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☆ New sickle cell gene therapies are a breakthrough, but solving how to pay their high prices is a struggle
▼ + stars: | 2024-02-23 | by ( Bertha Coombs | ) www.cnbc.com time to read: +2 min

The approval of two gene therapies to treat sickle cell disease has given hope to patients who suffer from the debilitating disease, which overwhelmingly affects Black people and people of color. Sickle cell has forced him to leave his job and at times taken him away from his family. Still, he's hesitant to try the new one-time gene therapies because they require months of intensive medical preparation, including chemotherapy, to prepare patients' bone marrow stem cells for extraction and gene editing. Vertex Pharmaceuticals ' gene therapy Casgevy lists for $2.2 million, while Bluebird Bio 's treatment Lyfgenia lists for $3.1 million. Kanter said it will take time to ramp up capacity and to set up facilities across the country to treat patients at scale.

Persons: Michael Goodwin, Goodwin, I've, I'm, he's, …, Goodwin's hesitancy, Julie Kanter, Kanter Organizations: Health, Vertex Pharmaceuticals, University of Alabama, National Alliance of Sickle Cell Centers, for Disease Control, National Alliance of Sickle Cell Locations: Birmingham

☆ US FDA approves Vertex/CRISPR gene therapy for an inherited blood disorder
▼ + stars: | 2024-01-17 | by ( ) www.cnbc.com time to read: +1 min

The headquarters of US biopharmaceutical company Vertex Pharmaceuticals in Boston, Massachusets, on November 4, 2023. The U.S. health regulator has approved Vertex Pharmaceuticals and CRISPR Therapeutics' gene therapy to treat a rare blood disorder requiring regular blood transfusions, in patients 12 years and older, Vertex said on Tuesday. The decision earns the therapy, branded as Casgevy, the second U.S. approval after it was greenlighted in December for sickle cell disease, another inherited blood disorder. The approval by the Food and Drug Administration comes more than two months ahead of its expected action date of March 30. Oppenheimer analyst Hartaj Singh said he expects a "slow and steady launch" for the therapy and estimated combined peak sales of about $400 million.

Persons: Oppenheimer, Hartaj Singh Organizations: Vertex Pharmaceuticals, Pharmaceuticals, CRISPR Therapeutics, Food and Drug Administration Locations: US, Boston, Massachusets, U.S, United States

LONDON (AP) — Britain's medicines regulator has authorized the world's first gene therapy treatment for sickle cell disease, in a move that could offer relief to thousands of people with the crippling disease in the U.K. The agency approved the treatment for patients with sickle cell disease and thalassemia who are 12 years old and over. Casgevy is currently being reviewed by the U.S. Food and Drug Administration; the agency is expected to make a decision early next month, before considering another sickle cell gene therapy. Millions of people around the world, including about 100,000 in the U.S., have sickle cell disease. Scientists believe being a carrier of the sickle cell trait helps protect against severe malaria.

Persons: ”, Helen O'Neill Organizations: Medicines and Healthcare Regulatory Agency, Vertex Pharmaceuticals, CRISPR Therapeutics, Helen O'Neill of University College London, Medicines, U.S . Food, Drug Administration, Associated Press Health, Science Department, Howard Hughes Medical Institute’s Science, Educational Media Group, AP Locations: Europe, CRISPR, South Asian, Britain, U.S, Africa, India, Eastern

☆ UK regulators approve medical treatment involving CRISPR gene editing in world first
▼ + stars: | 2023-11-16 | by ( Katie Hunt | ) edition.cnn.com time to read: +4 min

CNN —The United Kingdom has become the first country to give regulatory approval to a medical treatment involving the revolutionary CRISPR gene editing tool. The treatment, made by Vertex Pharmaceuticals, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a lab. “Modifying the stem cells from the bone marrow of the patient avoids the problems associated with immune compatibility, i.e. The release from the MHRA did not say how much the treatment would cost, but it’s likely to be expensive. CRISPR-Cas9 has had a major impact on biomedical research, clinical medicine and agriculture and is widely used in labs around the world.

Persons: —, ”, Julian Beach, “, — Emmanuelle Charpentier, Jennifer A, Doudna —, Casgevy, Alena Pance, ” Pance, Cas9, Jiankui Organizations: CNN, country’s Medicines, Healthcare, Agency, Beta, Vertex Pharmaceuticals, US Food and Drug Administration, University of Hertfordshire, Science Media Centre Locations: United Kingdom, South Asian

☆ Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval
▼ + stars: | 2023-11-16 | by ( Gina Kolata | More About Gina Kolata | ) www.nytimes.com time to read: +1 min

Regulators in Britain on Thursday approved the first treatment derived from CRISPR, the revolutionary gene-editing method. Called Casgevy, the treatment is intended to cure sickle-cell disease and a related condition, beta thalassemia. The companies anticipate that the Food and Drug Administration will approve Casgevy for sickle-cell patients in the United States in early December. The agency will decide on approval for beta thalassemia next year. That treatment does not rely on gene editing, insteading using a method that inserts new DNA into the genome.

Organizations: Vertex Pharmaceuticals, CRISPR Therapeutics, and Drug, Bluebird Bio Locations: Britain, Boston, Switzerland, United States, Somerville, Mass

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