That drove gene editing stocks higher this week
This has been a monumental week for the gene editing pioneer Intellia Therapeutics (NASDAQ: NTLA). This was the last of the three original CRISPR companies to start a clinical trial, but it was worth the wait. Clear indications that Intellia’s revolutionary new method of treating hereditary diseases is actually working have propelled shares up 93% since the market closed last Friday.
The notable news has shares of radiotherapy (NASDAQ: BEAM), Editas medicine (NASDAQ: EDIT), and CRISPR therapeutics (NASDAQ: CRSP), 42%, 35% and 10% higher before US markets opened this morning.
Image source: Getty Images.
Intellia’s first new drug candidate to begin clinical trials, NTLA-2001, is permanently working on a problematic gene in the body. CRISPR Therapeutics’ first clinical-stage therapy candidate, CTX001, alters genes in stem cells after they are removed from the patient. Editas Medicine’s first clinical-stage candidate, EDIT-101, will be injected into the patient’s eyeballs and is expected to remain there. Beam Therapeutics has still not tested any new drug candidates in the clinical phase.
Intellia’s lead candidate is a pair of messenger RNA strands surrounded by a specialized fat bubble or lipid nanoparticle (LNP). Unlike the Editas Medicine candidate, NTLA-2001 can safely travel through the bloodstream to reach the liver.
In 2020, it seemed like a distant dream to send strands of RNA safely through the bloodstream so that they can instruct cells to produce therapeutic proteins. Last year, Modern and BioNTech showed us that LNP technology has finally reached a stage that is making RNA-based drugs a real possibility.
BioNTech and Moderna’s RNA-based vaccines instruct human cells to make a spike protein that helps our immune system recognize the real virus immediately. Intellias gene therapy to treat transthyretin amyloidosis with polyneuropathy (ATTR-PN) also uses RNA, but there is much more.
The genetic instructions in NTLA-2001 cause liver cells to produce a CRISPR-Cas9 complex that specifically deactivates the transthyretin gene. This prevents transthyretin from entering the bloodstream, where it can fall apart and get jammed in places it doesn’t belong.
Intellia’s revolutionary gene therapy candidate appears to be working even better than hoped. A single dose of just 0.1 milligrams per kilogram of body weight reduced circulating transthyretin by 52% in three ATTR-PN patients in this group. Among the patients given 0.3 milligrams, investigators measured an average reduction of 87%.
Alnylam already commercializes successful therapies for a very limited population of ATTR-PN patients. Intellia investors are more excited about the successful use of LNP-encapsulated strands of mRNA to induce cells to make gene-editing machines. That’s because it should be relatively easy to develop more therapies by exchanging transthyretin for various problematic genes.
It was nice to see several gene editing stocks jump higher in response to the good news from Intellia, but they probably shouldn’t have. That’s because Intellia is miles ahead of its competitors.
Beam Therapeutics is working on LNP-encapsulated RNA strands for the treatment of glycogen storage disorders and alpha-1 antitrypsin deficiency. Beam’s unique approach does not create double-stranded breaks in DNA like NTL-2001 does. This should lead to more accurate editing and better results. It will be a while before we know exactly. The company hasn’t even nominated a candidate that uses LNPs and RNA to advance into human-stage testing.
Editas Medicine’s lead candidate is in the clinical trial phase. Instead of mRNA trapped in an LNP, EDIT-101 uses a viral vector to deliver DNA that instructs cells in the eye to make gene-editing machinery. At the moment, it doesn’t look like Editas Medicine or CRISPR Therapeutics have any plans to develop gene-editing therapies similar to Intellia.
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Cory Renauer has no position in any of the stocks mentioned. The Motley Fool owns shares of and recommends CRISPR Therapeutics and Editas Medicine. The Motley Fool recommends Alnylam Pharmaceuticals and Moderna Inc. The Motley Fool has a disclosure policy.
The views and opinions expressed herein are those of the author and do not necessarily reflect those of Nasdaq, Inc.