Alnylam Pharmaceuticals, Inc. (ALNY) has submitted a Clinical Trial Authorization application to The Medicines and Healthcare Products Regulatory Agency in the United
Kingdom to begin a phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein for the treatment of Alzheimer's disease and cerebral amyloid angiopathy.
Alnylam Pharmaceuticals plans to begin the phase 1 study in patients with early-onset Alzheimer's disease in early 2022, upon obtaining MHRA approval, and expects to report initial human data at or around year-end 2022.
ALN-APP, the first-ever RNAi therapeutic targeting CNS diseases, is being advanced in
partnership with Regeneron as part of Alnylam Pharma's 2019 agreement.
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