The DREAMS consortium is entering a new strategic phase with the incorporation of University College London (UCL), a global leader in biomedical research and regenerative medicine. This addition marks a significant reinforcement of the project’s scientific foundation and its ambition: to accelerate the discovery of new therapeutic opportunities for rare neuromuscular diseases (NMDs) using AI-supported drug repurposing, iPSC-derived skeletal muscle models, and high-throughput phenotypic screening.
Rare neuromuscular diseases 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 arrival of UCL brings a decisive boost to the consortium’s capacity to generate high-quality biological models, deepen mechanistic insight and bridge computational predictions with experimental validation. Their involvement directly strengthens three core areas of the project’s scientific work: biomarker identification, iPSC-derived disease modelling, and in-vitro drug validation.
A strategic partner for advancing DREAMS’ scientific mission
UCL’s laboratories are internationally recognised for their contributions to neuromuscular biology, stem-cell research and human cell modelling. These strengths align precisely with DREAMS’ objectives, making the institution a natural fit within the consortium.
The partnership strengthens DREAMS’ ability to produce robust, human-relevant data that can guide drug selection, refine biomarkers and support clinical translation.
UCL’s role across DREAMS work packages
UCL contributes to several foundational components of DREAMS, helping consolidate the scientific workflow that moves from disease mechanisms to potential therapeutic candidates.
WP1 – Biological materials, proteomic data and biomarkers in five rare NMDs
As part of WP1, UCL supports the identification of dysregulated genes and proteins, providing essential insights into shared mechanisms across the five targeted neuromuscular diseases. The team contributes to biomarker validation, using advanced human muscle cell models to assess the relevance and robustness of candidate markers emerging from the project’s experimental and computational analyses.
WP3 – Biological materials for new diseases and new biomarkers
UCL leads WP3, positioning the university at the heart of DREAMS’ experimental engine. In this work package, UCL is responsible for:
- generating new patient-derived induced pluripotent stem cell (iPSC) lines
- differentiating these lines into skeletal muscle cells and myotubes
- performing drug-response studies to explore disease phenotypes and mechanism-of-action signals
This leadership role enables the project to expand beyond its initial disease models and explore additional neuromuscular indications that share pathological features, broadening the potential impact of DREAMS’ findings.
WP4 – Candidate selection and in-vitro validation of drug candidates
DREAMS leverages advanced AI tools to identify drug candidates, both repurposable compounds and new molecular entities, that may act on molecular targets shared across several neuromuscular disorders. In Task 4.4, UCL conducts in-vitro assays in iPSC-derived muscle cells to evaluate:
- Therapeutic efficacy
- Mechanism-of-action signatures
- Cellular phenotype rescue
- Early safety and toxicity markers
This is a critical step in determining which AI-nominated candidates hold the most promise for advancing toward preclinical pathways.
What UCL brings to DREAMS
The incorporation of UCL adds significant scientific value to the consortium. Their contribution is not generic, it is highly specialised and aligned with the core experimental needs of the project.
UCL contributes:
- High-quality patient-derived iPSC lines for additional neuromuscular diseases, expanding DREAMS’ biological sample diversity and enabling investigation into new disease models.
- Advanced muscle differentiation and cellular modelling, essential for exploring disease pathways and validating biomarker signatures using human-relevant systems.
- Specialised assays to evaluate drug efficacy and mechanisms of action, providing critical validation of AI-predicted therapeutic candidates.
- Deep expertise in neuromuscular disorders, strengthening the interpretation of disease mechanisms and enhancing the project’s translational potential.
“We are delighted that UCL is joining the DREAMS consortium to advance research into rare neuromuscular disorders. This collaboration allows us to combine UCL’s expertise in stem cell modelling, disease phenotyping, and advanced therapies with the consortium’s collective strengths. By harnessing our strategic partnerships with the Francis Crick Institute, the NIHR Great Ormond Street Hospital Biomedical Research Centre and the MAGIC consortium, we aim to accelerate the translation of cutting-edge science into meaningful therapies for patients.” Francesco Saverio Tedesco & Sara Benedetti, University College London
Together, these contributions equip DREAMS with a more comprehensive experimental platform to study rare neuromuscular diseases and test therapeutic hypotheses emerging from computational discovery.
A milestone for the future of rare neuromuscular disease research
The integration of UCL into DREAMS represents more than a consortium expansion, it is a strategic enhancement of the project’s scientific capabilities. The added expertise accelerates the transition from in-silico predictions to biological confirmation, a crucial step in drug discovery for rare diseases.
By expanding its cellular model portfolio, strengthening its biomarker studies and reinforcing its drug-validation pipeline, DREAMS is now better positioned 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. With UCL now part of this journey, the consortium enters a new phase of scientific strength and translational potential, one that brings renewed hope for patients, researchers and the broader neuromuscular disease community.
