Regulus Therapeutics Inc. announced today that the United States Patent
and Trademark Office (USPTO) has allowed claims in U.S. Application
Serial No. 11/977,506 covering methods of antagonizing miR-181a to
regulate immune response. This patent is owned by Stanford University
and licensed exclusively to Regulus. miR-181a has been shown to regulate
the response of immune cells, such as T lymphocytes, to specific stimuli
and modulation of miR-181a could lead to a novel treatment of
inflammatory disease.
"We are pleased with the decision of the USPTO to allow this first
application based on the Li et al. patent filing. We are applying our
expertise in microRNA biology and nucleic acid chemistry to develop
novel medicines for treating inflammatory disease, and this new
allowance will expand the scope of our exclusivity surrounding the
anti-miR approach to miR-181a,” said Garry E. Menzel, Ph.D., Executive
Vice President of Corporate Development and Finance of Regulus
Therapeutics. "More broadly, we continue to build a robust patent
portfolio that supports our strategy of developing and commercializing
high-impact microRNA-based therapeutics in multiple disease areas.”
Data published in 2007 by scientists at Stanford University and Alnylam
Pharmaceuticals in the journal Cell [Li QJ et al. 2007
Apr. 6;129(1):147-61] demonstrated that modulation of miR-181a levels in
an immune cell modified the sensitivity of the cell to specific stimuli.
Increased expression of miR-181a led to an increase in immune cell
response to an inflammatory stimulus. Conversely, decreasing levels of
miR-181a in immune cells de-sensitized the cell to the stimulus.
Consistent with this observation, using an anti-miR to antagonize
miR-181a function reduced immune cell response to a stimulus.
The newly allowed claims cover methods of modulating miR-181a in T
cells, to raise the T cell receptor signaling threshold, and decrease
the sensitivity of T cells to antigen stimulation. The claims cover the
use of a broad class of anti-miRs targeting miR-181a, including
anti-miRs having varying lengths and chemical modifications.
About microRNAs
The discovery of microRNA in humans is one of the most exciting
scientific breakthroughs in the last decade. microRNAs are small RNA
molecules, typically 20 to 25 nucleotides in length, that do not encode
proteins but instead regulate gene expression. Nearly 700 microRNAs have
been identified in the human genome, and more than one-third of all
human genes are believed to be regulated by microRNAs. As a single
microRNA can regulate entire networks of genes, these new molecules are
considered the master regulators of the genome. microRNAs have been
shown to play an integral role in numerous biological processes
including the immune response, cell-cycle control, metabolism, viral
replication, stem cell differentiation and human development. Many
microRNAs are conserved across multiple species indicating the
evolutionary importance of these molecules as modulators of critical
biological pathways. Indeed, microRNA expression or function has been
shown to be significantly altered in many disease states, including
cancer, heart failure and viral infections. Targeting microRNAs opens
the possibility of a novel class of therapeutics and a unique approach
to treating disease by modulating entire biological pathways.
About Regulus Therapeutics Inc.
Regulus Therapeutics is a biopharmaceutical company leading the
discovery and development of innovative new medicines based on
microRNAs. Regulus is targeting microRNAs as a new class of therapeutics
by working with a broad network of academic collaborators and leveraging
oligonucleotide drug discovery and development expertise from its
founding companies Alnylam Pharmaceuticals (Nasdaq:ALNY) and Isis
Pharmaceuticals (Nasdaq:ISIS). Regulus is advancing microRNA
therapeutics towards the clinic in several areas including hepatitis C
infection, cardiovascular disease, fibrosis, oncology,
immuno-inflammatory diseases, and metabolic diseases. Regulus’
intellectual property estate contains both the fundamental and core
patents in the field as well as over 600 patents and more than 300
pending patent applications pertaining primarily to chemical
modifications of oligonucleotides targeting microRNAs for therapeutic
applications. In 2008, Regulus entered into a major alliance with
GlaxoSmithKline to discover and develop microRNA therapeutics for
immuno-inflammatory diseases. In 2010, Regulus entered into a new
collaboration with GlaxoSmithKline to develop and commercialize microRNA
therapeutics targeting microRNA-122 for the treatment of Hepatitis C
Viral infection. For more information, visit www.regulusrx.com.
Forward-Looking Statements
This press release includes forward-looking statements regarding the
future therapeutic and commercial potential of Isis’, Alnylam’s and
Regulus’ business plans, technologies and intellectual property related
to microRNA therapeutics being discovered and developed by Regulus,
including statements regarding the therapeutic potential of targeting
miR-181a. Any statement describing Isis’, Alnylam’s or Regulus’ goals,
expectations, financial or other projections, intentions or beliefs is a
forward-looking statement and should be considered an at-risk statement.
Such statements are subject to certain risks and uncertainties,
particularly those inherent in the process of discovering, developing
and commercializing drugs that are safe and effective for use as human
therapeutics, and in the endeavor of building a business around such
products. Such parties’ forward-looking statements also involve
assumptions that, if they never materialize or prove correct, could
cause their results to differ materially from those expressed or implied
by such forward-looking statements. Although these forward-looking
statements reflect the good faith judgment of the management of each
such party, these statements are based only on facts and factors
currently known by Isis, Alnylam or Regulus, as the case may be. As a
result, you are cautioned not to rely on these forward-looking
statements. These and other risks concerning Regulus’, Alnylam’s, and
Isis’ programs are described in additional detail in Alnylam’s and Isis’
annual reports on Form 10-K for the year ended December 31, 2009. Copies
of these and other documents are available from Alnylam or Isis.
