NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals this week unveiled new Phase II data on its transthyretin-mediated amyloidosis (ATTR) therapy patisiran, which showed that the drug is capable of knocking down its disease-causing protein target by about 80 percent in patients — in line with previous clinical findings.

Release of the findings come as the company moves ahead with a recently initiated Phase III trial of patisiran in patients with a manifestation of ATTR known as familial amyloidotic polyneuropathy that is characterized by damage to the peripheral nervous system.

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Researchers hope to tease out the signature effects that different carcinogens leave on the genome to determine their contributions to disease, Mosaic reports.

The Wall Street Journal looks into the cost of new gene therapies.

An Imperial College London-led team reports that it was able to use a gene drive to control a population of lab mosquitos.

In PNAS this week: genomic effects of silver fox domestication, limited effect of mitochondrial mutations on aging in fruit flies, and more.

Nov
05
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Sophia Genetics

With the Next Generation Sequencing (NGS), genomes sequencing has been democratized over the last decades with the detection of genomic alterations, thus replacing Sanger sequencing.