New User:

-or-
Username:
Password:
Forgot your password?

Stock Market & Financial Investment News

News Breaks
May 21, 2014
08:03 EDTALNYAlnylam announces publication of pre-clinical results with ALN-AS1
Alnylam Pharmaceuticals announced the publication in the Proceedings of the National Academy of Sciences of pre-clinical results with RNAi therapeutics targeting aminolevulinic acid synthase-1 for the treatment of hepatic porphyrias, including acute intermittent porphyria. In the paper, titled “RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice,” Alnylam scientists and collaborators at the Icahn School of Medicine at Mount Sinai in New York City documented results from pre-clinical models of the human disease showing that RNAi therapeutics targeting ALAS-1 can completely block the abnormal production of toxic intermediates of the heme biosynthesis pathway that cause the symptoms and disease pathology of AIP. This new paper provides proof of concept for an RNAi therapeutic for the treatment of AIP. “Our data in a mouse model of AIP, now published in PNAS, demonstrate that RNAi therapeutics targeting ALAS-1 can achieve potent, rapid, and durable suppression of the toxic heme biosynthesis intermediates that cause the symptoms and disease pathology of AIP. As such, these findings provide key pre-clinical proof-of-concept data for our ALN-AS1 program. We believe ALN-AS1 has the potential to be a transformative therapy for patients with hepatic porphyrias including AIP, an ultra-rare genetic disease with enormous unmet medical need,” said Rachel Meyers, Ph.D., Vice President, Research and RNAi Lead Development of Alnylam. “We are currently advancing our Development Candidate for ALN-AS1, which employs our Enhanced Stabilization Chemistry-GalNAc-conjugate technology. This technology enables subcutaneous dosing with improved potency and durability, and a wide therapeutic index, and is now clinically validated based on results from our hemophilia program. We are on track to file an Investigational New Drug application for ALN-AS1 in late 2014 or early 2015, and look forward to advancing this investigational medicine to patients.”
News For ALNY From The Last 14 Days
Check below for free stories on ALNY the last two weeks.
Sign up for a free trial to see the rest of the stories you've been missing.
August 27, 2014
08:02 EDTALNYAlnylam receives notice of allowance from USPTO for new patent
Subscribe for More Information
August 21, 2014
09:16 EDTALNYAlnylam to hold a roundtable webinar
Subscribe for More Information
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
Subscribe for More Information
August 14, 2014
07:24 EDTALNYAlnylam to hold a roundtable webinar
Subscribe for More Information

Sign up for a free trial to see the rest of the stories you've been missing.

I agree to the theflyonthewall.com disclaimer & terms of use