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September 23, 2013
07:12 EDTALNYAlnylam announces positive results from ALN-TTRsc Phase 1 trial
Alnylam Pharmaceuticals announced positive interim results from its Phase I clinical trial of ALN-TTRsc. Results show that ALN-TTRsc administration led to robust, consistent, and statistically significant 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 to be generally safe and well tolerated in this study. These human data are the first to be presented for Alnylam’s proprietary GalNAc-siRNA conjugate delivery platform, enabling subcutaneous dosing of RNAi therapeutics with a wide therapeutic index, and demonstrate human translation for this platform. Moreover, these results establish a new benchmark for consistent TTR knockdown of approximately 90% for RNA therapeutics in development for the treatment of ATTR. Upon completion of this Phase I trial, the company expects to start a pilot Phase II clinical study of ALN-TTRsc in FAC patients in late 2013 and, assuming positive results, plans to start a pivotal Phase III trial with ALN-TTRsc in FAC patients in 2014
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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.
August 19, 2014
15:50 EDTALNYAlnylam to hold a roundtable webinar
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