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Sep 15, 2008 (Vol. 28, No. 16)

Capitalizing on the RNAi Space Expansion

Players Are Buoyed by a Fast-Growing Field with a Tremendous Amount of Potential

  • Cellular Pathways

    “The availability of cellular models with silenced genes offers the possibility of hands-on studies based on RNAi. Access to such cell lines helps the researcher focus on experimental questions without the need to first construct a cellular model,” says Jean-François Têtu, Ph.D., product specialist at tebu-bio. “Tebu-bio offers SilenciX human adherent cells that have been modified by nonviral and safe transfection for long-term silencing of genes by RNAi.”

    According to Dr. Têtu, SilenciX was developed in collaboration with Denis Biard from Commissariat à l’Energie Atomique. “SilenciX is the first ready-to-use cellular model that is stable and has guaranteed knock down at more that 70 percent extinction at the RNA level.”

    This third-generation cell line is a robust cell model they produced with a new algorithm called designer of small interfering RNA that Ecole de Mines de Paris developed. This algorithm for predicting siRNA efficiency is based on a simple linear model.

  • RNA Duplexes

    As an RNA duplex progresses from discovery phase to the clinic several hundred grams of the duplex, manufactured to GMP standards, need to be sourced over the lifetime of the trials. “Since 1999, Avecia Biotechnology has been a supplier of GMP oligonucleotides,” reports Kevin Fettes, process development group leader.

    “Recently, as the therapeutic potential of RNAi has become established, the demand for GMP materials to be used in clinical trials has increased,” Fettes says. “My presentation will discuss the technology used to manufacture RNA duplexes and considerations that need to be addressed as demand for the duplex increases throughout clinical trials and as process validation and commercial launch approach,” he says.

    Avecia recently installed new large-scale high-pressure purification equipment for RNA/aptamers and the capacity to manufacture hundreds of kilograms of commercial product per year, according to Fettes. “The objective of our process-development group at Avecia is to provide efficient, robust, and scalable processes that can be used for clinical manufacture, process validation, and beyond,” he adds.

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