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April 28, 2014
08:05 EDTALNYAlnylam, collaborators present new clinical data for patisiran
Alnylam Pharmaceuticals and collaborators announced new clinical data for patisiran, an RNAi therapeutic targeting transthyretin in development for the treatment of TTR-mediated amyloidosis. These data are being presented at the International Symposium on Amyloidosis held April 27 – May 1 in Indianapolis, Indiana. First, the company presented updated Phase 2 results in patients with Familial Amyloidotic Polyneuropathy confirming robust TTR knockdown of up to 96% with a mean TTR knockdown of approximately 80%. Further, Alnylam presented preliminary results from the open-label extension study with patisiran in patients that were enrolled in the Phase 2 study. Preliminary results from the OLE study showed that multiple doses of patisiran achieved sustained knockdown of serum TTR protein levels at the 80% target level through 168 days. Moreover, OLE results showed a favorable tolerability profile with up to eight doses administered. Finally, the company presented results of a natural history, cross-sectional analysis study of 283 FAP patients aimed at measuring the rate of neuropathy progression and its correlation with disease severity. These results provide support for Alnylam’s Phase 3 APOLLO trial where patisiran is being evaluated for its potential efficacy and safety in the FAP indication.
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August 27, 2014
08:02 EDTALNYAlnylam receives notice of allowance from USPTO for new patent
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August 21, 2014
09:16 EDTALNYAlnylam to hold a roundtable webinar
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August 20, 2014
08:07 EDTALNYAlnylam receives USPTO notice of allowance for new patent
Alnylam Pharmaceuticals announced that the United States Patent and Trademark Office has issued a Notice of Allowance for claims in the Tuschl et al. patent application 13/725,262. The ’262 patent application includes newly allowed claims directed to compositions that mediate RNAi, and comprising a double-stranded molecule with up to 25 base pairs and at least one nucleotide analogue. Specifically, the newly allowed patent application broadly covers small interfering RNA molecules of various designs, including so-called “dicer substrate” RNAi triggers (Amarzguioui et al., Nat Protoc. 2006;1(2):508-17; Rose et al., Nucleic Acids Res. 2005 Jul 26;33(13):4140-56) and double-stranded, RNAi-mediating molecules containing moieties that include “unlocked nucleobase analogs” amongst other naturally or non-naturally occurring nucleotide analogues. The allowed claims of the Tuschl ’262 patent application, as well as other granted, Alnylam-owned or -licensed patents, in aggregate broadly cover compositions, methods, and uses of RNAi therapeutics.

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