Alnylam Releases Proof-of-concept Data on Preclinical Porphyria Drug | GenomeWeb

Alnylam Pharmaceuticals last week released preclinical data demonstrating proof of concept for its porphyria treatment ALN-AS1.

The drug is designed to silence ALAS-1, a liver-expressed rate-limiting enzyme upstream of porphobilinogen deaminase. Inhibition of ALAS-1 has been shown to reduce the accumulation of heme precursors that cause the clinical manifestations of acute intermittent porphyria — a form of porphyria Alnylam is focusing on.

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