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Welcome to Alnylam

Welcome to Alnylam

Alnylam is developing an entirely new class of medicines based on the breakthrough discovery in biology known as RNA interference, or RNAi. Alnylam believes that RNAi therapeutics are a promising approach to silence disease-causing genes, and represent an opportunity to transform the treatment of many diseases.

Alnylam and Genzyme Form Transformational Alliance for RNAi Therapeutics as Genetic Medicines

Alnylam and Genzyme Form Transformational Alliance for RNAi Therapeutics as Genetic Medicines

In January 2014, Alnylam and Genzyme formed a transformational alliance with Genzyme for the development and commercialization of RNAi therapeutics as genetic medicines. This new alliance will allow for the accelerated and expanded development and commercialization of RNAi therapeutics across the world. Under the terms of this alliance, Alnylam retains broad product rights in North America and Western Europe, while Genzyme obtains the rights to Alnylam’s genetic medicines pipeline in the rest of the world plus co-development and co-commercialization rights or global rights to three programs. In addition, Genzyme will make a $700 million equity investment in Alnylam. Importantly, this new partnership significantly expands our balance sheet to over $1 billion in cash to increase our investment in new RNAi therapeutic programs, while securing a cash runway that we believe will allow us to develop and launch multiple products as breakthrough medicines for patients in need.



Alnylam Acquires Merck’s wholly owned subsidiary, Sirna Therapeutics

Alnylam Acquires Merck’s wholly owned subsidiary, Sirna Therapeutics

In January 2014, we entered an agreement with Merck, whereby we are acquiring Merck’s wholly owned subsidiary Sirna Therapeutics, Inc. This acquisition provides us with intellectual property and RNAi assets including pre-clinical therapeutic candidates, chemistry, siRNA-conjugate and other delivery technologies. We believe the acquisition of Sirna Therapeutics will complement and extend our own progress and continued focus on RNAi therapeutics, including siRNA-conjugate technologies. The RNAi assets and intellectual property that we are acquiring from Merck will strengthen our efforts to build a new class of medicines, advancing them to patients in need.


Summer RNAi Roundtable Series

We are hosting a series of online “RNAi Roundtables” during July and August, at which Alnylam scientists and key clinical collaborators will review recent progress from several “Alnylam 5×15” programs and discuss the related disease areas.

Next RNAi Roundtable:

Advances in Delivery of RNAi Therapeutics with Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA Conjugates
Tuesday, July 22: 11:00 a.m. – 12:00 p.m. ET

  • Rachel Meyers, Ph.D., Vice President, Research and RNAi Lead Development
  • Moderator: Laurence Reid, Ph.D., Senior Vice President and Chief Business Officer

To register and access the webcast for this event, please click here.

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Pre-Clinical Data with ALN-CC5 for Treatment of Complement-Mediated Diseases

Pre-Clinical Data with ALN-CC5 for Treatment of Complement-Mediated Diseases

We presented pre-clinical data with our Development Candidate for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases, at the 7th International Conference on Complement Therapeutics, held June 6 – 11, 2014, in Olympia, Greece.  These data demonstrate that ALN-CC5 led to an up to 98.7% knockdown of serum C5 and an up to 96.8% inhibition of complement activity in non-human primates (NHP) with weekly subcutaneous dose administration.



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Pre-Clinical Results with RNAi Therapeutics Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) Published in Proceedings of the National Academy of Sciences (PNAS)

Pre-Clinical Results with RNAi Therapeutics Targeting Aminolevulinic Acid Synthase-1 (ALAS-1) Published in Proceedings of the National Academy of Sciences (PNAS)

We have published pre-clinical results with RNAi therapeutics targeting aminolevulinic acid synthase-1 (ALAS-1) for the treatment of hepatic porphyrias in the Proceedings of the National Academy of Sciences (PNAS). The new paper highlights work that was done in collaboration with researchers at the Icahn School of Medicine at Mount Sinai in New York City. Data from studies performed in a mouse model of acute intermittent porphyria (AIP) demonstrate that RNAi therapeutics targeting ALAS-1 can achieve potent, rapid, and durable suppression of aminolevulinic acid (ALA) and porphobilinogen (PBG), the toxic heme biosynthesis intermediates that cause the symptoms and disease pathology of AIP.



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Positive Top-Line Data from Phase 1 Clinical Trial with ALN-AT3 for Treatment of Hemophilia and Rare Bleeding Disorders

Positive Top-Line Data from Phase 1 Clinical Trial with ALN-AT3 for Treatment of Hemophilia and Rare Bleeding Disorders

We presented positive top-line data from our Phase 1 clinical trial with ALN-AT3, an RNAi therapeutic targeting antithrombin (AT) in development for the treatment of hemophilia and rare bleeding disorders (RBD). In “Part A” of the Phase 1 study, we gave a single dose of drug to healthy volunteers to evaluate the drug’s safety and tolerability. Importantly, we had a dose escalation stopping rule at a maximum of 40% AT knockdown. Initial results, presented at the World Federation of Hemophilia (WFH) 2014 World Congress held May 11 – 15, 2014 in Melbourne, Australia, show that a single, low subcutaneous dose of ALN-AT3 at 0.03 mg/kg resulted in an up to 28-32% knockdown of AT at nadir relative to placebo (p <0.01 by ANOVA) and a temporally associated increase in peak thrombin generation (p <0.01). ALN-AT3 was found to be well tolerated with no significant adverse events reported.  With these results in hand, we have now proceeded to “Part B” of the Phase 1 study.



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