FDA Approves Gene-Editing Treatment
Shares of Crispr Therapeutics (CRSP) had a volatile day on Friday after the Food and Drug Administration approved the company's groundbreaking gene-editing treatment for sickle cell disease. CRSP stock rallied ahead of the news, but shares then fell sharply shedding more than 8%.
The FDA approved the treatment that Crispr developed based on its gene-editing technology known as CRISPR. Vertex Pharmaceuticals (VRTX) co-developed the treatment.
"Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited," Nicole Verdun, M.D., director of the Office of Therapeutic Products at the agency's Center for Biologics Evaluation and Research, said in a statement.
In November, the U.K.'s Medicines and Healthcare products Regulatory Agency approved the Crispr treatment for patients age 12 and older with blood diseases known as sickle cell disease or beta thalassemia. CRSP stock surged on the news, and is up nearly 50% year to date.
CRSP stock sank 8.1% to close at 64.54.
Shares of Crispr Therapeutics (CRSP) had a volatile day on Friday after the Food and Drug Administration approved the company's groundbreaking gene-editing treatment for sickle cell disease. CRSP stock rallied ahead of the news, but shares then fell sharply shedding more than 8%.
The FDA approved the treatment that Crispr developed based on its gene-editing technology known as CRISPR. Vertex Pharmaceuticals (VRTX) co-developed the treatment.
"Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited," Nicole Verdun, M.D., director of the Office of Therapeutic Products at the agency's Center for Biologics Evaluation and Research, said in a statement.
In November, the U.K.'s Medicines and Healthcare products Regulatory Agency approved the Crispr treatment for patients age 12 and older with blood diseases known as sickle cell disease or beta thalassemia. CRSP stock surged on the news, and is up nearly 50% year to date.
CRSP stock sank 8.1% to close at 64.54.
Vertex, CRISPR Therapeutics Get FDA Approval for First CRISPR Gene-Editing Therapy in US
By BILL MCCOLL
Published December 08, 2023
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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 Food and Drug Administration (FDA) gave them approval for use of the first-ever gene-editing 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.1
Dr. Reshma Kewalramani, CEO of Vertex, said Casgevy “offers the potential of a one-time transformative therapy for eligible patients with sickle cell disease.”
Vertex noted that because the administration of Casgevy requires specialized experience 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%.
By BILL MCCOLL
Published December 08, 2023
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 Food and Drug Administration (FDA) gave them approval for use of the first-ever gene-editing 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.1
Dr. Reshma Kewalramani, CEO of Vertex, said Casgevy “offers the potential of a one-time transformative therapy for eligible patients with sickle cell disease.”
Vertex noted that because the administration of Casgevy requires specialized experience 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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