In contrast to other classes of therapeutics such as proteins, antibodies and small molecules, RNAi does not require “drugable” domains on the molecular target as it is active
on more universal level – the level of nucleic acid. Furthermore, siRNAs constitutes a well defined chemical entity. Even different siRNAs molecules (sequences) against the same or
a different target gene will have similar ADME and toxicology / safety profiles in animals and humans. This implies that once the first siRNAs molecules have been approved
for marketing by the regulatory authorities, the development and regulatory process for subsequent siRNAs molecules will be facilitated and more cost efficient.
This is not the case for small molecules or antibodies, where each new chemical entity (NCE) commonly has a different composition and synthesis protocol.
The lead identification and optimization of siRNA molecules takes in our labs less than 3 months (vs. up to several years for the same process or e.g. small molecules). The significant reduction of the preclinical development time will lead to a sooner introduction into the clinic.
Because of these advantages Science magazine has elected both the RNAi molecules and the RNAi mechanism as Molecule of the Year in 2001 and as Scientific Discovery of the Year in 2002, respectively. Discoveries of RNAi were awarded Nobel prize2006 in medicine and physiology.
Challenges of RNAi as a Therapeutics:
Optimization of siRNA delivery to improve its cellular uptake
Improvement of oligonucleotides stability in serum
“off-target” effect elimination to avoid unspecific silencing
RNAi – mechanism of action
The lead identification and optimization of siRNA molecules takes in our labs less than 3 months (vs. up to several years for the same process or e.g. small molecules). The significant reduction of the preclinical development time will lead to a sooner introduction into the clinic.
Because of these advantages Science magazine has elected both the RNAi molecules and the RNAi mechanism as Molecule of the Year in 2001 and as Scientific Discovery of the Year in 2002, respectively. Discoveries of RNAi were awarded Nobel prize2006 in medicine and physiology.
Challenges of RNAi as a Therapeutics:
Optimization of siRNA delivery to improve its cellular uptakeImprovement of oligonucleotides stability in serum“off-target” effect elimination to avoid unspecific silencing
RNAi – mechanism of action
Celon Pharma Ltd
ul. Ogrodowa 2A
05-092 Lomianki, Kielpin
ul. Ogrodowa 2A
05-092 Lomianki, Kielpin
Celon Pharma © 2009 All Rights Reserved.
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Designed by GREGMAR