The aim of MAGIC is to accelerate the development of gene therapies and genome editing for muscular dystrophies by creating advanced humanized muscle models and innovative approaches. The goal is to improve the lives of people living with muscular dystrophies.

Each month, we gather partners from the MAGIC project to discuss aspects in relation to gene therapies. Readers will learn who the people behind the MAGIC Project are, why we are committed to advancing gene therapies, and how our roles in MAGIC are crucial for achieving better health outcomes for people living with muscular dystrophies

 

In brief words, please let us know who you are individually and on behalf of which project partner organization. 

I am Frédéric Relaix, a Professor at Université Paris-Est Créteil Val de Marne (UPEC). Within the MAGIC project, I lead Work Package 5, focusing on preclinical assessment of the therapeutic strategies developed by the consortium. My expertise lies in muscle stem cells and translational research for neuromuscular disorders, combining basic research in myogenesis and adult muscle regeneration with preclinical modeling and the development of innovative therapies. I lead a large academic research team comprising experts in biological muscle research, medical doctors, and veterinary specialists.

 

Why are you participating in the MAGIC project? How can your perspectives complement the MAGIC project goal to accelerate the development of genetic therapies for muscular dystrophies? 

Our participation in the MAGIC project stems from our commitment to advancing therapeutic strategies for neuromuscular disorders. By leveraging our extensive experience in skeletal muscle and muscle stem cells biology, as well as preclinical modeling, we aim to contribute to the development of innovative models of human skeletal muscle disorders in complement to the preclinical models we are currently using. These models will be instrumental in developing, screening, and validating new neuromuscular gene therapy vectors and genome editing strategies with high efficacy and selective tropism, coupled with low toxicity and limited immunogenicity.

 

What have been the current challenges regarding the development of genetic therapies for muscular dystrophies for you? 

One of the significant challenges in developing genetic therapies for muscular dystrophies is the lack of robust, humanized models for the development of advanced therapies. This gap poses a major barrier toward the clinical translation of genetic therapies for muscle diseases. Additionally, the diverse pathophysiology and cellular compartments impaired in each specific condition complicate treatment strategies. The abundance and syncytial architecture of skeletal muscle, along with challenges in targeting critical supporting cell types, further delay progress, leading to inadequate delivery, ineffective gene expression, and dose-limiting interactions with the host immune system.

 

What are the main outcomes (direct results) you expect from the project? 

We anticipate that the MAGIC project will yield several key outcomes. First the development of advanced, humanized muscle models that accurately replicate skeletal muscle pathophysiology and complement current preclinical models. A second important point is the development and testing of innovative gene therapy vectors and genome editing strategies with high efficacy and selective tropism. Finally, the project will be instrumental in establishing of robust preclinical pipelines for testing and validating innovative therapies, facilitating their rapid translation into clinical settings. These outcomes will collectively contribute to reducing premature mortality and health burdens associated with muscular dystrophies and myopathies across Europe and worldwide.

 

What are the expected impacts on your organisation from participating in the project? 

Participation in the MAGIC project is expected to enhance our organization’s research capabilities and reputation in the field of neuromuscular disorders. It provides opportunities for collaboration with leading experts across Europe and beyond, fostering interdisciplinary research and innovation. Additionally, the project’s outcomes will inform and potentially transform our approaches to developing and implementing genetic therapies, ultimately improving patient care and treatment options for those affected by muscular dystrophies