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CRISPR eyes autoimmune disease in revamp of cell therapy plans

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Dive Brief:

  • CRISPR Therapeutics is changing up its cell therapy plans, announcing Monday it will shelve its most advanced cancer drug prospects and pursue cell-based medicines for autoimmune diseases.
  • The biotechnology company is discarding two blood cancer treatments in early human testing, choosing instead to advance two “next generation” alternatives it claims are more potent. CRISPR also plans to evaluate one of those candidates in autoimmune conditions like lupus, with an early clinical study expected to begin in the first half of 2024.
  • The announcement comes days before the Food and Drug Administration is set to decide whether to approve the biotech’s sickle cell treatment Casgevy, which by Friday could become the first CRISPR gene editing medicine cleared for use in the U.S.

Dive Insight:

CRISPR Therapeutics achieved a major scientific milestone last month with the U.K. approval of Casgevy, the first in the world for a CRISPR medicine. Wall Street analysts are expecting a similar outcome with U.S. regulators.

Yet the sales outlook for Casgevy are unclear, and CRISPR partner Vertex Pharmaceuticals leads commercialization. For CRISPR to become profitable, some of its other programs will need to succeed as well.

The company lost about $112 million in the third quarter, and Fierce Biotech recently reported the company laid off some staff before Casgevy’s U.K. approval.

CRISPR’s most advanced work outside of Casgevy is in cancer, where it has advanced through early clinical testing a pair of similar programs in certain blood cancers and solid tumors. Both are off-the-shelf, or “allogeneic,” cell therapy treatments, which use healthy donor cells and are seen as more convenient to the personalized CAR-T cell therapies sold by Gilead Sciences, Novartis and Bristol Myers Squibb.

Like other allogeneic cell therapy developers, CRISPR has struggled to prove its treatments can match up to personalized CAR-T, however. Earlier data for a single dose disappointed, and newly released results on Monday showed an optimized dose regimen wasn’t much better. According to the company, preclinical results suggest a pair of newer medicines, dubbed CTX112 and CTX131 and now in Phase 1 testing, should be more potent. Those drugs are about a year behind the two it shelved, Jefferies analyst Maury Raycroft wrote in a Tuesday note to investors.

“While we don’t anticipate this announcement will have a substantial near-term impact on the stock, we do note this decision will elongate timelines for these programs,” added Baird analyst Jack Allen in a separate note.

CRISPR also now plans to test its cell therapies in autoimmune disease, a strategy that’s seen by large and small drugmakers as a way of expanding the medicines’ use. Lupus, in particular, is a disease of interest, following the release of promising data from an academic study in 2022. CRISPR is starting with lupus and could expand into other diseases afterwards, the company said.

It isn’t alone. A growing group of companies including startups Kyverna Therapeutics and Cartesian Therapeutics, publicly traded biotech Gracell Biotechnologies, as well as larger companies like Novartis and Bristol Myers, all have lupus cell therapies in development.

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