Intellia Therapeutics (NASDAQ: NTLA) is a biotechnology company using CRISPR gene-editing technology to develop several experimental drugs, many of with having the potential to save countless lives. As a relatively new drug development technology, Intellia is one of several companies at the forefront of CRISPR-based drug research and development. With a strong network of pharmaceutical partners and several proprietary methodologies, Intellia stands out in a crowded field as one of the most promising pharmaceutical companies conducting research and development with CRISPR.

What is Intellia Therapeutics (NTLA)?

Intellia Therapeutics was established in 2014 by a group of American scientists working on CRISPR-related research in academic research labs across the country. Notable within this group is Jennifer Doudna, co-founder and one of the scientists who received a Nobel Prize in 2020 for her work on pioneering CRISPR work. CRISPR, short for clustered regularly interspaced short palindromic repeats, is a technology developed as a result of observing a process that naturally occurs within viruses. By isolating this CRISPR segment of the virus responsible for replication and deletion, scientists have been able to develop “genetic scissors”, which can delete, remove, and modify portions of DNA.

Based in Cambridge, Massachusetts, the company employs just over 200 staff in a variety of research and clinical settings. As with many startup pharmaceuticals, Intellia needed to raise substantial capital to start conducting research and developing a pipeline of promising therapeutics. Novatris, a Swiss pharmaceutical company, was one of Intellia’s early backers. Intellia entered an agreement to work with Novatris to develop CRISPR-based Chimeric antigen receptor T cells therapies in exchange for $15 million in funding. Relationships with Novatris and other large pharmaceutical companies such as Regeneron allowed Intellia to sustain itself through the startup stage. With this support, the company was able to develop several breakthroughs in its early years. A few short years later in 2016, Intellia listed on the NASDAQ.

Why does Intellia Therapeutics (NTLA) Matter?

Using CRISPR-based drug development technologies, the company has several drugs in the pipeline at various stages of development. Furthest along in the clinical trial process is Intellia’s OTQ923/HIX763 Sickle Cell Disease drug. Several other companies in the biotech space, such as CRISPR Therapeutics and Editas Medicine, are also using CRISPR to develop treatments for sickle cell disease. Intellia Therapeutics was recognized for its work, receiving a substantial boost in November 2020 when the Bill and Melina Gates Foundation awarded them with a grant to further their research on sickle cell disease therapeutics. The grant was awarded to further Intellia’s work on their proprietary non-viral delivery system for in vivo genome editing.

While several companies are working on Sickle Cell Disease treatments using CRISPR, Intellia is also focusing efforts on advancing therapies for diseases with high unmet need. One of the more novel drugs in development is their Transthyretin Amyloidosis drug, which is currently in Phase 1 of clinical trials. Transthyretin Amyloidosis, which is a condition that results in the buildup of protein deposits in organs and tissues, has a life expectancy of 2-15 years after diagnosis. Approximately 50,000 around the world are born with the disease and an additional estimated 200,000 to 500,000 additional individuals have developed the disease over their lifetime.  There are currently no treatment options available, and Intellia is the company furthest along in finding a cure.

Intellia Therapeutics (NTLA) Prospects

The company has focused efforts on a “full-spectrum” approach to CRISPR drug development. This approach means that drugs are being developed for both ex-vivo and in-vivo uses. In in-vivo drugs, genes are edited within the body, while ex vivo medicines edit genes outside of the body and transfer them back into the patient. In this full-spectrum approach, the company is employing a proprietary lipid nanoparticle (LNP) delivery system for drugs. The LNP delivery system involves two parts: a messenger RNA that encodes the protein with the required genetic changes and the guide RNA that can target a segment within the DNA sequence. This technique involves the ability to either remove, restore, or perform both actions at the same time. This greatly expands the ability to edit genes, allowing drugs to either knockout specific sequences, insert them, or consecutively edit using a combination of the two techniques. Mixing and matching in this way allow for a combination of different gene-editing techniques to be applied simultaneously.

With several drugs in the pipeline with costly research and development price tags, the company has partnered with larger pharmaceuticals who have provided financial and scientific support. Most notable are their partnerships with Regeneron and Novatris. Regeneron is currently is a partner on three of the six in vivo drugs being developed by Intellia, while Novatris is a partner on two of five ex vivo drugs currently under development. Regeneron provided Intellia with $125 million in 2016 for an equity stake in 10 drugs at various stages of development. This was supplemented by an additional $100 million in 2020, with the money earmarked for drug development to treat hemophilia A and B.

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Intellia Therapeutics is one of the premier companies currently working on CRISPR-based medicines. The company has made major headway on several drugs, all of which have the potential to change countless lives. Backed by major pharmaceutical companies and a stellar scientific team, Intellia has the financial and scientific basis necessary to continue moving drugs along in the pipeline. With their investments into research and development, several proprietary methods have been developed which are helping expedite the process. Combined, all of these factors make Intellia Therapeutics a promising company that has plenty of room for growth.

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