I want to bring your attention to what is, in my opinion, “The Next Big Thing.” This technology — which is behind my Top Pick for aggressive investors in 2022 — involves one of the most profound wonders of nature: the code of life embedded within human DNA, notes John Persinos, editorial director of Investing Daily.
Intellia Therapeutics (NTLA) is by far the best way to profitably leverage the accelerating trend of creating new drugs by editing genes. But first, let’s take a quick trip through the wonders of gene editing.
Silicon Valley’s titans transformed the economy, financial markets and society. They started out as tech geeks of modest means, but now they’re gazillionaires who move markets and bask in social adulation. The pandemic made these information technology pioneers even richer, as people increasingly rely on their innovative products and services to work and play at home.
Well, make way for the new heroes of innovation: the biologists, chemists, and other scientists who are forging new medicines from gene-editing “toolkits.” This is the new frontier of science.
The race for a Covid-19 vaccine, which produced effective treatments with astonishing speed, underscores the life-and-death role of med-tech. New techniques for gene editing are coming to the fore that promise upheavals on a par, or perhaps even greater, then those wrought by social media, e-commerce or personal computing.
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 scourge 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 Nobel-winning work of these two women has triggered a wave of venture capital funding for bioengineering start-ups involved in CRISPR. Indeed, these techniques were applied in 2020 and 2021 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 $9.4 billion, Intellia currently has license and collaboration agreements with Novartis (NVS) and Regeneron Pharmaceuticals (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.
NTLA doesn’t have an approved drug…yet. But when it comes to CRISPR, the company is the best “pure play” you can find. With $529 million in cash on hand (most recent quarter) and staffed with some of the best scientific minds in the world, Intellia has the wherewithal to continue its groundbreaking research.
The company’s partnerships with Big Pharma also add safety and a modicum of revenue ($41.4 million over the trailing 12-months) as it develops its future drug pipelines.