The past few months have demonstrated that mRNA vaccines can be highly effective, economically viable, and rapidly developed. It’s easy to imagine future applications where mRNA vaccines will become the standard approach and displace traditional technologies.
At the heart of these successes lies advances in drug delivery, which may continue to provide momentum for not only the expanding repertoire of mRNA therapeutics, but a broad array of gene editing technologies (like CRISPR) as well. The exciting news last week from Intellia Therapeutics, is no exception.
Exciting News From Intellia Therapeutics
On June 26, results of a Phase 1 trial for Intellia’s NTLA-2001 therapy were published in The New England Journal of Medicine. Trial participants underwent treatment for transthyretin amyloidosis, a progressive and life-threatening disease affecting many parts of the body. Participants experienced only mild adverse reactions and demonstrated up to an 87% reduction in the disease causative agent. This was excellent news for Intellia and the gene-editing sector in general.
NTLA-2001 is an in-vivo technology—gene editing conducted within the patients themselves, and injected intravenously. Intravenous delivery is inherently non-specific; an injected drug can travel to almost any tissue. This is a problem from an economic perspective because it is inefficient; more importantly, it is a safety concern. CRISPR technology in general may be used to “knock-in” or introduce new genetic material; “knock-out” or remove genetic material; or conduct a combination of the two. Intellia’s therapy knocks out the target gene, called TTR.
Removing the mutated TTR gene in the liver cells of patients with hereditary transthyretin amyloidosis reduces or eliminates the pathological buildup of the mutated protein, transthyretin; however, removing TTR in cells outside the liver could potentially have a harmful effect. Intellia achieved targeted delivery of NTLA-2001 to the liver, demonstrating that tissue-specific CRISPR medicines can be delivered intravenously.
Beneficial Implications
Although the trial involved only 6 participants, Intellia’s recent success with NTLA-2001 is a triumph not only for this specific trial and not only for Intellia, but for the entire gene-editing sector. Intellia has demonstrated that tissue-specific CRISPR medicines can be delivered intravenously with minimal off-target effects. Dozens of targets, like TTR, have already been identified for which the DNA editing itself is straightforward and delivery remains the primary hurdle.
Furthermore, the lipid-nanoparticle delivery mechanism used by Intellia is similar in many ways to the platforms used by other companies in the CRISPR and mRNA medicines space. While the liver may be somewhat of an “easy target” for lipid-nanoparticle delivery, Intellia’s success likely signals more favorable odds for not only their ongoing and future trials but any trial reliant on in-vivo editing.
Challenges remain for mRNA medicines and the CRISPR gene-editing space, but we continue to follow this closely.
Investing In Biotechnology
I have a personal passion for investing in the Biotech field and believe this sector offers excellent long-term value and growth. We will likely see advancements in medicine and health that no one could have imagined even 10 or 20 years ago. I encourage clients to create an investing strategy and to think about how their money can make a positive impact on the world while also providing a financial foundation for retirement.
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About Rick
Richard Gurz is co-founder and managing principal of Quantis Wealth Management, an independent financial services, wealth management, and tax planning and consulting firm in Tysons Corner, Virginia, Washington, D.C., and Glenn Dale, Maryland. With over 20 years of experience, Rick is committed to providing comprehensive, personalized strategies to help his clients achieve their financial goals. He is known for building long-term relationships and walking his clients through everything life throws their way, making sure their financial plan is on track for their ideal future. Rick specializes in serving high-net-worth pre-retirees and is passionate about empowering and educating his clients to make prudent financial decisions that help them secure their quality of life.
Rick graduated from Mary Washington College with a bachelor’s degree in political science and international affairs and holds the CERTIFIED FINANCIAL PLANNER™, Certified Investment Management Analyst® (CIMA®), Certified Private Wealth Advisor® (CPWA®), and Accredited Investment Fiduciary® (AIF®) professional designations. When he’s not working, you can find Rick spending time with his wife, Julie, and their two daughters, Adeline (Addy) and Josephine (Josie), usually watching his daughters play soccer or tennis or going on hiking and camping adventures. He also enjoys fishing, biking, traveling, golf, and serving his community. To learn more about Rick, connect with him on LinkedIn.