MENU
  • Alnylam Alnylam
  • Alnylam Alnylam
  • Alnylam Alnylam
  • Alnylam Alnylam
  • Alnylam Alnylam
  • Alnylam Alnylam
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.

Pipeline Growth Strategy for RNAi Therapeutics in Three Strategic Therapeutic Areas, or “STArs”

Pipeline Growth Strategy for RNAi Therapeutics in Three Strategic Therapeutic Areas, or “STArs”

In December 2014, we announced a pipeline growth strategy for development and commercialization of RNAi therapeutics across three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease.  The strategy builds on our progress in the advancement of RNAi therapeutics, including our “Enhanced Stabilization Chemistry” (ESC)-GalNAc conjugate technology, as a modular and reproducible platform for discovery of innovative medicines.  The three STArs will remain focused on liver-expressed and genetically validated or pathogen-derived disease targets, with biomarkers for assessment of clinical activity early in Phase 1 trials.  Across our three STArs, we believe that we can address major unmet needs in a wide range of diseases with high-impact, differentiated medicines, and continue to build what we believe to be one of the most robust pipelines in biotech.  By executing on this strategy, we believe we have the potential to make a meaningful difference in the lives of patients, and maximize value for shareholders.




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 R&D Day 2014 Webcast and Presentations

Alnylam R&D Day 2014 Webcast and Presentations

On December 12, we hosted an R&D Day in New York City. Alnylam management and key opinion leaders discussed the latest progress as well as plans for the future development of our RNAi therapeutics pipeline. At this event, we announced our pipeline growth strategy for development and commercialization of RNAi therapeutics across three Strategic Therapeutic Areas (STArs): Genetic Medicines, Cardio-metabolic Disease, and Hepatic Infectious Disease.



Read More

Positive Initial Phase 1 Clinical Results for ALN-AT3, in Development for the Treatment of Hemophilia and Rare Bleeding Disorders

Positive Initial Phase 1 Clinical Results for ALN-AT3, in Development for the Treatment of Hemophilia and Rare Bleeding Disorders

We presented positive initial Phase 1 data for ALN-AT3, an investigational RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders. Preliminary results, presented at the 56th Annual Meeting of the American Society of Hematology (ASH), showed that subcutaneous administration of ALN-AT3 given once weekly for three weeks at low doses of 15 (N=3) or 45 (N=1) micrograms/kg (mcg/kg) resulted in an up to 57% knockdown of AT in hemophilia subjects.  The effects of ALN-AT3 lasted for about 60 days after a single dose. ALN-AT3 was found to be well tolerated in both healthy volunteers and hemophilia subjects enrolled in the study.  These initial results show preliminary evidence for potency and durability of RNAi therapeutics at mcg/kg subcutaneous doses in human studies, and are the first clinical data to be reported for Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc conjugate technology.




Read More

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

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

We presented pre-clinical with ALN-CC5, an investigational RNAi therapeutic targeting complement component C5 for the treatment of complement-mediated diseases, at the American Society of Hematology (ASH) Meeting. Data showed an up to 99.2% knockdown of serum C5 and up to 96.2% inhibition of serum hemolytic activity in non-human primates (NHPs) with continued dosing for over seven months.



Read More

New Paper Documenting Alnylam’s Pioneering Discovery of GalNAc-Conjugated siRNA

New Paper Documenting Alnylam’s Pioneering Discovery of GalNAc-Conjugated siRNA

We have published an article in the Journal of the American Chemical Society describing the discovery of GalNAc-conjugated siRNA as a novel strategy for delivery of RNAi therapeutics. This publication as a JACS “Communication” documents the landmark discovery by our scientists of GalNAc-conjugates as a potent and durable approach for subcutaneous administration of RNAi therapeutics with a wide therapeutic index.



Read More