Aurélie Goyenvalle, PhD
University of Versailles
Biotherapy for Neuromuscular Diseases
How did you become interested in the field of oligonucleotides?
I started my PhD in 2002 in Genethon in France working on therapeutics for muscular dystrophies, and it was the time when antisense oligonucleotides-mediated exon-skipping was starting to emerge as a therapeutic option for Duchenne muscular Dystrophy (DMD). Being in a large gene therapy center, we decided to vectorised the antisense sequences in order to take advantage of the delivery properties of viral vectors such as AAV to muscle tissues. My PhD project led to the development of AAV-U7snRNA tools which turned out to be particularly effective at inducing exon skipping and restoring dystrophin expression in DMD animal models. I still feel very lucky that my PhD project was successful and so inspiring for me; I guess my interest in RNA therapeutics and the versatility of antisense tools never ceased to fascinate me ever since.
Who were your early mentors? What is special about the type of research/work you’ve done?
My early mentor was Luis Garcia who passed on his passion for science and has always been very supportive and inspiring. For my postdoc, I joined Prof. Kay Davies’ laboratory at the University of Oxford and she also played an important role in my career and was in inspiration and an example of successful woman in science.
How did you become involved in OTS?
I attended my first OTS meeting in 2011 in Copenhagen after recommendations from my fellow postdocs at the university of Oxford. I received the Dr Alan Gewirtz memorial scholarship that year and discovered an excellent meeting, encouraging interactions between academia and industry, but also allowing friendly exchanges between young and senior scientists (not to mention the excellent OTS parties!) I am happy to support the OTS society which keeps growing and has become an essential part of the field.
What is special about the type of research/work you’ve done?
My research projects are focusing on developing therapeutic approaches for neuromuscular diseases based on antisense oligonucleotides made of tricyclo-DNA (tcDNA). We have shown in various disease models that tricyclo-DNA ASOs displays unique pharmacological properties and unprecedented uptake in many tissues after systemic administration.
Besides the exciting scientific aspects of this research, what is special about these projects is the collaborative effort they have emerged from. Most of our work was supported by the Association Monegasque against myopathies based in Monaco and his president, Luc Pettavino successfully founded a unique type of event called ONLY WATCH with the continuous support of HSH Prince Albert II of Monaco. Only watch is a biennial auction of one-off luxury timepieces made by the finest watch manufactures for research into Duchenne muscular dystrophy and on November 2019, the 8th edition of the auction raised the record breaking amount of CHF 38,593,000 (https://www.onlywatch.com/). It is quite fascinating that such a collective effort was able to support research to the stage of early clinical trials and I think this is a very inspiring model.
What do you like to do in your free time?
I love traveling, so if I am out of office, I am probably somewhere exploring new territories and new cultures, and maybe petting Alpacas (on behalf of Annemieke. ;)
Any other fun facts/tidbits you would like us to know!
Well I am a typical French and true lover of French food including the most stereotyped one, so do not hesitate to add frog legs, oysters or escargots/snails to next OTS meeting menu. :)