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Translation of RNA Medicines from Design to Clinic
characterization can be useful to generate
a sound database. By performing many
measurements for all parameters, the process
conditions needed to manufacture and store
a pharmaceutical product can be defined.
RNA is labile, highly charged, and complex.
As a result, pharmaceutical development
becomes a greater challenge than that for small
molecules. CMC (chemistry, manufacturing,
and controls) aspects are extremely important
for successful development of complex
nanoparticles that consist of RNA and vehicular
molecules. Formulation is of key importance
because it may affect the broader qualities.
Additional Resources...
Application Notes
Discover rich application
data, tools and resources
Precision NanoSystems™
to support your next milestone
A thorough understanding of the coherencies
inside the delivery systems, such as structure
and function as derived from advanced
characterization, including SAXS analysis,
assists performance of rational formulation
development. This also helps define the critical
quality attributes required to specify the
product that will be important for successful
translation of RNA into clinical development.
Listen to Dr. Haas' full keynote presentation from
Precision NanoSystems' Virtual Symposium:
Genetic Medicine from Concept to Clinic. n
1. Damase TR, Sukhovershin R, Boada C, Taraballi
F, Pettigrew RI., Cooke JP. The Limitless Future of
RNA Therapeutics. Frontiers in Bioengineering and
Biotechnology 9, 161 (2021).
View Here
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