Gene engineering as envisioned by science fiction isn’t coming. It’s already here. How can you cash in?

Small-cap biotech Intellia Therapeutics (NSDQ: NTLA) is by far the best way to profitably leverage the accelerating trend of creating new drugs by editing genes.

Today’s heroes of innovation are the biologists, chemists, and other scientists who are forging novel medicines from gene-editing “toolkits.” This is the new frontier of science.

Biotech firms are editing DNA to treat and prevent diseases such as Alzheimer’s, Huntington’s, Parkinson’s, multiple sclerosis, sickle cell anemia, a wide range of cancers… just about any ailment you can think of.

In 2020, the Nobel Prize in Chemistry went to Jennifer Doudna and Emmanuelle Charpentier for their advances in genome editing. (The “genome” is the entirety of a living organism’s hereditary information.)

Doudna and Charpentier created a revolutionary gene-editing tool called CRISPR, an acronym for “clustered regularly interspaced short palindromic repeats.” Let’s put aside the jargon.

Here’s what you need to know: CRISPR is the hottest area in biotech research today. The following chart shows how it works:

The Nobel-winning work of these two women has triggered a wave of venture capital funding for bioengineering startups involved in CRISPR. Indeed, these techniques were applied in 2020 and into 2022 in the concerted global effort to find COVID vaccines.

Several gene-editing tools already were in existence, but CRISPR is the most cost-effective and precise. CRISPR guides molecular scissors to a targeted section of DNA to disable or repair a gene, or to insert a new molecule where the scissors have made a cut. Think of CRISPR as a genetic-engineering “cruise missile.”

Doudna went on to co-found Intellia, which was incorporated in 2014 and is headquartered near the Massachusetts Institute of Technology (MIT) campus in Cambridge, Massachusetts.

The company started as a venture capital-backed startup and launched its initial public offering on May 6, 2016.

Currently sporting a market cap of $3.9 billion, Intellia has license and collaboration agreements with Novartis (NYSE: NVS) and Regeneron Pharmaceuticals (NSDQ: REGN).

Intellia uses the CRISPR system to develop new treatments. The company’s in-vivo (taking place in a living organism) product development programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis (a neurodegenerative disease); and NTLA-2002 for the treatment of hereditary angioedema (which causes severe under-the-skin swelling); as well as liver-focused programs comprising hemophilia (blood-clotting disorders).

Intellia’s ex-vivo (outside of a living body) programs include NTLA-5001 for the treatment of acute leukemia and proprietary programs focused on developing engineered cell therapies to treat various oncological and autoimmune disorders.

A promising clinical trial…

Intellia has teamed up with Regeneron to begin a clinical trial to treat transthyretin amyloidosis, a rare disease in which proteins generated by a malfunctioning liver accumulate in the bloodstream to the point of becoming lethal.

Under the auspices of Intellia and Regeneron, physicians are injecting CRISPR molecules into the volunteers’ livers to turn off the defective gene.

Speaking at a scientific conference on June 24, Intellia researchers reported that a single dose of the gene editing treatment produced a substantial decline in the protein level in volunteers’ blood for as long as a year.

NTLA doesn’t have an approved drug… yet. But when it comes to CRISPR, the company is the best “pure play” you can find.

Staffed by some of the best scientific minds in the world, Intellia has the financial wherewithal to continue its groundbreaking research. The company’s partnerships with Big Pharma also add safety and a modicum of revenue ($37.8 million over the trailing 12 months) as it develops its future drug pipelines. Intellia also sports ample cash on hand of $855.2 million (most recent quarter).

For aggressive investors eager to tap a technological mega-trend, Intellia suggests enormous upside potential.

Speaking of tech innovation…we’ve uncovered the first “Apple” of the electric vehicle industry. One unusual, under-appreciated company is secretly siphoning the EV industry’s trillion-dollar profits into investor accounts over and over again, like clockwork. Want to get in on the action? Click here for details.

John Persinos is the editorial director of Investing Daily.

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