A biocatalysis reactor for the industrial-scale production of therapeutic siRNA molecules
PD Dr. med. Jacques Rohayem
Technical University Dresden | Riboxx GmbH, Radebeul
Recipient: Technical University Dresden
Funding: GO-Bio Phase I (01.10.2007 - 31.12.2010, 1.297.446 Euro)
Recipient: Riboxx GmbH
Funding: GO-Bio Phase II (01.01.2011 - 31.12.2013, 1.142.730 Euro)
In recent years, molecular medicine researchers have been investigating the phenomenon of RNA interference, which can be used to selectively repress the activity of specific genes. This method is based among others on small RNA molecules – so-called ‘small interfering RNAs’ (siRNAs) that can be deployed to targetedly interrupt the process of protein production. These molecules are considered to have high potential for use in medicine, in particular for the treatment of diseases in which it is possible to locally administer siRNAs, such as cancer diseases or viral infections. In the first phase of GO-Bio funding, Jacques Rohayem successfully developed an enzymatic method of duplication that enables the quick and cost-efficient production of RNAi therapeutics in large quantities.
The researchers have validated the effect of their siRNA molecules in animal testing. Riboxx GmbH, which was founded in 2009 and is based in Radebeul near Dresden, Germany, offers the design and synthesis of small RNAs as a service. The company has already filed patent applications for ten of its innovations. In the second phase of GO-Bio, the Rohayem-based team is planning to transfer production of siRNAs to an industrial scale.
This will encompass the construction of a prototype biocatalysis reactor that can produce customised RNA molecules in volumes of up to 50 litres. The objective of the researchers is not only to significantly increase production capacity but to also test the produced molecules for their efficacy and selectivity in animal experiments. In addition, the Riboxx RNAi reactor (R-Cube) will be optimised for the production of RNAs under GMP conditions.