Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, announced today the presentation of new
pre-clinical data from its hypercholesterolemia program, performed in
collaboration with scientists at the University of Texas Southwestern
Medical Center at Dallas. The data were presented at the PCSK9
Conference: From Gene to Therapeutics held in Nantes, France from March
11-13, 2010. Alnylam has an ongoing development program focused on using
RNAi therapeutics targeting proprotein convertase subtilisin/kexin type
9, or PCSK9, as a novel strategy for reducing LDL (or “bad”)
cholesterol. The new data demonstrated durable reductions of cholesterol
levels in both rodents and non-human primates with an RNAi therapeutic
targeting PCSK9 using second generation lipid nanoparticle (LNP)
formulations. Further, data also showed the ability to utilize siRNA
combination approaches to achieve efficient silencing of separate and
distinct genes to achieve cholesterol lowering.
“We are very encouraged by the progress we have made in our development
efforts for an RNAi therapeutic targeting PCSK9 for the treatment of
hypercholesterolemia. With an RNAi approach, both intracellular and
extracellular PCSK9 levels can be reduced, thereby replicating the human
genetics”
“We are very encouraged by the progress we have made in our development
efforts for an RNAi therapeutic targeting PCSK9 for the treatment of
hypercholesterolemia. With an RNAi approach, both intracellular and
extracellular PCSK9 levels can be reduced, thereby replicating the human
genetics,” said Kevin Fitzgerald, Ph.D., Director of Research at
Alnylam. “In addition to the markedly improved potency we demonstrated
last year with second generation LNPs, we are now able to show that
PCSK9 silencing effects are durable over a remarkably extended period of
time. Further, these new data highlight our ability to investigate
liver-specific silencing of target combinations in order to develop
novel RNAi therapeutics for the treatment of hypercholesterolemia, as
well as potentially other metabolic disorders.”
The new in vivo research findings presented at this meeting were
performed using a systemically delivered RNAi therapeutic targeting
PCSK9 and formulated in a second generation LNP, developed in
collaboration with Tekmira Pharmaceuticals Corporation, The University
of British Columbia, and AlCana Technologies, Inc. Data from these
studies include: