porphyria
Alnylam Expands Genetic Testing Program
The company will now offer free genetic testing to patients at risk for acute hepatic porphyrias, a class of diseases for which it is developing a treatment.
NEW YORK (GenomeWeb) – Officials from Alnylam Pharmaceuticals last week provided updates on the two drug candidates from the company's flagship transthyretin-mediated amyloidosis program, stating that the intravenously delivered agent patisiran is proceeding toward a possible market approval in t
Officials from Alnylam Pharmaceuticals last week provided updates on the company’s pipeline programs, highlighting efforts involving its GalNAc conjugation technology, which enables subcutaneous siRNA delivery, and a fledgling effort in complement-mediated diseases.
While RNAi drug developers continue to pursue major indications such as cancer and hepatitis C, a number of these companies are looking to niche diseases as good opportunities to apply the gene-silencing technology.
Alnylam Pharmaceuticals last week released preclinical data demonstrating proof of concept for its porphyria treatment ALN-AS1.