RNA Therapeutics Workshop held March at the Calouste Gulbenkian Foundation in Lisboa
The Harvard Medical School Portugal Program together with the European Network of Excellence on Alternative Splicing EURASNET organized an RNA Therapeutics workshop at the Calouste Gulbenkian Foundation in Lisbon early in March.
RNA Molecules, as intermediates in the genetic information flow inside all the cells, have been already proven to be a key target for disease therapy as well as a tool for the development of new drugs. Since the discovery of small non-coding RNAs and all the associated phenomena like RNA interference, the possibility of using RNA-based drugs has become a subject of great interest in the pharmaceutical and clinical fields. RNA is at the core of many cellular processes and might become an outstanding landmark that will modify how we will understand and fight disease in the future.
This Workshop joined together specialists in the clinical area and in basic experimental research within the field in a pleasant atmosphere to discuss the forthcoming and present goals of RNA therapeutics. State of the art technologies and applications of RNA molecules as drugs or targets for therapeutics were presented by prestigious invited speakers. Topics ranged from targeted delivery RNA molecules to applications of RNAi technology, miRNAs and their antagonists, use of therapeutic aptamers, and splice-switching strategies.
The Workshop was intended for a wide audience of biomedical scientists from academia as well as industry. The two day workshop brought speakers, all experts in their fields, from various European countries, Canada and the United States. The audience gathered medical, science students and researchers from Porto in the north to Évora in the south to listen to innovative aspects in RNA.
It opened on Thursday March 4th with a presentation by Gert Jan Van Ommen from Leiden University Medical Center in the Netherlands, followed by presentations by Adrain Krainer from Cold Spring Harbor Laboratory and Mariano Garcia-Blanco from Duke University Medical Center, both from the US.
Day two on the 5th began with Jamal Tazi from the Institute of Molecular Genetics in France, followed by presentations by Tony de Fougerolles from Alnylam Pharmaceuticals - US, Hazel Pinheiro from Exiqon Denmark, Troels Koch from Santaris Pharma, Denmark, Jorg Kaufmann from Silence Therapeutics Germany, Michael P Czech from the University of Massachusetts Medical School, Sarah Fredriksson from Genovis, Sweden, Michael G Kaplitt (pictured above) from Weill Cornell Medical College, Sven Klussman from Noxxon Pharma Germany, Ryszard Kole from AVI BioPharma and Gerard J McGarrity from VIRxSYS both from the US.
During the workshop several applications of RNA molecules either as drugs or as therapeutic targets were described and analyzed. Some of the most interesting practical cases presented by the invited speakers were the development of RNA-based drugs against hepatitis C virus (HCV) described by Troels Koch from Santaris Pharma, and the use of gene therapy for the treatment of Parkinson patients as already implemented in Cornell University by Dr Michael Kaplitt. European biotech companies represented by Noxxon Pharma and Silence Therapeutics both in Germany and Genovis from Sweden showed their advances in new RNA-based drugs for disease treatment and in their delivery systems. Hazel Pinheiro, from Exiqon, pointed out future trends for the use of RNA molecules as diagnostic and prognostic markers. In the academic field Michael Czech from the University of Massachusetts discussed some of the most promising systems for oral RNA delivery based on the use of baker's yeast cells.
Maria Carmo-Fonseca and Francisco Enguita, organizers of the workshop, said they were pleased at the turnout - over 200 people atended the two day event. "As this was a more specific theme workshop we were pleased to see so many people who contributed to make this event a success" commented Prof. Carmo-Fonseca, Executive Director at the Institute of Molecular Medicine (IMM) in Lisbon and a renowned scientist herself and also the Program Director in Portugal of the Harvard Medical School Portugal Program. "This was another kick-off for the Harvard Program giving it exposure amongst the medical community in Portugal," she said.
Francisco Enguita, principal investigator at the IMM's Cellular Biology Unit, remarked "the reception among the public of this event has been overwhelming and there are already plans to organize another similar workshop in the following years. We would like to establish and celebrate this workshop on an annual basis as a meeting point for fruitful discussions and networking within the field." He encouraged the audience to contribute with suggestions, and thanked the esential support from the Harvard Medical School - Portugal Program for the organization of the workshop.