Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that its scientists and collaborators will present pre-clinical and clinical data with patisiran (ALN-TTR02) and ALN-TTRsc – RNAi therapeutics targeting transthyretin (TTR) for the treatment of TTR-mediated amyloidosis (ATTR) – at the XIV th International Symposium on Amyloidosis (ISA) being held April 27 – May 1, 2014 in Indianapolis, Indiana. Amongst other new data, the company presentations include results from a 283-patient natural history study of ATTR patients with familial amyloidotic polyneuropathy (FAP) that document the rate of disease progression.
Presentations and posters from Alnylam scientists and collaborators at the meeting include:
- an oral presentation titled “Neuropathy progression rate in patients with familial amyloidotic polyneuropathy” in the “ATTR: Diagnosis and pathogenesis” plenary session being held on Wednesday, April 30 from 11:30 a.m. – 1:00 p.m. ET;
- an oral presentation titled “Further analysis of phase II trial of patisiran, a novel RNAi therapeutic for the treatment of familial amyloidotic polyneuropathy” in the “ATTR: Prognosis and therapy” plenary session being held on Wednesday, April 30 from 2:30 p.m. – 4:30 p.m. ET; and,
- a poster titled “Preclinical Evaluation of RNAi Therapeutics for the Treatment of ATTR: An Update” on Wednesday, April 30.
About RNA Interference (RNAi)
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.