06.08.2008 12:00:00
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Alnylam Awarded Continued Funding Under U.S. Government Contract to Develop RNAi Therapeutics for Biological Threats
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi
therapeutics company, today announced that the National Institute of
Allergy and Infectious Diseases (NIAID), a component of the National
Institutes of Health (NIH), has committed to $7.5 million of continued
funding related to the 2006 contract for the development of a broad
spectrum RNAi anti-viral therapeutic against hemorrhagic fever virus,
including Ebola virus. Ebola virus can cause a severe, often fatal
infection, and poses a potential biological safety risk and bioterrorism
threat.
"We are pleased with the continued support of
the federal government for the development of an anti-viral RNAi
therapeutic for Ebola,” said Barry Greene,
President and Chief Operating Officer. "To
date, we have been granted more than $63 million in federal contracts
for Alnylam Biodefense, and we are looking forward to continuing our
work with the NIH to help strengthen our nation’s
capabilities to counter serious biological security threats.” "We are encouraged by the data we have seen
with the Ebola program. These data have demonstrated potent and specific in
vivo efficacy mediated by an RNAi mechanism,”
said Antonin de Fougerolles, Ph.D., Senior Director of Research at
Alnylam. "This funding from the NIAID allows
us to continue to develop our technology as we advance our pipeline
programs.”
In September 2006, Alnylam was awarded a federal contract (No.
HHSN266200600012C) providing the company with up to $23 million in
funding over a four-year period to develop small interfering RNAs
(siRNAs), the molecules that mediate RNAi, as anti-viral drugs targeting
Ebola virus. To date, the government has committed to paying Alnylam up
to $14.2 million for the first two years of the contract. As a result of
the continued progress of this program, the government has committed to
fund an additional $7.5 million over year three of the contract.
At the RNAi Keystone Symposia in March 2008, Alnylam presented
pre-clinical data from this program utilizing an optimized RNAi
therapeutic formulated in a lipid particle for systemic delivery. These
data showed that potent siRNAs with in vitro anti-viral activity
were identified against all genes in the Ebola genome. A greater than 95
percent decrease in viral titer was seen when an RNAi therapeutic
targeting one of these genes, VP35, was administered to mice infected
with Ebola. Moreover, the VP35 siRNA, as compared with a control
non-specific siRNA, protected both mice and guinea pigs from lethal
Ebola infection. This work was done in collaboration with Tekmira
Pharmaceuticals Corporation using their lipid particle delivery
formulation technology.
As part of a public sector-private sector partnership with its Ebola
program, Alnylam is working with the United States Army Medical Research
Institute of Infectious Diseases (USAMRIID), an organization which is
uniquely experienced in the handling, safety, and security requirements
of specialized biological agents. Alnylam produces drug candidates which
are then sent to USAMRIID for in vitro and in vivo testing
against Ebola virus.
About Alnylam BioDefense™
Alnylam Biodefense was established to build a robust platform for
developing RNAi therapeutics targeting threats of bioterrorism. Funding
for the company’s Ebola virus, pandemic flu,
and viral hemorrhagic fever programs represents an example of broad
public health and federal interest in the potential of RNAi technology
to treat and prevent disease from these, and other serious and
life-threatening viruses. Government funding extends Alnylam’s
anti-viral pipeline, which includes programs for treatment of:
respiratory syncytial virus (RSV) infection with ALN-RSV01, which is in
Phase II human clinical trials; pandemic influenza with ALN-FLU01 in a
partnership with Novartis; and JC virus in a partnership with Biogen
Idec.
About RNA Interference (RNAi)
RNAi (RNA interference) is a revolution in biology, representing a
breakthrough in understanding how genes are turned on and off in cells,
and a completely new approach to drug discovery and development. Its
discovery has been heralded as "a major
scientific breakthrough that happens once every decade or so,”
and represents one of the most promising and rapidly advancing frontiers
in biology and drug discovery today which was awarded the 2006 Nobel
Prize for Physiology or Medicine. RNAi is a natural process of gene
silencing that occurs in organisms ranging from plants to mammals. By
harnessing the natural biological process of RNAi occurring in our
cells, the creation of a major new class of medicines, known as RNAi
therapeutics, is on the horizon. RNAi therapeutics target the cause of
diseases by potently silencing specific messenger RNAs (mRNAs), thereby
preventing disease-causing proteins from being made. RNAi therapeutics
have the potential to treat disease and help patients in a fundamentally
new way.
About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel therapeutics
based on RNA interference, or RNAi. The company is applying its
therapeutic expertise in RNAi to address significant medical needs, many
of which cannot effectively be addressed with small molecules or
antibodies, the current major classes of drugs. Alnylam is leading the
translation of RNAi as a new class of innovative medicines with
peer-reviewed research efforts published in the world’s
top scientific journals including Nature, Nature Medicine,
and Cell. The company is leveraging these capabilities to build a
broad pipeline of RNAi therapeutics; its most advanced program is in
Phase II human clinical trials for the treatment of respiratory
syncytial virus (RSV) infection. In addition, the company is developing
RNAi therapeutics for the treatment of a wide range of disease areas,
including hypercholesterolemia, liver cancers, and Huntington’s
disease. The company’s leadership position in
fundamental patents, technology, and know-how relating to RNAi has
enabled it to form major alliances with leading companies including
Medtronic, Novartis, Biogen Idec, Roche, Takeda, and Kyowa Hakko. To
reflect its outlook for key scientific, clinical, and business
initiatives, Alnylam has established "RNAi
2010” which includes the company’s
plan to significantly expand the scope of delivery solutions for RNAi
therapeutics, have four or more programs in clinical development, and to
form four or more new major business collaborations, all by the end of
2010. Alnylam is a joint owner of Regulus Therapeutics LLC, a joint
venture focused on the discovery, development, and commercialization of
microRNA therapeutics. Founded in 2002, Alnylam maintains headquarters
in Cambridge, Massachusetts. For more information, visit www.alnylam.com.
Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s
future expectations, plans and prospects, including without limitation
our plans with respect to the discovery and development of an RNAi
therapeutic for Ebola virus, constitute forward-looking statements for
the purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995. Actual results may differ materially from
those indicated by these forward-looking statements as a result of
various important factors, including risks related to: Alnylam’s
approach to discover and develop novel drugs, which is unproven and may
never lead to marketable products; obtaining, maintaining and protecting
intellectual property; Alnylam’s ability to
enforce its patents against infringers and to defend its patent
portfolio against challenges from third parties; Alnylam’s
ability to obtain additional funding to support its business activities,
including but not limited to additional funding under government
contracts; Alnylam’s dependence on third
parties for development, manufacture, marketing, sales and distribution
of products; obtaining regulatory approval for products; competition
from others using technology similar to Alnylam’s
and others developing products for similar uses; Alnylam’s
dependence on collaborators; and Alnylam’s
short operating history; as well as those risks more fully discussed in
the "Risk Factors”
section of its most recent annual report on Form 10-Q on file with the
Securities and Exchange Commission. In addition, any forward-looking
statements represent Alnylam’s views only as
of today and should not be relied upon as representing its views as of
any subsequent date. Alnylam does not assume any obligation to update
any forward-looking statements.
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