Ultragenyx to spin off company to develop ‘high risk, high return’ gene therapy for Alzheimer’s disease
17 Oct 2023
Gene TherapyAcquisition
Ultragenyx plans to spin off a company dedicated to developing a novel gene therapy for Alzheimer’s disease that makes use of a lysosomal enzyme, the company
revealed Monday during its investor day
.
The drugmaker expects to retain majority ownership of the spinoff, which will be named Amlogenyx. The new company plans to close a Series A by the end of the year.
Ultragenyx is known for its work in rare lysosomal storage disorders, and the shift to a common neurodegenerative disease may come as a surprise. The company explained in the presentation on Monday that a specific lysosomal compound, known as protective protein/cathepsin A (PPCA), was found to affect amyloid beta in studies of another disease, galactosialidosis.
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