porphyria | GenomeWeb

porphyria

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.

In Nucleic Acids Research this week: nanopore sequencing workflow to detect antibiotic resistance in gut microbes, TSSPlant tool, and more.

Because gene-edited organisms can cross borders, Gizmodo wonders whether there should be an international body to govern their use.

HHS Secretary nominee Tom Price is to go in front of the Senate Committee on Health, Education, Labor and Pensions today, NPR's Morning Edition reports.

Prior to being closed, Theranos' Arizona lab failed an inspection by regulators, according to the Wall Street Journal.