The project is accelerating the discovery of new therapeutic opportunities for rare neuromuscular diseases using interdisciplinary expertise from biology, AI, clinical science and patient engagement.

Rare neuromuscular diseases (NMDs) pose profound challenges to patients and health systems alike. Many remain without approved treatments, and progress in translational research is often slowed by limited disease models, small patient populations and fragmented datasets. DREAMS, funded by the European Union under the Horizon Europe programme, addresses these gaps by bringing together interdisciplinary expertise from biology, AI, clinical science and patient engagement. The multi-disciplinary approach seeks to uncover single drug treatments that can be individually repurposed to treat multiple rare diseases.

Research will be undertaken by Saverio and his group, who are seconded from UCL to the Crick's Stem cells and neuromuscular regeneration laboratory. The group's involvement in the consortium strengthens three core areas of the project’s scientific work: biomarker identification, iPSC-derived disease modelling, and in-vitro drug validation. The added expertise accelerates the transition from in-silico predictions to biological confirmation, a crucial step in drug discovery for rare diseases.

DREAMS marks the second Horizon Europe consortium involving Saverio, who also leads the MAGIC group, a four-year project to accelerate development of gene therapies for muscular dystrophies. 

By expanding its cellular model portfolio, strengthening its biomarker studies and reinforcing its drug-validation pipeline, DREAMS will work to uncover therapeutic opportunities that could benefit patients with rare neuromuscular conditions. These advances contribute to the project’s long-term goal: establishing a scalable, reproducible platform to identify treatments for disorders that share underlying molecular mechanisms.

The coming years will see DREAMS deepen its research work, generate new biological data and drive forward AI-assisted therapeutic discovery in a field where innovation is urgently needed. 

Joining the DREAMS consortium represents a unique opportunity to accelerate research into rare neuromuscular disorders. By combining our expertise in stem cell biology, disease modelling and advanced therapies with the consortium’s collaborative strength, we aim to transform fundamental discoveries into tangible therapeutic strategies that can improve patients’ lives.

– Francesco Saverio Tedesco