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Vertex, CRISPR Therapeutics Get FDA Approval for First CRISPR Gene-Editing Therapy in US

CRISPR Therapeutics

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Key Takeaways

  • Vertex Pharmaceuticals and CRISPR Therapeutics received regulatory approval for the first gene-editing therapy using CRISPR technology in the U.S.
  • The treatment, called Casgevy, is used to treat sickle cell disease, an inherited blood disorder.
  • Casgevy uses the Nobel Prize-winning CRISPR technique to edit the cells that cause the illness.

Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP) announced Friday that the 澳洲幸运5开奖号码历史查询:Food and Drug Administration (FDA) gave them approval for use of the first-ever gene-editin♎g therapy using CRISPR🍬 technology in the U.S.

The companies’ Casgevy is a treatment for sickle cell disease, an inherited blood disorder. Casgevy uses the Nobel Prize-winning CRISPR genetic modification technique to edit patients’ DNA to target the effects of the disease.

Dr. Reshma Kewalramani, CEO of Vertex, said Casgevy “offers the potential of a one-time transformati♍ve therapy for♑ eligible patients with sickle cell disease.”

Vertex noted that because the administration of Casgevy requires specialized experienceඣ in stem cell transplantation, the company is “engaging with experienced hospitals to establish a network of independently operated, authorized treatment centers.” It explained that currently, nine🐭 medical centers across the country have been activated. 

Shares of Vertex finished 1.1% lower Friday following the news, while CRISPR Therapeutics shares lost 8.1%. However, both remained higher for the year, with Vertex shares up 22.4% year-to-date, while CRISPR Therapeutics shares have gained over 57%.

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