Monaco, May 12, 2026
Building on its long-standing support for SQY Therapeutics[1], which the association has accompanied and funded from its early stages through to its current clinical phase, Only Project continues to strengthen its support for innovative therapeutic research and the advancement of knowledge in neuromuscular diseases. Following the success of the first edition of the Pioneers Grant in late 2025, Only Project brought together the scientific community it supports in Monaco for a two-day workshop enabling researchers to exchange insights and share progress on their work.
Launched in spring 2025, the Pioneers Grant pursues Only Project mission to empower visionary and determined research teams whose work offers genuine therapeutic hope for patients living with Duchenne muscular dystrophy.
For the inaugural edition of the grant, the program attracted strong international interest with eleven applications from eight countries (Belgium, France, South Africa, Spain, the United States, the Netherlands, the United Kingdom, and Zambia) submitted to a dedicated Scientific Advisory Board (SAB). In close collaboration with Only Project’s Board of Directors, the SAB awarded four grants totaling more than €2.8 million to particularly outstanding projects:
Restoring brain dystrophin isoforms Split-Intein AAV Gene Therapy – Led by Aurélie Goyenvalle, PhD, and Cyrille Vaillend, PhD, at the University of Versailles Saint-Quentin-en-Yvelines (France), this project explores an innovative gene therapy approach aimed at restoring dystrophin forms present in the brain using split-intein AAV technology.
Beyond DNA cutting: advanced prime-editing systems for Duchenne muscular dystrophy – Initiated by Dr Manuel Gonçalves and Professor Annemieke Aartsma-Rus at Leiden University Medical Center (LUMC), Netherlands, this project seeks to develop a novel gene-editing therapy for DMD, capable of permanently repairing the genetic code responsible for the disease.
“BBRiDGE-DMD”, Brain and Behavioral Research in DMD across Global Environments – Led by Professor Liesbeth De Waele, at KU Leuven (Belgium), this global multicenter study mapping brain and behavioral development in boys with DMD aims to create the first DMD-specific brain development charts.
Unravelling the potential of postnatal dystrophin restoration in the DMD brain – Led by Dr Maaike van Putten and Dr Peter Hohenstein at Leiden University Medical Center (LUMC) in the Netherlands, this project aims to identify critical postnatal periods during which restoring dystrophin in the brain remains possible.
As highlighted by Tess Pettavino, Board Member of Only Project: “At a time when clinical advances are accelerating and offering renewed hope to many families, we felt it was essential to convene researchers and encourage the exchange of their scientific findings as well as the sharing of their experiences. This is precisely the ambition of the Pioneers Grant, which emphasizes initiatives that enhance research quality and reliability, particularly through large datasets and large-scale studies. These efforts are essential to deepen our understanding of Duchenne muscular dystrophy and continuously refine the interpretation of clinical outcomes. Each new therapy must be built on robust scientific foundations. The four recipients embody this ambition, and we warmly congratulate them and look forward to following their progress closely.”
Luc Pettavino, President of Only Project, added: “The best way to predict the future is to create it. This philosophy guides our association’s ongoing commitment to innovation, collaboration, and progress, whether in scientific breakthroughs or fundraising efforts. Through Only Project, our mission remains clear: to turn hope into tangible outcomes for patients and families affected by Duchenne muscular dystrophy. The workshop held in Monaco brought together outstanding researchers, which the association has supported over the past years. Launched in 2025, the Pioneers Grant represents a new milestone, supporting bold research projects with the potential to change lives. It is a significant commitment, marked by challenges, meaningful encounters, and concrete therapeutic promise. We are proud to support these first four pioneering projects. Through these meetings these researchers were able to join the community of scientists we have built over the past twenty years, and the association commitment will continue into 2026.”
Call for New Applications for the Pioneers Grant
Researchers with bold ideas and innovative approaches are encouraged to apply for the Pioneers Grant. Together, we can accelerate discovery and open new pathways toward improved treatments and care for people living with neuromuscular diseases. Applications for the next round are open until mid-August, beginning with an expression of interest, followed by the submission of a full application.
Read the official press release.
*****
Contact: media@onlyproject.org – Aliénor Miens – +33 (0)6 64 32 81 75
[1] (SQY Therapeutics is a biotechnology company specialising in antisense oligonucleotides, focused on developing clinical-stage R&D programs for genetic diseases, particularly Duchenne muscular dystrophy, with the goal of slowing or halting disease progression.)
The year 2024 marked a major turning point for AMM-Only Project. It was defined by continued progress on the clinical trial of SQY51, a promising drug candidate developed by SQY Therapeutics which was co-founded and financed by the association. The year also brought strengthened governance, the launch of a new endowment fund to scale our impact and prepare for the future, the expansion of our operational team, and nearly 30 million euros raised through Only Watch. All of these milestones are detailed in the 2024 Annual Report, available in both French and English.
In a global context marked by uncertainty, funding scientific research has never been more essential. This report is a testament to our commitment to supporting, with both rigor and ambition, the scientists who work every day to find a treatment for Duchenne muscular dystrophy and other neuromuscular diseases.
The association is also proud to share its 2024 financial statements, which have been certified by an independent auditor, Mr. Christian Boisson, of the firm Grant Thornton Monaco.
