Scientific Leadership

These are exciting times for RNAi therapeutics.  Important scientific and clinical advancements are being made at a very rapid pace. The discovery of RNAi has been widely acknowledged as a major breakthrough in biology, and the belief that this technology has the potential to make a broad and significant impact in medicine is stronger than ever.   Alnylam is leading the effort in translating the science of RNAi into a sustainable product pipeline of innovative medicines.

At Alnylam, we are unwavering in our commitment to scientific excellence, and we are driven by our dedication to the highest scientific standards. A testament to this underlying tenet at Alnylam is the publication of our research in world-leading, peer-reviewed scientific journals, such as Nature, Nature Medicine, Nature Biotechnology, Cell, New England Journal of Medicine, and The Lancet, amongst others.  We believe our numerous publications – numbering over 200 to date – and collaborations with some of the world’s best research labs are important measures of our position as the scientific and clinical leaders in the RNAi
field. Our scientific and clinical papers also serve to demonstrate to leading academic scientists, clinicians, and pharmaceutical executives the potential impact of our discoveries and the opportunity for RNAi therapeutics to become a new therapeutic approach in medicine, in addition to Alnylam’s commitment to and leadership in translating and advancing the field.

Novel Method for Detection of Tissue-Specific, RNAi-Mediated Gene Silencing Published in “RNA”

Novel Method for Detection of Tissue-Specific, RNAi-Mediated Gene Silencing Published in “RNA”

We have published a paper in the journal RNA describing a novel method that enables the quantification of circulating messenger RNA (mRNA) and micro-RNA (miRNA) as a way of monitoring tissue-specific RNA silencing, and demonstrating RNAi mechanism of action.  The method, called circulating Extracellular RNA Detection (cERD), could have broad applicability in clinical studies since it allows for monitoring of tissue-specific mRNA levels using a non-invasive technique.



Read More

Positive Phase I Clinical Trial Results with ALN-PCS Published in “The Lancet”

Positive Phase I Clinical Trial Results with ALN-PCS Published in “The Lancet”

We have published complete study results from our Phase I trial with ALN-PCS in The Lancet. As reported in the paper, ALN-PCS administration resulted in a rapid, dose-dependent reduction in plasma PCSK9 of up to 84% relative to baseline and placebo, with a corresponding reduction in serum levels of low-density lipoprotein cholesterol (LDL-C) – or “bad” cholesterol – of up to 57% relative to baseline and placebo. The knockdown of PCSK9 and lowering of LDL-C were also found to be durable, with effects lasting for weeks after a single dose.  In addition, ALN-PCS was shown to be generally safe and well tolerated in this Phase I study and there were no serious adverse events related to study drug administration.  This new paper documents the first human proof of concept for an RNAi therapeutic impacting a clinically validated endpoint, namely LDL-C.



Read More

Positive Clinical Trial Results with ALN-TTR01 and ALN-TTR02 Published in the “New England Journal of Medicine”

Positive Clinical Trial Results with ALN-TTR01 and ALN-TTR02 Published in the “New England Journal of Medicine”

We have published complete study results from Phase I trials with ALN-TTR01 and ALN-TTR02 in the New England Journal of Medicine. RNAi therapeutics targeting transthyretin (TTR) achieved rapid, dose-dependent, durable, and specific knockdown of TTR, the disease-causing protein in TTR-mediated amyloidosis (ATTR). The Phase I studies of ALN-TTR01 and ALN-TTR02 demonstrate key human proof of concept for RNAi therapeutics targeting TTR as well as liver-expressed target genes in general.



Read More