End of year 2024 update from the Only Project

AMM-Only Project's Research Impact

→ 13 projects have received funding in 2024 amongst which 10 new beneficiaries. These teams are working on topics such as myogenic reprogramming, muscle stem cells, advancing knowledge of cardiac damage in Duchenne muscular dystrophy or a 3D sensory platform for wheelchair users.

→ There are currently 49 people working on projects financially supported by AMM-Only Project.

→ An update on our flagship project: drug-candidate SQY51, developed by non-profit owned startup SQY Therapeutics. The first phase of the AVANCE1 trial, which included 12 patients, has shown encouraging results, with no major side effects reported. The second phase (2A) is now underway, continuing to assess the drug’s effectiveness. SQY Therapeutics is optimistic that SQY51 could significantly improve the lives of numerous patients. The startup has also recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to SQY51 that will favor its development.

This biotech based in France, has spent over a decade developing a groundbreaking treatment for Duchenne muscular dystrophy (DMD). Currently being tested in the AVANCE1 clinical trial, SQY51 is administered through an IV and reaches all the muscles in the body, including the heart. It acts like a “molecular patch”, which induces on demand exon skipping to rescue the translation of the mRNA from the mutated gene that causes DMD. By skipping over the damaged part of the gene, SQY51 enables the body to produce a truncated but still functional key protein that preserves muscle.

→ There is a lot happening in the field of DMD research, with numerous promising pathways being explored through clinical trials. While it is wise to stay cautious and precise (most therapies cover portions of the DMD population), it is very encouraging news for all of us involved in this cause! We are monitoring things closely and strengthening our connections within the scientific ecosystem to make sure to be as useful and relevant as possible.

Meet one of our beneficiaries: PhD student Chloé Bonnot, 28 years old

As a recipient of an AMM-Only Project doctoral grant, Chloé is currently in the final year of her PhD at Claude Bernard University Lyon 1. Her thesis, titled "The Role of T Cells in Skeletal Muscle Regeneration and Their Potential in Treating Dystrophies", explores the critical involvement of T cells (cells that help the body fight infections and diseases) in the process of muscle repair in mice. Chloé’s research sheds light on the unique ability of T cells to fuse with regenerating muscle fibers, revealing an exciting new avenue for treating muscle disorders such as Duchenne Muscular Dystrophy (DMD).

Following the completion of her PhD, Chloé plans to pursue postdoctoral research abroad, aiming to deepen her investigation into tissue regeneration and intercellular communication. The AMM-Only Project team extends their best wishes as she prepares to pursue a career in scientific research.

🎥 Spotlight: Documentaries bringing Duchenne Stories to Life

→ The World Duchenne Organization launched a documentary that portrays the lives of people living with Duchenne muscular dystrophy across the globe and to share their journeys, challenges, and successes.
👉 Watch it here

→ “6 of 9” is a personal documentary by Shannon Dix that tells the story of his brother, Kieran, who passed away at 24 after battling Duchenne Muscular Dystrophy (DMD). Through interviews with family members, the film explores their day-to-day life living with Kieran, the sixth of nine boys, and offers a heartfelt look at the impact of DMD on their family.
👉 Watch it on Vimeo

→ You may have already come across Netflix's touching documentary, “The Remarkable Life of Ibelin”, which brought DMD awareness to millions by sharing how Mats Steen, a young man with Duchenne, built a vibrant community and meaningful connections through online gaming, overcoming the challenges of his condition.
👉 Read the NYT's review here

Thank you for being part of a journey to improve the lives of those affected by DMD. The AMM-Only Project team wishes you a very happy new year!