CARLSBAD, Calif., Oct. 23, 2018 /PRNewswire/ -- Ionis
Pharmaceuticals, Inc (NASDAQ: IONS), the leader in antisense
therapeutics, today announced that C. Frank
Bennett, Ph.D., senior vice president of research and
franchise leader for neurological programs at Ionis, has received
the Hereditary Disease Foundation's (HDF) 2018 Leslie Gehry Brenner
Prize for Innovation in Science. Dr. Bennett was honored for his
leadership and continued commitment to developing antisense
therapies for Huntington's disease (HD), a rare, progressive,
neurodegenerative disorder resulting in deterioration in mental
abilities and physical control. IONIS-HTTRx is designed
to treat patients with HD and is the first and only drug to
demonstrate reduction of neurotoxic mutant huntingtin protein, the
underlying cause of HD, in patients.
The Leslie Gehry Brenner Prize for Innovation in Science award
is given to a HD researcher who embodies qualities of inventiveness
and imagination in science. Since its creation in 1967, the
foundation's mission has been to fund innovative research towards
curing HD and impacting other brain disorders. The Foundation
created the Leslie Gehry Brenner Prize for Innovation in Science to
honor the memory of HDF founder Frank
Gehry's daughter.
"I am honored and grateful to be recognized by the Hereditary
Disease Foundation and the Gehry family," said Dr. Bennett. "For
nearly 30 years, our technology and learnings from developing drugs
for diseases like Huntington's have paved the way for new potential
therapies for diseases where no therapeutic approaches exist. I am
proud of these achievements, but they would not have been possible
without the vision and leadership of Dr. Stanley T. Crooke, founder of Ionis
Pharmaceuticals, and the commitment of my colleagues to bringing
new antisense therapies to patients living with unmet medical
needs."
Nancy Wexler, President of the
Hereditary Disease Foundation, said "We are thrilled to recognize
Dr. Frank Bennett for his innovative
work on IONIS-HTTRx for patients with Huntington's.
Frank's accomplishments honor the spirit and memory of Leslie Gehry by embodying originality,
spontaneity, precision and rigor – all critical attributes in a
scientist."
Dr. Bennett's development of an antisense technology therapy for
HD is being leveraged to develop antisense drugs for other
neurological diseases, such as Alzheimer's disease and Parkinson's
disease. Demonstrating that it is possible to treat HD by using
antisense to target the messenger RNA from a specific gene would
provide a proof-of-principle for researchers studying other
neurodegenerative conditions, encouraging the development of
similar treatments for many more patients.
As announced on Oct. 17, Dr.
Bennet is also a recipient of the 2019 Breakthrough Prize in Life
Sciences. This award acknowledges the innovation behind
SPINRAZA® (nusinersen), the first and only approved
treatment for patients with spinal muscular atrophy, a devastating
neurodegenerative disease that is the leading genetic cause of
infant death. The Breakthrough Prize honors the world's top
scientists who have made transformative advances toward
understanding living systems and extending human life, with one
prize dedicated to work that contributes to understanding of
neurological diseases. Dr. Bennett and collaborator Adrian Krainer, Ph.D., of Cold Spring Harbor
Laboratory were co-recipients of this award.
About Antisense Technology
The instructions for
making a protein are transcribed from a gene, or DNA, into a
different genetic molecule called messenger RNA (mRNA). This
process starts with the partial uncoiling of the two complementary
strands of the DNA. One strand acts as a template and information
stored in the DNA template strand is copied into a complementary
RNA. Messenger RNA, or mRNA, are mature, fully processed RNA that
code for proteins. Ribosomes, the cell's factories for
manufacturing proteins, translate mRNA into proteins.
The mRNA sequence that carries the information for protein
production is called the 'sense' strand. The complementary
nucleotide chain that binds specifically to the mRNA sense strand
is referred to as the "antisense" strand. Information contained in
mRNA can be used to design chemical structures called antisense
oligonucleotides (ASOs) or antisense drugs, which resemble DNA and
RNA and are the complement of RNA.
Antisense drugs bind with high selectivity to the mRNA they are
designed to target and interrupt the cell's protein production
process by preventing the mRNA instructions from reaching the
ribosome, thus inhibiting the production of the protein. Antisense
drugs can also be designed to increase protein production for
diseases caused by the lack of a particular protein or can modify
the processing, or splicing, of the mRNA, which can alter the
composition of the protein.
About Ionis Pharmaceuticals, Inc.
As the leader in
RNA-targeted drug discovery and development, Ionis has created an
efficient, broadly applicable, proprietary antisense technology
platform with the potential to treat diseases where no other
therapeutic approaches have proven effective. Our drug discovery
platform has served as a springboard for actionable promise and
realized hope for patients with unmet needs – such as children and
adults with spinal muscular atrophy (SMA). We created
SPINRAZA® (nusinersen)* and are proud to have brought
new hope to the SMA community by developing the first and only
approved treatment for this disease.
Our sights are set on all the patients we have yet to reach with
a pipeline of more than 40 drugs with the potential to treat
patients with cardiovascular disease, rare diseases, neurological
diseases, infectious diseases and cancer. We created TEGSEDI™
(inotersen) the world's first RNA-targeted therapeutic approved for
the treatment of polyneuropathy of hereditary transthyretin (TTR)
amyloidosis (ATTR) in adult patients that our affiliate Akcea
Therapeutics is commercializing. Together with Akcea, we are also
bringing new medicines to patients with cardiometabolic lipid
disorders.
To learn more about Ionis follow us on twitter @ionispharma or
visit http://ir.ionispharma.com/.
*Spinraza is marketed by Biogen.
Ionis' Forward-looking Statement
This press release
includes forward-looking statements regarding the therapeutic and
commercial potential of Ionis' technologies and products in
development, including SPINRAZA® and TEGSEDI™
(inotersen). Any statement describing Ionis' 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 drugs. Ionis' forward-looking
statements also involve assumptions that, if they never materialize
or prove correct, could cause its results to differ materially from
those expressed or implied by such forward-looking statements.
Although Ionis' forward-looking statements reflect the good faith
judgment of its management, these statements are based only on
facts and factors currently known by Ionis. As a result, you are
cautioned not to rely on these forward-looking statements. These
and other risks concerning Ionis' programs are described in
additional detail in Ionis' annual report on Form 10-K for the year
ended December 31, 2017, and most
recent Form 10-Q quarterly filing, which are on file with the SEC.
Copies of this and other documents are available from the
Company.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals™ is a trademark of Ionis
Pharmaceuticals, Inc. Akcea Therapeutics™ is a trademark
of Akcea Therapeutics, Inc. TEGSEDI™ is a trademark of
Akcea Therapeutics, Inc. SPINRAZA® is a registered
trademark of Biogen.
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SOURCE Ionis Pharmaceuticals, Inc.