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RNAi Therapeutic Successful for Treatment of SARS Respiratory Virus in Rhesus Macaque

    ROCKVILLE, Md., Sept. 12 /PRNewswire/ -- Intradigm Corporation, a leader
in RNAi therapeutics, announced today its collaboration with Chinese academic
and biotech groups has achieved clear evidence of prophylactic and therapeutic
effects of siRNA (small interfering RNA) agents in Rhesus monkey to treat SARS
coronavirus (SCV) respiratory infection.  The results are published as a
research article in the September 2005 issue of Nature Medicine.
    The published results show that potent siRNA inhibitors against SCV,
validated in previous in vitro study (Antiviral Therapy 9:365-374), are active
in vivo using a mouse model system and subsequently in a rhesus macaque
(Macaca mulatta) SARS disease model, using a clinically viable delivery
formulation by intranasal administration.  The studies in macaques included
two control groups and three treatment groups, using prophylactic, concurrent
or early post exposure regimens, with a large number of animals in each group
for statistically meaningful results.  A pair of siRNA oligos, SC2 and SC5,
targeting two different genes in the virus genome were administrated at 10 to
40 mg/kg.  The numerous observations, including clinical SARS-like symptoms,
lung histopathology and levels of viral RNA, confirm achievement of an siRNA-
mediated inhibition of the respiratory virus and, importantly, no sign of
siRNA treatment-mediated toxicity or off target effects.  These results
provide strong support for clinical investigation.  Most importantly the
results clearly illustrate the prospects for siRNA therapeutics to address
unmet clinical need but also to dramatically reduce the time and cost of drug
development.  This capability is especially important in the critical area of
newly emerging respiratory viral infections.
    Nature Medicine also recognized the importance of the work in a news
release entitled "New treatment against SARS effective in monkeys," which
claimed that, "These results constitute the first successful therapeutic use
of siRNA in primates, and significantly boost up the potential of this tool to
prevent and treat SARS in people."
    Dr. Patrick Lu, a corresponding author of the published paper and
executive vice president of Intradigm pointed out, "The international
collaborative effort was critical to the success of this study.  Also, the
clinically viable delivery formulation and route of administration are keys to
achieve prophylactic and therapeutic efficacy of siRNA inhibitors in this non-
human primate study."  Dr. Martin Woodle, CSO of Intradigm, said, "The success
of this study illustrates key advantages of siRNA-based therapeutic agents,
including a massive reduction in discovery and development time for novel
targeted therapeutics, essential for combating many emerging infectious
diseases, and the coming revolution by combining siRNA sequences to allow
rapid development of multi-targeted therapeutics."
    "We are very excited by this Nature Medicine publication on the successful
primate study with our siRNA-based antiviral therapeutics, which is further
evidence that siRNA agents could become a very powerful drug modality for
treatment of various diseases. With results from this study, we are more
confident about our ongoing effort on ICS-283, an siRNA-based anticancer drug
in late stage of preclinical development, expecting reach to the clinic in mid
2006," said John Spears, Chairman and CEO of Intradigm.
    The study was conducted in collaboration and co-authored with scientists
from Sun Yat-Sen University, Top Genomics, Ltd. of Guangzhou, Institute of
Laboratory Animal Science, Chinese Academy of Medical Sciences & Peking Union
Medical College, Guangzhou Institute of Respiratory Diseases of Guangzhou
Medical College, Hong Kong University, in China, led by professors Nanshan
Zhong and Bao-jian Li.  This study is supported in part by Guangdong
Provincial and Guangzhou Municipal Governments, and China World Trade
Corporation.

    About Intradigm Corporation
    Intradigm Corporation is a privately held Delaware corporation,
established in 2001, that is developing therapeutic products based on RNAi
technology for cancer and other critical diseases.  The Company is developing
a portfolio of novel cancer therapeutics using its proprietary TargeTran(TM)
technology for systemic targeted delivery of siRNA, in addition to exploring
other therapeutic areas with partnerships.  Investors include Emerging
Technology Partners, Novartis Venture Fund and the State of Maryland's
Business and Economic Development Department.  Intradigm is located in the
Association for Entrepreneurial Sciences Inc. facilities in Rockville, MD.


SOURCE Intradigm Corporation




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Related links:
  • http://www.intradigm.com
    CONTACT:
    Dr. Martin Woodle of Intradigm Corporation,
    +1-301-984-0185 mwoodle@intradigm.com