Ultragenyx to spin off company to develop ‘high risk, high return’ gene therapy for Alzheimer’s disease
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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