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For Intellia and its gene-editing competitors Crispr Therapeutics, Editas, and Beam, the first findings on Crispr genomic-editing within people was promising.
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Stocks of
Intellia Therapeutics is a pharmaceutical company based in the United States.
are up 45 percent to $129 in early Monday trade after the biotech firm said over the weekend that its gene-editing treatment for a lethal liver ailment appears to be a cure. The company’s market valuation of $9 billion demonstrates investor confidence in its single-dose treatments for other hereditary disorders in its sights, such as heart disease, hemophilia, and pulmonary hypertension.
On a conference call early Monday morning, Intellia (ticker: NTLA) CEO John Leonard remarked, “These data represent a major milestone for medicine for patients with these serious conditions.” For some, the information was unwelcome.
Alnylam Pharmaceuticals is a pharmaceutical company based in the United States.
(ALNY), whose gene-editing solution could eliminate the need for pharmacological therapies for the same genetic illness. In early Monday trade, Alnylam’s stock fell 10% to $159. After 28 days, Intellia’s single-dose Crispr-Cas9 treatment reduced blood levels of a misfolded protein by up to 96 percent in patients suffering from ATTR amyloidosis, a genetic illness in which TTR protein accumulation causes deadly nerve and heart dysfunction. The paper is the first to describe a systemic application of the Crispr-Cas9 gene editing technique, which was awarded the Nobel Prize in Chemistry last year for its invention.
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Analysts that suggested Intellia stock were ecstatic, and their target prices will almost certainly be raised. In a weekend note, RBC Capital Markets analyst Luca Issi called Saturday’s dataset “a home run.” Joon Lee of Truist increased the price objective on his Buy rating from $80 to $160. RBC’s Issi anticipates retail investors to flock to Intellia’s stock, as well as those of gene-editing rivals, now that the company’s gene-editing approach has been “de-risked.”
Crispr Therapeutics is a company that specializes in the treatment of
(CRSP),
Editas Medicine is a non-profit organization that publishes
(EDIT), as well as
Beam Therapeutics is a company that specializes in the treatment of
(BEAM). Those stocks were up 10% to 15% in early morning trading on Monday. Yatin Suneja of Guggenheim also greeted the early, encouraging data as good news for the firm.
Regeneron Pharmaceuticals is a pharmaceutical company based in the United States.
(REGN), which is Intellia’s ATTR amyloidosis project partner. If the gene therapy makes it to an ATTR market worth more than $15 billion per year, the businesses will split profits. Suneja wrote on Sunday, “We believe Regeneron might potentially buy Intellia to gain exclusive access to this new, game-changing technology suite.” Regeneron’s stock climbed 4% to $571 in Monday trade. Regeneron stock has a Buy recommendation from Guggenheim, with a target price of $685. Alnylam, a biotech that has seen skyrocketing revenues and shares thanks to its medications that prevent the function of the genetic mutation targeted by Intellia’s gene-editing therapy, had a less favorable weekend. Alnylam’s medicines must be administered every few months and have been shown to reduce misfolded ATTR protein levels in the blood by more than 80%. Intellia’s one-time treatment, which may produce higher degrees of knock-down, poses a strong competitive challenge.
Pfizer
(PFE) has also seen rapid growth in sales of high-priced medications that help to mitigate some of the effects of ATTR amyloidosis. While a gene-editing cure could reduce demand for such treatments, Pfizer is a large corporation with a stock that scarcely dipped on Monday. In the midst of the enthusiasm, keep in mind that Intellia’s Phase 1 trial is only the first step on a long road to commercial approval. The FDA has been particularly cautious in its regulation of genetic medicine trials, ordering several halts as developers improve production and investigate potential side effects. According to the study’s lead investigator, Julian Gillmore of London’s Royal Free Hospital, there were no serious adverse events in the first six patients whose data were released on Saturday, and no evidence that the Crispr molecules caused editing on genes other than the ATTR amyloidosis mutations targeted in liver cells. On Monday’s teleconference, Gillmore said, “This first demonstration of Crispr-based in-vivo gene-editing in people gives proof-of-concept of this therapy.” Laura Sepp-Lorenzino, Intellia’s chief scientific officer, told listeners that the company’s Crispr-targeting compounds and lipid-particle delivery vehicles could be used to treat various ailments. The first patient in a study of a hereditary condition that causes hazardous swelling all over the body has been dosed. Although liver cells can be targeted to treat this disease, Sepp-Lorenzino said Intellia’s pipeline includes gene-editing medicines that might target organs like bone marrow and the nervous system to treat diseases like hemophilia and pulmonary hypertension, a life-threatening lung ailment. On Monday’s call, CEO Leonard stated, “We are just getting started.” Bill Alpert can be reached at william.alpert@barrons.com.
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