Gene-editing tech like CRISPR is being commercialized, largely by these companies.
In science, discoveries build upon discoveries; breakthroughs scarcely happen in isolation, instead building upon decades or perhaps centuries of hard work and prior research. Gene-editing companies are a prime example of this snowballing knowledge: DNA was first discovered back in the 1860s, but the double helix structure wasn’t identified until 1953. It took another 50 years to completely map the human genome, and in 2021, the first-ever in vivo human gene-editing trial was conducted, with recent data from that study showing major promise for CRISPR, an exciting new frontier in medicine, to treat genetic diseases. This recent breakthrough is arguably one of the most meaningful milestones in science in years. Here’s a look at some of the top gene-editing stocks to buy in this incredible space.
Intellia Therapeutics (ticker: NTLA)
Gene-editing using CRISPR-Cas9 technology — the incredible technology that allows scientists to selectively delete or change problematic genes — has the long-term potential to actually cure genetic diseases. Emmanuelle Charpentier and Jennifer Doudna won the Nobel Prize in Chemistry in 2020 for their discovery of CRISPR tech eight years earlier. And Doudna went on to co-found Intellia, which on June 26 announced that its phase one trial for NTLA-2001, a drug candidate aiming to treat transthyretin (ATTR) amyloidosis, showed impressive data in early human trials. “These are the first ever clinical data suggesting that we can precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR,” said Intellia president and CEO John Leonard. That’s great news for NTLA, which doesn’t have an approved drug yet but is one of the few pure-play CRISPR stocks around. Within three trading days, NTLA stock had added as much as 128% on the news.
Regeneron Pharmaceuticals (REGN)
Regeneron earns a spot among the best gene-editing stocks to buy largely by virtue of its partnership with Intellia on the aforementioned ATTR drug. Regeneron, a roughly $60 billion biotechnology company, is the kind of partner cash-strapped but innovative biotech companies often seek out in early days. The established player infuses cash into the smaller one, gaining the opportunity to co-develop and ultimately commercialize the promising drug or treatment. Regeneron saw promise in Intellia early, reaching a deal in 2016 to work with Intellia on in vivo CRISPR therapies. REGN stock trades for about 15 times earnings and is consistently profitable, with its blockbuster macular degeneration drug Eylea hauling in billions a year in sales. It’s not a pure play on CRISPR by any means, but Regeneron has a stake in one of medicine’s most promising areas.
Crispr Therapeutics (CRSP)
Doudna wasn’t the only trailblazing CRISPR researcher to start a company based on the technology; Emmanuelle Charpentier, who shared the 2020 Nobel Prize in Chemistry with Doudna, founded Crispr Therapeutics in 2014, another upstart focused solely on CRISPR-Cas9 treatments. The company has four therapies in clinical trials, including treatments for sickle cell disease, the blood disorder beta thalassemia, and multiple myeloma. Although three of the four treatments in clinical trials are 100% owned by Crispr Therapeutics, the one focusing on patients with sickle cell and beta thalassemia, CTX001, will be co-developed and co-commercialized with Vertex Pharmaceuticals (VRTX), a $52 billion biotech company with the resources and experience in bringing successful drugs to market that CRSP lacks. The stock has been rising in sympathy with Intellia’s breakthrough, adding 35.8% between June 2 and July 2.
Editas Medicine (EDIT)
Another “pure play” on the remarkable CRISPR-Cas9 gene-editing technology, Editas is in a similar position to Intellia and Crispr Therapeutics, with no approved drugs to date but a bevy of promising medicines in development. At a $3.8 billion valuation, Editas is relatively small potatoes in the biotech industry, but its recent shareholders are certainly happy, with the stock up more than 56% between June 2 and July 2. After all, what’s good for the goose is good for the gander, and Editas has four in vivo gene-editing treatments in various stages of production that look a lot more likely to succeed following Intellia’s own promising early results with an in vivo CRISPR-based drug. The furthest along of those candidates is EDIT-101, the company’s treatment for Leber congenital amaurosis, the most common cause of inherited blindness in children.
Beam Therapeutics (BEAM)
Founded in 2017, Beam Therapeutics is newer on the block than many of its peers but has raced its way to a valuation of more than $8 billion since hitting the public markets in 2020. There’s a lot of excitement around Beam and its growth potential, as indicated by the stock’s meteoric ascent to around $130 a share within 18 months of its IPO at $17. Unlike the previous gene-editing stocks, Beam uses a base editing approach, utilizing CRISPR to change a single letter in the genome. In some cases, a single letter change can be the root of lifelong health issues, and Beam’s vision is to be able to completely cure such patients through its approach. Beam Therapeutics is developing treatments for sickle cell disease, beta thalassemia, and various liver and eye diseases.
Bluebird bio (BLUE)
Although not engaged in CRISPR-based treatments, the Cambridge, Massachusetts-based Bluebird bio uses gene therapy approaches to target severe genetic diseases and cancer. It does this by either adding genes or editing genes, and has 17 known medicines in different stages of its pipeline, with multiple unannounced treatments in the preclinical phases. One of the company’s gene therapies for beta thalassemia is now approved in the European Union, and another, the multiple myeloma immunotherapy Abecma, was approved by the U.S. Food and Drug Administration in March. Bluebird bio, which is less valuable than all other mentioned stocks at $2.2 billion, is spinning off its oncology unit into another publicly traded company by the end of the year, allowing it to focus fully on its genetic diseases segment. Shares have underperformed for years now, so a more targeted mission can only help.
Six of the top gene-editing stocks to buy now:
— Intellia Therapeutics (NTLA)
— Regeneron Pharmaceuticals (REGN)
— Crispr Therapeutics (CRSP)
— Editas Medicine (EDIT)
— Beam Therapeutics (BEAM)
— Bluebird bio (BLUE)
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Update 07/06/21: This story was published at an earlier date and has been updated with new information.