics since Watson and Crick discovered the chemical structure of DNA in 1953. Basically, it allows the selective editing of genes, potentially allowing humans to fix errors that cause genetic diseases and any other number of science fiction-like applications.

The main problem is CRISPR can cause mistakes in DNA after the cut strands are excised and the rest glued back together. This can lead to transcription problems, causing unforeseeable issues. That makes it very scary to apply for practical purposes because new errors can cause worse problems than the disease you are trying to cure.


This problem is one step closer to being resolved thanks to a group of scientists out of Poland. In a paper published in the journal Frontiers in Neuroscience on Feb. 26, Drs. Magdalena Dabrowska, Wojciech Juzwa, Wlodzimierz Krzyzosiak and Marta Olejniczak detail a new way to apply CRISPR. With their technique, the cutting mechanisms in CRISPR are altered to nick DNA rather than cut it completely. The nicking, by what's called a nickase, causes single-strand breaks, which are fixed by a much more accurate DNA repair mechanism, precluding most transcriptions errors.

The researchers were able to show the nickase application of CRISPR effectively cuts out faulty genes, in this case Huntington's disease, a dominant degenerative motor disease with similar symptoms to ALS that strikes in middle age. It is caused by a repeating code at the end of the huntingtin gene, which in turn causes a mutated protein to clump together, becoming toxic.

The researchers used a cell line from a Huntington's patient, edited the genes of these cells using CRISPR-cas9 nickase and found that up to 82% of the toxic protein was eliminated without errors. So where can this lead in terms of investment?

The corporate history of CRISPR can be useful here to understand what could happen now. The history is very short, basically consisting of three public companies. Two of them sprung out of the two co-discoverers of the technique, and a third out of another scientist who discovered how to apply it to human cells. Given the history, the nickase technique could soon spur a fourth, or else be merged in to one of the other three or some other company interested in the space.

All three CRISPR pure-plays have market caps over $1 billion and some are up over 100% since their initial public offering, so there is a lot of money in going public with CRISPR technology. Editas Medicine (EDIT), at $1.6 billion, was founded by Dr. Fang Zheng, who discovered who to apply CRISPR to humans. Intellia Therapeutics (NTLA), founded by Dr. Jennifer Doudna, a co-discoverer of CRISPR itself, is at $1.1 billion, and though shares have zigzagged since IPO they are still up about 20%. Finally, there is the aptly named CRISPR Therapeutics AG (CRSP), at $2.2 billion, up 250% since IPO, founded by the other co-discoverer, Dr. Emmanuelle Charpentier.

Could these four Polish scientists follow up on the CRISPR tradition of quickly vaunting out into the public markets to raise $1 billion or more? Possibly, or the technology could be acquired by one of the partners of these three CRISPR pure-plays, like Amgen (AMGN), Celgene (CELG) or Vertex (VRTX).

So far, developments have proceeded very quickly, so if this group of scientists can show their corporate counterparts there is potential here, we could see the CRISPR nickase editing technique enter the biotech space relatively soon.

Disclosure: No positions.

This article first appeared on GuruFocus.