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Regeneron Pharmaceuticals (NASDAQ: REGN) is a leading biotechnology innovator developing transformative treatments for serious diseases through advanced platforms like VelociSuite®. This page serves as the definitive source for official company announcements, research milestones, and therapeutic developments.
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Regeneron Pharmaceuticals (Nasdaq: REGN) and Alnylam Pharmaceuticals (Nasdaq: ALNY) announced positive interim results from the Phase 1 clinical study of ALN-APP, an RNAi therapeutic targeting amyloid precursor protein for Alzheimer’s disease and cerebral amyloid angiopathy. The study enrolled 20 patients, revealing that single doses of ALN-APP achieved dose-dependent reductions of soluble APPα and APPβ in cerebrospinal fluid, with decreases of up to 90%. Safety and tolerability were encouraging, with only mild to moderate adverse events reported. This marks the first demonstration of RNAi gene silencing in the human brain, utilizing Alnylam's C16 platform. Further data will be disclosed at an upcoming scientific conference, and the trial continues to explore longer-term effects.