MAGIC Research Consortium Meets in Lisbon
- Cure CMD
- 6 days ago
- 2 min read
The MAGIC consortium held its annual General Assembly from 28–30 October in Lisbon, hosted by the Gulbenkian Institute for Molecular Medicine (GIMM). Consortium members from around the world came together to share progress and plan next steps in this project that aims to accelerate the development of new treatments for rare neuromuscular conditions.
This meeting was a powerful reminder that the building of global networks consisting of researchers, clinicians, and advocates, who are united by a shared mission, is critical to our success. It was inspiring to see how far we’ve come in our understanding of neuromuscular conditions - taking cells donated by affected individuals and developing them into ‘mini muscles’ - bringing lab models closer to real-life disease. I am especially encouraged by how thoughtfully the team analyzes both wins and setbacks, to refine and tailor gene therapy and gene editing tools for testing in these muscle organoids. Cure CMD is proud to be a member of this this international collaboration to accelerate treatments for those living with a neuromuscular condition. - Gustavo Dziewczapolski PhD, Scientific Director, Cure CMD
Key topics from the meeting included:
Challenges in delivering gene therapies
Gene therapy often uses harmless viruses called AAV vectors to carry genetic instructions into cells. While these viruses don’t make people ill, they can trigger an immune response. Another challenge is that AAVs can spread to many tissues in the body, not just the muscles where treatment is needed. This can raise safety concerns, which have been in the news recently. MAGIC partners discussed potential solutions such as designing new AAVs, using alternative delivery systems, and making vectors more muscle-specific.
Advances in gene editing
Gene editing works by targeting our genetic code. Many neuromuscular conditions are caused by changes in a gene that stop a protein from working properly. Gene editing aims to correct these changes so the protein functions as it should. The MAGIC team showcased progress in gene editing approaches for Duchenne muscular dystrophy (DMD) and congenital muscular dystrophies. They also highlighted research into ways to edit other genes that may help protect muscles from damage.
Developing ‘mini muscles’ in the lab
Partners shared updates on creating 3D muscle models that look and behave more like real human muscle and mimic what happens in specific neuromuscular conditions. These “mini muscles” will help scientists test gene therapies more accurately and identify the most promising approaches to move forward.
Looking ahead
The meeting was a hub of innovation, collaboration, and fresh ideas – keeping the project on track to achieve our goals.
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