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Alnylam
Pharmaceuticals, Inc.
ALNY, a
leading RNAi therapeutics company, announced today positive interim
results from its Phase I clinical trial of ALN-TTRsc, a subcutaneously
delivered RNAi therapeutic targeting the transthyretin (TTR) gene for
the treatment of TTR-mediated amyloidosis (ATTR). The data
are being presented today at the Heart Failure Society of America 17th
Annual Scientific Meeting being held September 22 – 25, 2013 in Orlando,
Fla. Results show that ALN-TTRsc administration led to robust,
consistent, and statistically significant (p<0.01) knockdown of serum
TTR protein levels of up to 94%. In addition, knockdown of TTR, the
disease causing protein in ATTR, was found to be rapid, dose dependent,
and durable. To date, ALN-TTRsc has been found
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