Crispr Therapeutics, Vertex get world's first regulatory green light for CRISPR-based medicine

By Eleanor Laise

U.K. regulator is first to approve a therapy based on groundbreaking gene-editing technology

Shares of Crispr Therapeutics (CRSP) and Vertex Pharmaceuticals Inc. (VRTX) gained ground premarket on Thursday after the companies got the world's first regulatory authorization for a medicine based on CRISPR, the transformative gene-editing technology.

A U.K. regulator approved Casgevy, a gene-edited therapy, for the treatment of sickle cell disease, an inherited disorder affecting red blood cells, and transfusion-dependent beta thalassemia, a condition that causes severe anemia and dependence on red blood cell transfusions.

"Today is a historic day in science and medicine," given the groundbreaking regulatory action, Dr. Reshma Kewalramani, president and CEO of Vertex, said in a statement.

Casgevy works by editing a faulty gene in patients' bone-marrow stem cells, prompting the body to produce functioning hemoglobin, the protein in red blood cells that delivers oxygen to tissues. To accomplish that, the stem cells are taken out of bone marrow, edited in a lab and then infused back into the patient, according to the U.K.'s Medicines and Healthcare Products Regulatory Agency. Patients may need to spend at least a month in the hospital while the treated cells get established in the bone marrow, the regulator said in a statement Tuesday.

The therapy, also known as exa-cel, is also under review by the U.S. Food and Drug Administration. The FDA has granted priority review for exa-cel in sickle cell disease, with a decision expected by Dec. 8.

Crispr Therapeutics shares soared earlier this month after an FDA advisory panel took a positive view of exa-cel for the treatment of sickle cell disease. The FDA is not obligated to follow advisory committees' advice, but it often does.

Exa-cel is "very likely to be approved" by the FDA by early December, Evercore ISI analysts wrote in a research note after the advisory committee meeting. An FDA decision on exa-cel in thalassemia is expected by Mar. 30.

"I hope this represents the first of many applications of this Nobel Prize-winning technology to benefit eligible patients with serious diseases," Samarth Kulkarni, CEO and chairman of CRISPR Therapeutics, said in a Thursday statement.

Crispr Therapeutics shares gained more than 5% premarket on Thursday and are up 38% in the year to date, while Vertex shares gained 0.5% premarket and have gained 21% so far this year.

-Eleanor Laise

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11-16-23 0850ET

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