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Exagamglogene autotemcel is the first cell-based gene therapy treatment utilizing CRISPR/Cas9 gene editing technology to be approved by the US Food and Drug Administration (FDA). [13] The most common side effects include low levels of platelets and white blood cells, mouth sores, nausea, musculoskeletal pain, abdominal pain, vomiting, febrile ...
In a meeting with US senators in December 2018, Doudna was asked about the potential high cost of a CRISPR-based treatment of sickle cell disease and what could be done to bring these costs down. When she returned to the IGI following this meeting, she decided to make affordability a part of the mission of the IGI, and a key goal for its sickle ...
The US Food and Drug Administration on Friday approved two gene-based treatments for sickle cell disease, including the first therapy that uses the gene-editing technique CRISPR, opening a new era ...
While CRISPR Therapeutics and Vertex estimate a target market of 35,000 patients in the U.S. and Europe, it won't matter if the medicine is inaccessible to many of them due to its cost.
Still, the new therapy is extremely expensive — $2.2 million per patient, Vertex said. The pricing strategy, experts argue, may place it out of reach for many families.
CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland.It was one of the first companies formed to utilize the CRISPR gene editing platform to develop medicines for the treatment of various rare and common diseases.