Accelerating the development of treatments for congenital muscular dystrophy – our £1 million partnership with LifeArc

14 April 2025

Researchers from University College London have jointly received £1 million to develop potential treatments for congenital muscular dystrophy, as part of our partnership with LifeArc.

Three research grants have been awarded as part of our partnership with LifeArc, who focus on supporting research which bridges the gap between laboratory-based research and clinical trials. Known as translational research, it aims to give potential new treatments the best chance possible of making it through the drug development pathway.

The three projects aim to identify and develop new treatments for congenital muscular dystrophy, a group of conditions where symptoms can start from birth, or in the first few months of life. Symptoms can include muscle weakness, reduced movement and tightness (contractures) in joints. There are currently no effective treatments.

Improving how to deliver treatments to muscle cells

Professor Haiyan Zhou and team have already identified potential treatments for collagen VI-related muscular dystrophy. However, they have found it difficult to deliver the treatments to where they are needed in body. The team have recently identified a targeting system which could help deliver the treatment. This project aims to link the targeting system with the potential treatment and test how well it works.

“We are deeply honoured to receive this joint award from LifeArc and Muscular Dystrophy UK, which will accelerate our work and the international collaboration to develop transformative RNA therapies for Collagen VI-related congenital muscular dystrophy (COL6-CMD).

This funding is pivotal not only for our team but for the entire muscular dystrophy research community. Success here would break the longstanding therapeutic bottleneck in COL6-CMD and establish a modular delivery platform adaptable to other neuromuscular disorders where muscle fibroblast targeting is critical. We are grateful to Muscular Dystrophy UK and LifeArc for their visionary support and shared commitment to turning science into hope.”

Professor Haiyan Zhou

Using ‘mini muscles’ to find new treatments

Professor Francesco Saverio Tedesco and his team aim to improve how potential treatments for LMNA-related congenital muscular dystrophy are tested. By growing ‘mini muscles’ in a dish that mirror the changes seen people with LMNA, Professor Tedesco aims to identify the most promising treatments to be taken to the next stage of research.

Can differences in DNA protect muscles?

Everyone has differences in their instruction manual – DNA – making them unique. Sometimes these differences can cause conditions such as congenital muscular dystrophy. Other times these differences can help protect the body from damage. Previous research has shown a specific DNA change may protect muscles in people with merosin-deficient congenital muscular dystrophy. In this project, Professor Francesco Muntoni and his team aim to design a treatment that can mirror this DNA change and potentially reduce the severity of the condition.

Stronger Together

“With no effective treatments for congenital muscular dystrophy, there is a need to develop new treatments. We’re delighted to partner with LifeArc to fund groundbreaking research that brings us closer to potentially life-changing treatments for those living with a condition. This partnership was made possible thanks to an incredibly generous legacy.”

Dr Kate Adcock

Our Director of Research and Innovation

“LifeArc is committed to supporting research that is focused on patient impact, in underserved areas. Congenital muscular dystrophy includes a group of devastating conditions, for which there is no cure and limited treatment options. By funding these research projects, in partnership with Muscular Dystrophy UK, we aim to improve the lives of patients living with these conditions.”

Dr Joanna Davidge

Head of Funding at LifeArc