Association Monégasque contre les Myopathies (AMM)

Duchenne Muscular Dystrophy (DMD)

A severe disease that causes muscular degeneration of motor abilities and progressively weakens respiratory, cardiac and digestive muscles.

 

The AMM's mission has been to create and consolidate the strong relationships between the different international research centers with expertise in the DMD field. In other words, turning competitors - even rivals - into collaborators in pursuit of the same cause. It is for this purpose that the Monaco round tables and the ICE programme (International Collaborative Effort for DMD) were created with the aim to evaluate the advances in preclinical research on DMD and supporting their transition into clinical trials.

 

"By the end of this first stage, two therapeutic strategies had been established," explained Luis Garcia, Research Director at the University of Versailles and the Monaco Scientific Centre (CSM - Centre Scientifique de Monaco). "We decided to support gene therapy and messenger RNA* repair technology through antisense oligonucleotides (AONs)*, without excluding the possibility of supporting other more fundamental, emerging projects as time went by."

 

With regards to repairing messenger RNA, the AMM significantly financed the development of a new class of antisense molecules, Tricyclo-DNA, invented by Professor Christian Leumann at the University of Berne. The development of this new generation of medicine involved several laboratories mainly based in Switzerland and France as well as in Sweden and the United Kingdom. The preclinical trial results, published in Nature Medicine, show the spectacular results of the treatment in a laboratory mouse carrying the illness. After several weeks of treatment, the missing protein was found to be present throughout the muscles and cardiac, respiratory and ambulatory related functions were restored. This work would not have been achieved without the creation of Synthena, a start-up at the University of Bern, the only entity capable of producing sufficient quantity of the molecules that are not yet commercially available.

 

For Luis Garcia "the next key phase will be the transfer of preclinical work to the patient, through new clinical trials of the molecules derived from Tricyclo-DNA. They have already proven their effectiveness in cells from patients and in different mice that have been tested. However certain safety issues still need to be addressed, particularly the 'host-medication' relationship which can vary from one species to another. In this respect, we have created a new start-up at the University of Versailles (SQY Therapeutics), which has been equipped to meet those needs in collaboration with Synthena. In addition, before undertaking long-term and costly regulatory toxicology studies required to apply for clinical trials, we have chosen to further refine our lead compounds in order to minimize potential problems of toxicity in Man. This effort was not in vain and it allowed us to design a new molecule safer and more effective”.

 

All this research is 100% financed by Association Monégasque contre les Myopathies, thanks to ONLY WATCH. At the same time, the AMM continues to support the gene therapy research of Professor Robert Kotin, a world-renowned specialist in the field of gene vector production, and the LIA-BAHN (International Associated Laboratory - Biotherapies applied to Neuromuscular Disabilities), a partnership between the University of Versailles and the Monaco Scientific Centre.

* mRNA, or messenger RNA, is a copy of a gene's DNA. It is transcribed in the nucleus then passes into the cytoplasm where it is translated into a protein.

An antisense oligonucleotide is a small sequence of DNA or RNA, generally produced in a laboratory, designed to link up with a natural messenger RNA (we talk of hybridization).

Tricyclo-DNA: a synthetic equivalent of DNA designed for the synthesis of antisense oligonucleotides (AON). AON-tricyclo- DNA offers greater affinity for their RNA targets and are better able to resist nucleic acid degrading enzymes.

Luc Pettavino - short bio

President of the Association Monégasque contre les Myopathies, Founder-Organizer of ONLY WATCH.

Former CEO and shareholder of the Monaco Yacht Show for nearly two decades. He brought this superyachting show up to its world leading position. He has always kept corporate social responsibility at the heart of his commitments (e.g. it was the 1st international yachting show to offset its carbon footprint / 50% of admission fees went directly to charity).

 

Co-founder of the Association Monégasque contre les Myopathies (AMM), he has been working since 2001 for the search of therapeutic solutions for Duchenne Muscular Dystrophy, a condition that afflicts his son Paul, aged 20 today.

 

He has created several thematic auction concepts in order to raise funds: the Projet Joconde, Adam & Eve, Only One Seasons 1 & 2, ONLY WATCH, Hermès Vintage & Rare Wines (for Mission Enfance). To this day, the AMM has contributed close to 25 million euros to international scientific and medical research.

 

On December 6, 2014, the Bern University honored him with a Doctor Honoris Causa title for his commitment in the fight against neuromuscular diseases.

 

He was also knighted Chevalier de l’Ordre de Saint Charles by H.S.H. Prince Rainier III of Monaco in 2003 and elevated to the rank of Officer in the Ordre de Saint Charles by H.S.H. Prince Albert II of Monaco in 2016.

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