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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
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September 18, 2014
07:49 EDTALNYAlnylam resumed with an Outperform at Leerink
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September 16, 2014
08:02 EDTALNYAlnylam presents ALN-CC5 pre-clinical data
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September 15, 2014
08:01 EDTALNYAlnylam names Karen Anderson as SVP, Chief Human Resources Officer
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07:21 EDTALNYHeart Failure Society of America to hold annual meeting
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September 12, 2014
08:02 EDTALNYAlnylam broadens pipeline with ALN-AGT
Alnylam Pharmaceuticals announced that it is broadening its pipeline with ALN-AGT. In a poster presented at the American Heart Association’s High Blood Pressure Research 2014 Scientific Sessions, Alnylam and collaborators at the Charite Universitatsmedizin in Berlin presented results of an ALN-AGT lead molecule in an established preeclamptic rodent model. The study showed that administration of ALN-AGT resulted in knockdown of maternal AGT in the liver without detectable evidence of fetal drug exposure, significantly improved pregnancy-related hypertension, ameliorated preeclamptic sequelae in the mother such as proteinuria, and improved fetal outcomes.

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