May 24, 2022 - May 25, 2022

RNA therapeutics and Neuroscience

Virtual meeting: instructions to connect are communicated upon registration.

The RNA & NEUROSCIENCE meeting aims to bring together graduate students, academic and industry scientists working in the fields of RNA and the Central Nervous System (CNS) to present their latest research results, ideas, developments, biological medical applications and discuss the current concepts and the challenges that we are facing for future research.

RNA therapeutics is a novel technology that provides a unique treatment for a variety of diseases.  While it has been a success for the COVID19 pandemic, the RNA community still has the challenge on delivering some of these RNA molecules in other parts of the body (e.g.  brain and cardiac tissue) and better specificity to have a full therapeutic effect. Therefore, this meeting will explore the novel RNA therapeutics that are been currently developing and their mechanisms to cure a variety of diseases.

Additionally, the complexity of the brain provides a unique challenge to find therapeutics to cure neurodevelopmental disorders and neurodegenerative diseases such as Alzheimer’s and ALS. These diseases are characterized by the lack of gene regulation causing protein imbalance at the synapses and therefore cell death. Thus, in this meeting we will also explore the mechanisms that cause these diseases and the novel therapeutics that are currently being developed for their cure.

This international conference is organised in the framework of the BIOPHARMACEUTICALS ARD CVL Programme.


RNA therapeutics, RNA molecules, Neurosciences, Neurodevelopment diseases, Neurodegenerative diseases, Central Nervous System (CNS), RNA therapy, RNA delivery and Gene regulation


Organising committee

Confirmed speakers


Tuesday 24th May (14:00-18:50 Gmt+2 Paris)


14:00 Official Opening by Sophie Gabillet, Dr Kathia Zaleta & Prof. Patrick Vourc'h 


Chairman: Dr Frédéric Laumonnier
  • 14:30 Prof. Antonella Riccio - RNA metabolism in developing neurons
  • 15:10 Dr Ana Cristina Calvo  - Peripheral RNA Biomarkers in Amyotrophic Lateral Sclerosis
  • 15:50 Dr Débora Lanznaster - Combined Metabolomics and targeted-transcriptomics analysis in the muscle of early-stage ALS patients
  • 16:30 Coffee break


Chairman: Prof Chantal Pichon & Dr Fédérico Perche
  • 16:50 Dr Horacio Cabral - RNA/Polymer-Based Supramolecular Approaches for mRNA Delivery
  • 17:30 Prof. Steven Dowdy Delivery of RNA Therapeutics: The Great Endosomal Escape!
  • 18:10 Director Jason Potter - What matters for making highly expressed mRNA.
  • 18:50 End of the conference day 

Wednesday 25th May (14:00-19:00 Gmt+2 Paris)

Session 3: RNA THERAPEUTICS - Part 1

Chairman: Prof. Patrick Vourc'h
  • 14:00 Prof. Jernej Ule  - How do protein-RNA condensates form and contribute to disease?
  • 14:40 Dr Liliane Massade - Squalenoyl siRNA PMP22 nanoparticles, a potent therapy for Charcot-Marie-Tooth disease type 1A
  • 15:20 Dr Jean-René Martin - The snoRNA jouvence, a new versatile tool to protect against the deleterious effects of ageing, as well as inversely, for the treatment of cancer.
  • 16:00 Prof. Krzystof Sobczak - Compounds which alleviate the pleiotropic toxicity of RNA harboring expanded CGG repeats in the Fragile X–associated syndrome
  • 16:40 Coffe break
Session 4: RNA THERAPEUTICS - Part 2
Chairman: Dr Kathia Zaleta
  • 17:00 Prof. Stefano Gustincich - Antisense long non-coding SINEUP RNAs: from molecular mechanism to therapeutics
  • 17:40 Prof. Michelle L. Hastings - Antisense Oligonucleotides for the Treatment of Disease
  • 18:20 Dr Lori L. Isom - Dancing to a different tune: TANGO offers a precision medicine approach to treating Dravet syndrome
  • 19:00 Conclusion




lestudiumconference_bookletofabstracts.pdf5.72 MB

Partners of the event