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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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Aug
28
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This webinar will provide an overview of alternatives to the popular Cas9 nuclease used in CRISPR gene editing.