The Gene-Editing Industry Weighs In on Outlook at J.P. Morgan Conference

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An Intellia Therapeutics Lab. The company is expanding its clinical trial of the first gene-editing treatment administered directly into patients.

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Courtesy of Intellia

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Biotech stocks have lost their sheen over the past six months, at least in the stock market as the Covid-19 panic subsides.

Particularly hard hit were start-up firms that were developing treatments to correct faulty genes. But at JPMorgan’s virtual healthcare conference this week, gene-editing companies such as Intelia Therapeutics (ticker: NTLA) and Editas Medicine (EDIT) are saying this year will bring exciting growth.

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Big pharma sees a buying opportunity.

Germany’s Bayer (BAYN.Germany) began the week by saying on Sunday it would pay $40 million, and potentially $1 billion more, to work with Mammoth Biosciences, a private company co-founded by Jennifer Doudna. organized ventures—which won the Nobel Prize. Emmanuel Charpentier for his discovery of the Crisp-Cas gene-editing technique. On Monday, Pfizer unveiled a $1.3 billion partnership with gene-editing firm Beam Therapeutics (BEAM).

“It’s moving very quickly,” Intelia chief executive John Leonard said. baron’s, “Further and roughly, at the same time.”

Intellia will expand its clinical trial of the first gene-editing treatment administered directly in patients. New England Journal of Medicine Called its August 2021 report on the first six patients one of the remarkable progress of the year. Using the homing potential of a Crisp molecule, Intelia and its partner Regeneron Pharmaceuticals (REGN) knocked out the faulty gene responsible for a toxic protein that damages nerve and heart tissue. In December, Intellia gave the first patient a therapy that knocks out another gene whose defects cause inflammation throughout the body. In the next few weeks, the company hopes to begin administering doses to the first patient in a cancer study that will modify immune cells to recognize and kill leukemia tumors.

With $1 billion in cash on its balance sheet, and partners like Regeneron, Intellia are all set to test these treatments and others.

CEO Leonard said investors should watch out for new delivery modes that direct gene-editing treatments into a particular tissue. Intellia’s first two treatments find their way to the liver inside fatty globes called lipid nanoparticles. Researchers are looking for vehicles that direct gene editors to blood stem cells and neural tissue.

Leonard also expects major firms in his field to diversify their gene-editing techniques. Intellia is developing its own version of base-editing—an evolution of crisp-cas editing that avoids cutting both strands of our DNA, and instead gently corrects an unwanted letter of the genetic code. Pfizer’s new partner Beam has pioneered base-editing development.

Intellia will discuss its road map with investors at JPMorgan’s conference on Wednesday. Also talking Wednesday is a crisp-cass rival, Editas.

Editas is proceeding more slowly than Intellia in its clinical trials. In September, doctors reported signs of efficacy in a Phase 1 study of the company’s treatment for a type of hereditary blindness. A gene-editing treatment for sickle cell disease will begin dosing patients in the first half of this year, Editas said in a Monday press release. Before the end of 2022, the company will also test the treatment in patients with a blood cell disease called beta thalassemia.

In partnership with Bristol Myers Squibb (BMS), Editas is developing treatments that modify immune cells to fight tumors. The gene-editing company had about $660 million in cash at the end of September, but in its Monday release it said it was looking for partners to help advance applications of its editing techniques in cancer treatments and regenerate damaged tissue. is looking for.

Over the past year, Editas’ stock has fallen from $88 to $26, while Intellia’s clinical trial results took it from $73 to $180, and then back up to $104 more recently. Both have buy ratings mostly from the Wall Street analysts who track them. Analysts’ price targets point to an upside 66% for Intelia shares and about 90% for Editas stock.

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