– Manoharan Patent Covers Antagomirs, a Chemical Class of microRNA
Inhibitors –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, and Regulus Therapeutics Inc., a leading microRNA
therapeutics company, announced today that the United States Patent and
Trademark Office (USPTO) has awarded a notice of allowance for the
Manoharan patent (application number 11/200,703), which covers several
chemical classes of anti-miRs, antisense oligonucleotide inhibitors of
microRNAs. This new patent in combination with Regulus’ existing
intellectual property estate broadly covers approaches to developing
microRNA-based therapeutics.
“Regulus Therapeutics is advancing a whole new frontier of
pharmaceutical research through the discovery of microRNA therapeutics,
which have the potential to block broad disease pathways as opposed to a
single target,” said Kleanthis G. Xanthopoulos, Ph.D., President and
Chief Executive Officer of Regulus Therapeutics. “Our access to the
pioneering research of our academic collaborators and Alnylam and Isis
on oligonucleotide-based therapeutics, as evidenced most recently by the
award of the Manoharan patent, has enabled Regulus to build a dominant
intellectual property estate for microRNA therapeutics. We believe this
is a strong foundation to advance our innovative approach to developing
novel medicines to address the needs of patients.”
microRNAs are small, non-coding RNAs that regulate a large number of
human genes; there are more than 700 microRNAs in the human genome that
play a role in controlling expression of over one-third of all human
genes. Abnormal expression and genetic mutation of microRNAs have been
linked to numerous diseases including cancer, cardiovascular disease,
metabolic disease, autoimmunity, and viral infection, amongst others. In
2005, Alnylam scientists and collaborators at The Rockefeller University
were the first to describe antagomirs, which are part of a larger class
of microRNA inhibitors known as anti-miRs (Krutzfeldt et al.,
(2005) Nature 438, 685-689). Anti-miRs were created to
selectively inhibit microRNAs expressed in a broad range of tissues and
to have pharmacological properties required for microRNA-based
therapeutics. This research has created new drug discovery strategies
focused on antagonizing microRNAs.
“We are pleased to have this important recognition from the USPTO
regarding our discovery of antagomirs, a new class of anti-miRs and a
novel invention in the field of microRNA-based therapeutics,” said
Muthiah Manoharan, Ph.D., Vice President for Drug Discovery at Alnylam.
“With this initial grant, we have received broad claims covering classes
of anti-miRs with certain important chemical features for optimal
delivery and in vivo pharmacology. Importantly, the inventive
nature of these novel compositions was allowed independent of any
specific microRNA sequence, thereby providing a broad set of patent
claims that add to the innovations Regulus can employ in designing novel
microRNA therapeutics, an entire new class of medicines.”
About microRNAs
microRNAs are a recently discovered class of genetically encoded
endogenous RNAs, approximately 20 nucleotides in length, that are
believed to regulate the expression of a large number of human genes.
microRNAs have been described as being involved in the regulation of
gene expression as part of physiology and development. The inappropriate
absence or presence of specific microRNAs in various cells has been
shown to be associated with specific human diseases including cancer,
viral infection, cardiovascular, metabolic disorders and
immuno-inflammatory disease.
