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.

Technology Review reports that researchers in the US have used CRISPR to modify a number of human embryos.

Plant researchers plan to sequence some 10,000 samples that represent the major plant clades, ScienceInsider reports.

By introducing genes from butterfly peas and Canterbury bells, researchers in Japan have developed a blue chrysanthemum, according to NPR.

In Nature this week: a Danish reference genome, and more.