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.

Biomedical research projects are generating a ton of data that still needs to be analyzed, NPR reports.

Theranos is retiring some of its board members, including Henry Kissinger and George Shultz, Business Insider reports.

The heads of 29 scientific societies and some 2,300 researchers call on President-elect Donald Trump to rely on and support science in two separate letters.

In Science this week: genetically modified flu virus could be key to new live vaccines, and more.