CRISPR Gene Therapy Gets First-Ever Regulatory OK

Casgevy, a CRISPR gene-editing therapy, has been authorized by UK authorities to treat people with two blood diseases..

(Photo by Nguyễn Hiệp on Unsplash)

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CRISPR, the game-changing genome-editing tool, is making its public debut.

UK drug regulator The Medicines and Healthcare products Regulatory Agency (MHRA) announced on Thursday that it had authorized the use of Casgevy, a CRISPR gene-editing therapy, to treat people with two blood diseases: sickle-cell disease and beta-thalassemia.

Time to Run with Scissors

CRISPR is a medical technology that’s sometimes described as genetic “scissors” — it’s able to go into cells and clip out strands from your DNA. You may remember it from about a million news cycles ago, when Chinese biophysicist He Jiankui shocked the medical community in 2018 after he used CRISPR to create two genetically edited human babies — a revelation that appalled the medical community and earned him three years in prison.

It hasn’t been all bad press, though. The 2020 Nobel Prize in Chemistry went to two scientists — Emmanuelle Charpentier and Jennifer Doudna — for developing the CRISPR/Cas9 genetic scissors, which the academy said would “lead to ground-breaking new medical treatments.” Well, it looks like those ground-breaking treatments just broke ground:

  • People with sickle-cell and beta-thalassemia have difficulty producing hemoglobin, and often require blood or even bone marrow transfusions as a result. The MHRA said the aim of Casgevy is to edit the genes of the cells in patients’ bone marrow stem cells, essentially fixing them so they can produce hemoglobin on their own.
  • The MHRA said that out of 29 sickle-cell patients who trialed the treatment, 28 reported they had been free of “severe pain crises” for 12 months following the treatment. For the beta-thalassemia trial group, 39 out of 42 said they hadn’t needed a blood transfusion for 12 months after starting treatment.

While the treatment holds plenty of promise for people living with those diseases, there is still something that could limit its impact: cost, which NPR said could run as high as $2 million per patient. Ken Shadlen, a professor in development studies at the London School of Economics, told The Daily Upside that the MHRA’s approval doesn’t automatically mean it will be available via the UK’s National Health Service. The desired price point of Casgevy’s makers, Vertex Pharmaceuticals and CRISPR Therapeutics, will determine whether British patients can access the treatment for free. “NICE [The National Institute for Health and Care Excellence] needs to undertake a cost-effectiveness assessment and determine if it’s worth paying what Vertex wants,” Shadlen said.

Crossing the Pond: Casgevy is making headway with the US Food and Drug Administration as well. Two weeks ago, the FDA said the companies could monitor for safety risks once Casgvey is out in the wild, and according to Reuters, analysts were hopeful that the treatment could get the FDA greenlight by early December. That would be one heck of a Christmas present.