Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, and collaborators from the David H. Koch Institute
for Integrative Research at the Massachusetts Institute of Technology
(MIT) today announced the publication of new data in the journal Proceedings
of the National Academy of Sciences (PNAS) describing further
advancements in discovery and development of novel “lipidoid”
formulations for the systemic delivery of RNAi therapeutics. Lipidoids
are lipid-like materials discovered for the delivery of RNAi
therapeutics, and were originally described by Alnylam and MIT
collaborators (Akinc et al., Nature Biotechnology, 26: 561-569,
2008). In particular, the new research findings demonstrate the
discovery of new lipidoid materials that facilitate significantly
improved in vivo potency for RNAi therapeutics.
“We are excited by the delivery performance of these new formulations”
“We are very encouraged with the substantial progress we and our
collaborators have made with lipid nanoparticles (LNPs) based on novel
lipid-like materials such as lipidoids,” said Victor Kotelianski, M.D.,
Ph.D., D.Sc., Senior Vice President, Distinguished Alnylam Fellow. “To
our knowledge, these new LNP formulations facilitate endogenous liver
gene silencing at doses that are orders-of-magnitude lower than those
required by previously described siRNA delivery approaches, thereby
setting a new standard in potency for the systemic delivery of RNAi
therapeutics. In addition, the current study is the first to report on
the simultaneous and highly specific RNAi-mediated silencing of as many
as five liver targets in vivo, serving as proof of principle that
multiple genes involved in similar or divergent biological pathways can
be silenced with a single administration of a single drug product. From
a therapeutic standpoint, this could enable novel pharmaceutical
strategies, where silencing of multiple targets could achieve an
enhanced level of efficacy.”
The new pre-clinical data describe a formulation based on a lipidoid
known as “C12-200” that was shown to: