Our goal is to support researchers, institutions and patients

The Charity’s actions are based on 3 pillars which govern the distribution of the funds raised thanks to auctions and generous donors.

Pillar 01

Funding of patient–oriented and charity-owned biotechs on proprietary technologies and therapeutic avenues – financing the setting up of our 1st clinical trial on a SQY51 molecule in the Paris region.

Since the very beginning our goal has been to find a cure for Duchenne muscular dystrophy. After years of organizing roundtables, financing projects here and there testing the tangibility of such and such therapeutic avenue — one of the key challenges being get the genetic « repair message » to all cells in a lasting yet non-toxic way — one pathway was found to be particularly promising. A professor had created a new class of antisense oligonucleotides (synthetic RNA fragments that attach to the mRNA) that could bring the message in the right places, efficiently. In 2012, a first biotech was created, financed by Only Watch means around this chemistry. In 2015, still thanks to Only Watch funds, the same group of parents and researchers launched a second biotech on the basis of this chemistry, this time focused on developing therapeutic compounds and working towards clinical trials. This organization currently employs 18 people (mainly biologists and chemists). This has been an important part of the charity’s spending in the past years.

 

Distribution

90,6% €39,5 million (on a 10 year basis, 2013—2023)

Main beneficiaries

Pillar 02

Funding of research projects within universities, hospitals and public institutions — including « Paul Pettavino » doctoral grants and post-doctoral fellowships.

Over the years, our charity has been attached to being an agile, potent and reliable supporter to researchers looking to investigate new pathways, launch new useful endeavors or to empower young talent to conduct doctoral theses and post-doctoral projects. That is why – in parallel with financing the development of the SQY51 molecule that is currently reaching clinical trial stage – we have never stopped financing ad hoc projects and giving out grants and fellowships from in-hospital or university research. To continue developing knowledge on Duchenne muscular dystrophy, the evolution of the disease, its underlying mechanisms, complementary therapeutic avenues… One can never get to the end of fundamental research. Moreover, financing the emergence of the new generation of researchers – young women and men – is a key part of our strategy and ambition for the charity.

Distribution

9,2% €4 million (on a 10 year basis, 2013—2023)

A selection of beneficiaries

Pillar 03

Funding of material for local patients and families (wheelchairs, mobility aids, vehicle adaptation…).

Because our team knows how a diagnosis brings a lot of material and logistical challenges in a family’s day-to-day life, even though the heart of our mission has always been to support research, we punctually support patients and their families from our local community in the acquisition of new essential material (electrical verticalizing wheelchair, adapting a vehicule to transporting a wheelchair, repairs, home adaptations…).

Distribution

0,2% €79 000 (on a 10 year basis, 2013—2023)

Portraits of researchers

Luis Garcia

Director of Research at CNRS, Director of the ENDICAP laboratory – UMR 1179 Inserm-University of Versailles-St-Quentin, UFR santé Simone Veil, Special scientific advisor for SQY Therapeutics

I’ve had one fixed goal for more than 25 years: find a cure for Duchenne muscular dystrophy. At the time of the last Only Watch we were filing for a clinical trial authorization from the sanitary authorities for a molecule we’ve developed. Now we have that authorization, and we are starting the safety phase. But beware, it’s a sprint type of effort with the duration of a marathon: the trial is starting now but it’s the result of years of work and there are many more steps and developments to come, we must stay focused.

Without Only Watch we would not be where we are today, at a clinical stage, we would still be people with ideas. Thanks to the means it gives us, not only have we shown it’s feasible – we went from ground 0 to clinical stage – but it allows us to have the ambition to develop our technology for other diseases. A big thank you to the Only Watch community.

Watch the interview

Helge Amthor

Professor of pediatrics specialized in muscle diseases in childhood, University Paris-Saclay UVSQ, UFR Simone Veil – Santé, Research Unit U1179, Raymond Poincaré University Hospital

I split my time between consultations at the hospital and research in the laboratory. On the clinical side, I see patients suffering from neuromuscular disorders, amongst whom many with Duchenne muscular dystrophy. Currently I’m also responsible for patients who take part in the clinical trial testing the SQY51 molecule – a clinical trial of which the promoter, SQY Therapeutics, is run by parents of Duchenne patients, and funded by charity funds, from Only Watch.

On the research side, I work with collaborators, some of whom are Master’s or PhD students, on projects aiming to gain a better understanding of the underlying mechanism of DMD, as well as to develop novel therapeutic avenues. This part of my work has also been supported by the charity for years. Being able to pursue both (clinical work and research) has always allowed me to go further in both fields.

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Dr. Angely Mendoza Cardozo

Clinical Chief in the Pediatric Neurology Department at Raymond Poincaré University Hospital, working in the AVANCE 1 clinical trial

This job allows me to develop as a human being and as a neuropediatrician. I thank the Only Project for its support and wish to continue working on this path of hope to improve the life quality of our patients and what we wish for them all, with all our efforts and all our hearts, their healing.

Karim Whabi

MD, PhD, Professor of cardiology at Paris Cité University, head of the reference center for neuromuscular cardiomyopathies in Cochin Hospital, Paris

My key areas of research are the prevention and treatment of heart failure and sudden death, which are the most frequent manifestations of cardiomyopathies in this condition, along with the study of their underlying molecular mechanisms.

I had the opportunity to set up the largest database worldwide dedicated to cardiac disease in Duchenne muscular dystrophy. This project, financed with Only Watch funds, contributed to improving the standard of patient care. I am also involved in various translational research programs involving specialists in both clinical and fundamental research, which are essential to develop innovative treatments to cure this complex genetic disease

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Sadia Belkacem

Member of Pr. Wahbi’s team dedicated to the advancement of knowledge on cardiac damage in Duchenne muscular dystrophy

As a project leader, I coordinate projects led by Prof. Karim Wahbi aimed at improving the cardiac care of DMD patients. These projects are based on the careful collection of quality clinical and genetic data from as many patients as possible in dedicated databases, the analysis of which allows to improve patient care and to set up trials evaluating new therapies.

Christophe Marcelle

Professor, Faculty of Biosciences, Deputy Director, MeLis (CNRS UMR 5284 / INSERM U1314), NeuroMyoGene Institute (INMG), University Claude Bernard Lyon1

As a Professor, I’m extremely keen on supporting the emergence of a next generation of researchers – women and men – and to help them to create bonds with each other, communities, so as to share knowledge, experience and create synergies. Funding granted by the association has allowed me to offer such perspectives to students.
As a researcher myself and a thesis director, it has also allowed me to continue to explore subjects that I would have had to give up otherwise.

Clémence Alibert

PhD student, University Claude Bernard Lyon1

Working under Pr. Marcelle’s guidance on the following thesis project « Muscle fusion as a delivery mechanism to repair ailing muscles from heritable muscle diseases ».

Marisa Jaconi

PhD, Professor at the University of Geneva (UNIGE), Scientific Director of the Lugano Cell Factory, Institute Cardiocentro Ticino‐EOC

Pr. Jaconi‘s ongoing work on « Immortal immunoprivileged myogenic stem cells for gene therapy of muscular dystrophy » spans over two years (2022-2023) and is financed by our charity’s « Paul Pettavino fellowship » granted through the Fondation Suisse pour la Recherche sur les Maladies Musculaires (FSRMM). The goal of her research is to overcome known obstacles to the prospect of effective stem cell therapy for Duchenne muscular dystrophy patients.

Robert M. Kotin

PhD, Adjunct Professor, University of Massachusetts Chan Medical School, Worcester, USA and Chief Science Advisor at Carbon Biosciences

The Only Project’s (AMM) funding was critical for supporting our research leading to one of the widely used production platforms for recombinant adeno-associated virus. The NIH has outlicensed our technology and materials to several companies including uniQure and BioMarin and both have FDA or EMA approve gene therapies based on our platform. (…) It’s unlikely that this would have occurred without the AMM’s support. I’ll always be grateful for the support, confidence, and trust.

Lucie Royer

Molecular biology engineer in Research Unit U1179, University Paris-Saclay, UFR Simone Veil – Santé, Research Unit U1179, working on Duchenne and Becker muscular dystrophy

Watch the interview

A selection of works supported by our Charity

To give you a better understanding of how we execute our mission and the type of work we enable thanks for the funding provided, here is a selection of recent projects supported by the charity:

Clinical trial AVANCE 1

Clinical trial AVANCE 1 to test the safety and efficiency of SQY51, a therapy entirely developed thanks for our non-profit funds and sponsored by a biotech run by our charity, researchers and parents of Duchenne patients (SQY Therapeutics).

A large-scale natural history study on the heart in DMD

Large-scale natural history studies, such as that of Prof. Karim Wahbi on cardiomyopathy associated with Duchenne muscular dystrophy, which will last at least 3 years and will use MRI with biomarker analysis, starting in spring 2023 at Necker Hospital (and which our organization will support until completion). The charity has already funded the same team to establish a national registry on cardiomyopathies associated with Duchenne muscular dystrophy, which can be expanded internationally over the next three years thanks to renewed funding.

Robert Kotin’s study on paroviruses

Dr. Robert M. Kotin’s study published in November 2022 in the journal PLOS Biology, co-funded by the Bill and Melinda Gates Foundation and the Association Monégasque contre les Myopathies entitled « Comparative analysis reveals the long-term coevolutionary history of parvoviruses and vertebrates ».

A project on the obstacles to effective stem cell therapy for DMD

Professor Marisa Jaconi’s ongoing work on « Immortal immunoprivileged myogenic stem cells for gene therapy of muscular dystrophy » spans over two years (2022-2023) and is financed by our charity’s “Paul Pettavino fellowship » granted through the Fondation Suisse pour la Recherche sur les Maladies Musculaires (FSRMM). The goal of her research is to overcome known obstacles to the prospect of effective stem cell therapy for Duchenne muscular dystrophy patients.

Clémence Alibert’s PhD thesis on muscle fusion

PhD student Clémence Alibert’s project « Muscle fusion as a delivery mechanism to repair ailing muscles from heritable muscle diseases » on which she is currently working under Professor Christophe Marcelle’s leadership for 3 years (2022-2024) with the aim of providing an effective cell and gene therapy for Duchenne muscular dystrophy, and the hypothesis that genetically modified blood cells expressing the fusion master genes Myomaker and Myomixer can be used to repair dystrophic muscles.

An AP-HP hospital group study

The study published by a group of doctors and scientists from the AP-HP hospital group (Cochin, Hôtel-Dieu, Necker-Enfants Malades) entitled « Association between prophylactic angiotensin-converting enzyme inhibitors and overall survival in Duchenne muscular dystrophy-analysis of registry data » in the European Heart Journal in March 2021.

The LIA-BAHN Laboratory’s work in Monaco

A large part of the work achieved and published by the Monégasque-French team of the LIA-BAHN laboratory on Biotherapies applied to neuromuscular disabilities, located at the Monaco Scientific Center (CSM) and whose creation was significantly supported by the charity, in 2013.

Amalia Stantzou postdoctoral work on skeletal muscle pathophysiology

The translational research work of Amalia Stantzou initiated in 2020 on « Skeletal muscle pathophysiology – Differential expression and restoration of dystrophin at critical expression sites in the muscle fiber » within the University of Versailles Saint-Quentin (France).

A 3D simulator for better diagnosis and treatment

Innovative technological projects that improve the understanding and treatment of Duchenne muscular dystrophy, such as Jean-Paul Carta’s 3D simulator to study neuromuscular and neurological diseases in a new way.

Funds & Ressources

We have the means to have an impact thanks for a unique fundraising project: Only Watch.

How we raise funds