Crispr Therapeutics’ Gene-Edited Treatment Stopped Cancer in Some Patients – Barron’s

Posted by btwatson on October 21, 2020 3:38 pm
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Using a gene-editing technique that won a recent Nobel Prize, Crispr Therapeutics cleared blood cancers... — Using a gene-editing technique that won a recent Nobel Prize, Crispr Therapeutics cleared blood cancers in patients with off-the-shelf immune cells. Previously, a patient’s own cells were required.

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Using the Crispr gene-editing technique that won a recent Nobel Prize,

Crispr Therapeutics

cleared blood cancers in patients with off-the-shelf immune cells. These so-called CAR-T therapies previously required a patient’s own cells.

In a Wednesday morning announcement, Crispr Therapeutics (ticker: CRSP) said that its gene-editing let doctors use cells from healthy donors—opening up prospects for broadly available, less-expensive use of CAR-T treatment.

In the Phase 1 trial, the lymphoma blood cancer in four of 11 patients responded completely to infusions of T cells whose genes were altered to target the cancer and prevent transplant rejection. Standard treatments had failed all participants. In patients that got higher doses, the complete responses have lasted for months.

One of the responders who got the highest dose died of an apparent viral infection, which revived after treatment. That death may explain why Crispr stock is down 10% in Wednesday morning trading, to $96.49.

“Crispr gene editing just makes better T cells and CAR-T,” said Crispr Therapeutics CEO Same Kulkarni in the conference call.

CAR-T treatment using a patient’s own T cells is a time-consuming, expensive procedure, but has produced dramatic responses in refractory blood cancers. On the Wednesday call, University of Kansas Medical Center hematology professor Joseph McGuirk said Crispr Therapeutics’ off-the-shelf treatment produced responses comparable to conventional CAR-T.

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A patient’s own cells are called autologous, while those from a donor are called allogeneic. “Over time, we believe Crispr-edited allogeneic CAR-T has the potential to leapfrog autologous CAR-T and benefit much broader patient populations,” said Kulkarni.

The Crispr treatment made three edits to genes in the donated T cells, helping them to evade transplant problems using the technology that won a chemistry Nobel Prize two weeks ago for the company’s scientific founder Emmanuelle Charpentier. “We’ve used multiple lots from healthy donors,” said Kulkarni.

The five-year old company has two other CAR-T trials under way and has also used gene-editing in a clinical trial to successfully treat the red-blood-cell disease known as sickle cell. Other companies are developing allogeneic donor CAR-T treatments, including

Allogene Therapeutics

(ALLO).

Crispr Therapeutics plans to expand its CAR-T trials to include blood cancers beyond lymphoma and then to solid tumors.

Write to Bill Alpert at william.alpert@barrons.com

Published at Wed, 21 Oct 2020 14:02:00 +0000

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