CRISPR and Vertex: Front-Runners in Gene-Therapy

03/21/2019 5:00 am EST

Focus: HEALTHCARE

Jim Powell

Principal Analyst, Global Changes & Opportunities

In addition to high-quality blue chip, long-term holdings, we also occasionally look to long-term opportunities that have the potential to return lottery-sized profits, asserts Jim Powell, editor of Global Changes & Opportunities Report.

Some of the most promising are in the field of gene therapy that is beginning to deliver life-saving therapies. Theories about why lifespans are finite suggest that changing key parts of our DNA should make repairs more effective.

With the new Crispr/Cas9 gene editing system — making changes in human DNA is now being studied throughout the world. Using the new tool to increase human lifespans — while improving our overall health — is now within sight.

Meddling with our DNA, of course, can be dangerous, but the hope is that Crispr/Cas9 technology can be used to change individual patients’ genes in search of genetic cures for cancer, cystic fibrosis, heart disease, diabetes, and AIDS — to name only a few.

Any investor who wishes to take an early position in what promises to become a life-changing biotech advance should consider buying CRISPR Therapeutics (CRSP). The company pioneered the Crispr/Cas9 gene-editing tool and is the leader in using it to develop new disease therapies.

CRISPR’s first target is beta thalassemia, a form of anemia. With its partner Vertex Pharmaceuticals (VRTX), the company is in Phase I/II trials to bring a cure to market. Coming up next will be a gene therapy cure for sickle cell disease.

Both companies are focused on changing the genes of individual patients, not making DNA modifications that could last for generations. The two pioneering companies — CRISPR and Vertex — are front-runners in the gene therapy field.

Both companies should be considered speculations. CRISPR is a pure-play that has yet to make a profit, and may be years away from doing so. Vertex is not a gene therapy pure play, but it is profitable and should appeal to somewhat more conservative investors who want to take a lower risk position in the field.

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