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Unformatted text preview: ns and RNAs
RNAs MirRNA Therapeutics COMPANY OVERVIEW: FIRST IN HUMAN
CLINICAL TRIALS for a chemically optimized siRNA
— a major milestone in the development of RNAibased therapeutics. Sirna-027, for the treatment of the
wet-form of age-related macular degeneration (AMD),
a condition in which the cells of the macula lutea
degenerate, resulting in blurred vision and ultimately
blindness. The macula is a small area at the back of
the eye, near the center of the retina, at which visual
perception is most acute. AMD occurs most often with
increasing age (age-related macular degeneration or
AMD), but there are hereditary forms of the disease
that affect children and teenagers as well. ADM is seen
in adults 15 million people in the United States over 60
years of age.
Our therapeutic programs encompass a broad range of
therapeutic areas including: Anti-Viral, Dermatology,
Metabolism, Neurology, Ocular, Oncology, and
Respiratory. Specific preclinical and clinical programs
include: viral hepatitis, an infection of the liver that is
caused by a variety of RNA viruses (hepatitis A virus,
hepatitis B virus, hepatitis C virus, etc.).(HBV & HCV),
hair removal, asthma and COPD, diabetes,
Huntington’s disease, and oncology. These programs
represent large market opportunities and significant
unmet medical needs. BIS101001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez ©2013
BIS101001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez 17 Flavr Savr Tomatoe and Antisense*
Antisense RNA methods have been used for
commercial food production. The Flavr Savr
tomato was developed by Calgene Inc. of Davis,
California in 1991 and was approved by the U.S.
FDA in 1994. The tomato was the first whole food
created by biotechnology that was evaluated by
the FDA. One of the problems associated with
tomato farming is that the fruit must be picked
while still green in order to be shipped to market
without being crushed. The enzyme that causes
softening in tomatoes is polygalacturonase (PG).
This enzyme breaks down pectin as the tomato
ripens, leading to a softer fruit. Calgene
suppressed the expression of the gene encoding
PG by introducing a gene encoding the antisense
strand of the mRNA. When the introduced gene
was expressed, the antisense strand bound to the
PG mRNA, suppressing the translation of the
enzyme. The Flavr Savr tomatoes therefore had
low PG levels and remained firmer when ripe.
This meant the Flavr Savr tomatoes could be
ripen on the vine and then be shipped to market.
Although the Flavr Savr tomatoes were approved
for sale in the U.S., production problems and
consumer wariness stopped the production of this
fruit in 1997.
BIS101001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez ©2013
BIS101001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez 18 Summary
Transcription can be “cosuppressed” or “knocked down” by
small interfering RNAs (siRNAs), complementary to the mRNA
Although it was thought that a single stranded anti-sense RNA
was responsible for cosuppression, we now know that a dsRNA
Extensive processing of the dsRNA is needed to produce the
siRNA. siRNA can inhibit gene expression by promoting
ribonuclease degradation or blocking the translation of the
This whole process is called RNA interference (RNAi) and it
has great potential for health and agriculture.
has BIS101001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez ©2013
BIS101001, Spring 2013—Genes and Gene Expression, R.L. Rodriguez 19...
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