Trials & Filings

Alnylam Begins Subcutaneous ATTR Trial

Tests RNAi teatment against rare heart disease

By: Gil Roth

President, Pharma & Biopharma Outsourcing Association

Alnylam Pharmaceuticals has begun a pilot Phase II study of ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin (TTR) gene in development for the treatment of TTR-mediated amyloidosis (ATTR). The trial, which is now open for enrollment, is aimed at evaluating the tolerability and preliminary clinical activity of ALN-TTRsc in TTR cardiac amyloidosis patients with familial amyloidotic cardiomyopathy (FAC) — which is caused by autosomal dominant mutations in the...

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