Swiss Crispr and Vertex receive approval for the USA
Published: Friday, Dec 8th 2023, 21:40
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The Nasdaq-listed biotech company Crispr Therapeutics, based in Zug, has achieved a success: The Swiss company and its partner Vertex Pharmaceuticals have received approval for the gene therapy Casgevy from the US Food and Drug Administration (FDA).
This is the first time that a gene therapy has been approved to treat patients with sickle cell anemia, the FDA announced in a press release on Friday evening. The approval applies to patients aged 12 and over.
Sickle cell anemia is a hereditary blood disorder in which defective red blood cells carry too little oxygen and can clog the blood vessels. This leads to tissue and organ damage, which can result in death. Around 100,000 people in the USA suffer from the disease, according to the FDA.
Vertex's Casgevy therapy uses the Crispr/Cas9 technique, which can be used to cut the genetic material (DNA) of blood-producing bone marrow cells. This removes faulty pieces of DNA. The modified blood-producing bone marrow cells are then reintroduced into the patient. This stimulates the production of healthy red blood cells, the FDA wrote.
This stem cell transplant is expensive: according to the news agency Reuters, a single treatment with Casgevy costs 2.2 million dollars.
As a result of the FDA approval, the Zug-based biotech company will now receive a milestone payment of 200 million dollars from Vertex, as both companies also announced. Crispr Therapeutics will receive 40 percent of the profits, Vertex 60 percent.
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