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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.


Clinical and Pre-Clinical Data on RNAi Therapeutics at 10th Oligonucleotide Therapeutics Society Meeting

Clinical and Pre-Clinical Data on RNAi Therapeutics at 10th Oligonucleotide Therapeutics Society Meeting

We presented new data from multiple clinical and pre-clinical studies at the 10th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held October 12 – 15, 2014 in San Diego.  Among multiple presentations, we presented additional data from our Phase 1 trial with ALN-TTRsc showing rapid, dose-dependent, stable, and durable knockdown of serum TTR of up to 96.2%.  In addition, we presented pre-clinical data from a new program, ALN-GO1, an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of primary hyperoxaluria type 1 (PH1), showing efficacy in rodent disease models.  Finally, we presented new pre-clinical research demonstrating that delivery of Enhanced Stabilization Chemistry (ESC)-GalNAc-siRNA conjugates to the lung achieves similar plasma exposure, efficacy, and duration of liver gene silencing as achieved by subcutaneous delivery.  This finding opens up the possibility for needle-less administration of RNAi therapeutics via inhalation for knockdown of liver disease genes.





Six-Month Clinical Data from Patisiran Phase 2 Open-Label Extension (OLE) Study

Six-Month Clinical Data from Patisiran Phase 2 Open-Label Extension (OLE) Study

We announced six-month clinical data from our ongoing Phase 2 open-label extension (OLE) study with patisiran (ALN-TTR02) for the treatment of transthyretin (TTR)-mediated amyloidosis (ATTR) in patients with familial amyloidotic polyneuropathy (FAP).  Data were presented at the American Neurological Association’s 2014 Annual Meeting held October 12 – 14, 2014 in Baltimore.  Results showed a mean 0.95 point decrease in modified Neuropathy Impairment Score (mNIS+7) at six months in 19 patients.  This decrease in neuropathy progression compares favorably with the 7-to-10 point increase in mNIS+7 at six months that can be estimated from historical data sets in untreated FAP patients with similar baseline characteristics.  In addition, patisiran treatment achieved a sustained mean serum TTR knockdown at the 80% target level for over nine months, with an up to 89.6% knockdown achieved between doses.  Patisiran was found to be generally well tolerated in this study out to one year of therapy, with no drug-related serious adverse events to date, and all 27 patients enrolled in the study continue to receive drug treatment.   Infusion-related reactions were infrequent (14.8%), mild in severity, and did not result in any discontinuations.  All other reported adverse events were mild to moderate, and there were no clinically significant changes in liver function tests, renal function tests, or other laboratory or hematological parameters.




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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 ALN-CC5, a subcutaneously administered RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases, at the 25th International Complement Workshop held September 14 – 18, 2014, in Rio de Janeiro, Brazil. Data demonstrated potent and clamped C5 knockdown as well as robust inhibition of complement activity in non-human primates for up to 100 days with a subcutaneous, monthly dosing regimen. Further, in a rat model of membranous nephropathy, ALN-CC5 administration resulted in a significant reduction in proteinuria due to complement-mediated disease activity in the kidney.



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Pre-Clinical Data with ALN-AGT for Treatment of Hypertensive Disorders of Pregnancy, Including Preeclampsia

Pre-Clinical Data with ALN-AGT for Treatment of Hypertensive Disorders of Pregnancy, Including Preeclampsia

We announced a new program, ALN-AGT, an RNAi therapeutic targeting angiotensinogen (AGT) in development for the treatment of hypertensive disorders of pregnancy (HDP), including preeclampsia. Pre-clinical data were presented at the American Heart Association’s High Blood Pressure Research 2014 Scientific Sessions, held September 9 – 12, 2014, in San Francisco. Data from experiments in an established rat model of preeclampsia demonstrated that an RNAi therapeutic targeting AGT ameliorates the clinical sequelae of preeclampsia and improves outcomes for the fetus.  This treatment approach has the potential for selective delivery to the pregnant mother without fetal drug exposure, as our study confirmed undetectable siRNA levels in the fetus.




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