How do we know Alzheimer’s drugs ‘work’?

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Good morning, everyone. Damian here with an update on a thwarted gene therapy study, a head-to-head fight in GLP-1, and a critical look at Alzheimer’s treatment.

The need-to-know this morning
• Regulators in the U.K. approved a CRISPR-based medicine to treat both sickle cell disease and beta thalassemia, making it the world’s first therapy built on the revolutionary gene-editing technology and ushering in a new phase of genetic medicine. The treatment, called Casgevy, was developed by Vertex Pharmaceuticals and CRISPR Therapeutics. In granting conditional approval Thursday, the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) jumped ahead of its counterparts in the U.S. and Europe, which are also reviewing the medicine.
• The FDA approved a new treatment for people with non-small cell lung cancer that contains a specific genetic alteration called Ros1. The genetically targeted medicine, called Augtyro, will be sold by Bristol Myers Squibb.
• Springworks Therapeutics said its experimental drug called mirdametinib achieved the main goal of a clinical trial by shrinking tumors in children and adults with neurofibromatosis type 1, a rare inherited disorder.
• Astellas announced the acquisition of Propella Therapeutics, a privately held developer of cancer drugs, for $175 million.

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