Last year ended difficulty for publicly traded gene editing companies but at J.P. Morgan’s virtual healthcare conference this week, gene-editing firms are saying that this year will bring exciting developments.
Intellia will expand its clinical trial of the first gene-editing treatment administered directly into patients. The New England Journal of Medicine called its August 2021 report on the first six patients one of the notable advances of the year. Using the homing ability of a Crispr molecule, Intellia and its partner Regeneron Pharmaceuticals (REGN) knocked out the defective gene responsible for a poisonous protein that damages nerve and heart tissue. In December, Intellia dosed the first patient with a therapy that knocks out another gene whose defects cause body-wide swelling. In the next few weeks, the company expects to start dosing 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 is well-fixed to test these treatments and others.
CEO Leonard said investors should watch for new delivery modes that direct gene-editing treatments to a particular tissue. Intellia’s first two treatments find their way to the liver inside fatty globes called lipid nanoparticles. Researchers are finding vehicles that direct gene editors to blood stem cells and nervous tissue.
Leonard also expects his sector’s leading firms to diversify their gene-editing technologies. Intellia is developing its own version of base-editing—an evolution of Crispr-Cas editing that avoids slicing both strands of our DNA, and instead gently corrects a single undesired letter of genetic code. Pfizer’s new partner Beam has pioneered base-editing development.
Intellia will discuss its road map with investors at J.P. Morgan’s conference on Wednesday. Also talking on Wednesday will be a Crispr-Cas rival, Editas.
Editas is moving more slowly than Intellia in its clinical trials. In September, doctors reported hints of efficacy in a Phase 1 study of the company’s treatment for a kind of hereditary blindness. A gene-editing treatment for sickle cell disease will start dosing patients in this year’s first half, Editas said in a Monday press release. Before the end of 2022, the company will also test the treatment in patients with the blood cell disease called beta thalassemia.
In partnership with Bristol Myers Squibb (BMS), Editas is developing therapies that modify immune cells to fight tumors. The gene-editing company had nearly $660 million in cash at September’s end, but in its Monday release it said that it is hunting for partners to help advance applications of its editing technologies in the treatment of cancer and regenerating damaged tissue.
To learn more about CRISPR & Gene Editing please visit the XDNA Index page or watch the video below
Thank you for visiting Kelly Intelligence
You are now being redirected to our [NAME] site.