Key Points
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Gene-editing is a handy tool for developing sophisticated medicines.
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A new CRISPR molecule can destroy cells that express specific signals.
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That could eventually create huge opportunities for new programs for the biotechs.
- 10 stocks we like better than CRISPR Therapeutics ›
An exciting new CRISPR-based tool is making waves in the scientific literature this spring. Because the new technology destroys targeted diseased cells outright rather than editing their genetic material, it’s being informally called the “DNA shredder.”
For shareholders of CRISPR Therapeutics (NASDAQ: CRSP) and its gene-editing peers like Intellia Therapeutics (NASDAQ: NTLA) and Beam Therapeutics (NASDAQ: BEAM), the emergence of a new form of genetic medicine could have major financial implications down the road. Let’s unpack what the new tech can do and what it means for CRISPR Therapeutics as well as these other stocks.
This is the frontier of science, not a near-term commercial event
The newly discovered tool reads a signal unique to a diseased cell and subsequently destroys that cell’s DNA by cutting it into fragments faster than the cell can repair it. That continues until the cell dies once it can’t reproduce its own cellular machinery; cells without the triggering sequence are spared.
In short, this is programmable cell-killing and not gene-editing. And that’s a capability that could be enormously useful across a range of clinical and research applications.
The new tool is already demonstrating its potential to treat serious illnesses. A May paper published in the journal Nature from researchers at the University of Utah showed the therapy cutting the growth of lung cancer cells that are driven by mutations in one of the most common cancer-driving genes by about 50% in a lab dish. That matched the chemotherapy drug cisplatin in the same test, with no effect on healthy cells.
A follow-up Nature paper in June from a lab at University of California, Berkeley — one of the two labs whose 2012 work established CRISPR-Cas9 as a programmable gene-editing tool — extended the shredding approach to target a gene that’s mutated in 40% to 50% of all cancers, and one of oncology’s most challenging targets to develop drugs for. All of this work remains in test tubes and animal models, with the obstacle of developing the shredder technology into a medicine that’s useful to treat human patients still unsolved.
None of this new tech is in CRISPR Therapeutics’ pipeline, nor in the disclosed pipelines of any of its major competitors. The closest it gets is using this editing to produce Casgevy, which CRISPR Therapeutics developed with Vertex Pharmaceuticals. Similarly, Intellia Therapeutics uses this editing as part of its candidates to treat hereditary angioedema (severe swelling) and transthyretin amyloidosis (a progressive, life-threatening disease caused by a buildup of abnormal protein deposits in organs and tissues). Beam Therapeutics uses base editors in alpha-1 antitrypsin deficiency and sickle cell disease.
The only company working to develop a therapy using the tool is Akribion Therapeutics, a private German biotech. Its lead program targets HPV-positive head and neck cancer, but it’s years from clinical trials, let alone the market.
What investors should actually take from this
The most important takeaway for investors here is that the new papers significantly expand the addressable market for CRISPR-based medicine.
The ability to perform efficient cell destruction based on a genetic sequence addresses a huge problem that gene-editing alone doesn’t address, specifically the possibility of competing in areas like oncology and virally driven cancers, where killing the diseased cells could constitute a breakthrough in treatment. None of the public CRISPR names is anywhere near such a breakthrough with this today, and it’s unclear if they even have enough capital or expertise in oncology to pursue new programs using the shredder technology.
But there is one vector where CRISPR Therapeutics and the other gene-editing stocks could become relevant players. The paper in the May issue of Nature showed promise in the same drug-delivery infrastructure that CRISPR Therapeutics, Intellia, and Beam have spent a half-decade building for their particular purposes. Having a functional delivery system for a gene-editing complex means that the next payload they invent doesn’t need to include spending on making new delivery infrastructure.
So the incumbents will likely have an advantage relative to newcomers if they choose to compete.
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Alex Carchidi has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends Beam Therapeutics, CRISPR Therapeutics, Intellia Therapeutics, and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy.
