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.

The US Food and Drug Administration has approved Kite Pharmaceuticals' CAR T-cell therapy for large B-cell lymphomas, the New York Times reports.

Kaiser Health News reports that gene therapies could cost more than a million dollars.

Worcester Polytechnic Institute researchers have received a grant to combine biology and computer science for high school students.

In Nature this week: variants associated with obsessive-compulsive disorder, review of key CRISPR enzymes, and more.