Funding for the SynLeigh Project to Advance New Therapies for Leigh Syndrome

IMP, Mitocon and Cure Mito Welcome ERDERA Funding

International Mito Patients (IMP), Mitocon and Cure Mito are delighted to announce that the SynLeigh consortium has been awarded funding by the European Rare Diseases Research Alliance (ERDERA). This important support will enable the consortium to accelerate the development of new therapeutic approaches for Leigh syndrome spectrum (LSS) disorders, a devastating and life-limiting form of mitochondrial disease.

Leigh syndrome spectrum disorders affect approximately 1 in 40,000 children worldwide. Often diagnosed in infancy or early childhood, these conditions lead to progressive neurological decline and, at present, there is no cure and very limited treatment options. For families living with Leigh syndrome, research initiatives such as SynLeigh represent a vital source of hope for meaningful, life-changing therapies.

The SynLeigh project aims to develop synergistic therapeutic approaches by building on existing progress in the field. Two promising therapeutic candidates that have already received Orphan Drug Designation (ODD) from the European Medicines Agency (EMA) will be further advanced. Their safety and efficacy will be assessed across diverse forms of Leigh syndrome spectrum disorders using an innovative and comprehensive preclinical platform. This includes the use of brain organoids, organ-on-chip systems, machine-learning–driven analyses, and both small and large mammalian models.

By integrating these advanced methodologies, SynLeigh seeks to generate the robust preclinical evidence required to accelerate the transition towards clinical trials, helping to bridge the gap between laboratory research and real-world impact for patients.

Below is a video of Professor Alessandro Prigione announcing the funding

A defining strength of SynLeigh is the active involvement of patient advocacy organisations, including IMP, Mitocon and Cure Mito. Their engagement ensures that the research remains firmly grounded in the real needs and priorities of patients and families, helping to guide strategies that focus on outcomes that truly matter to those living with Leigh syndrome.

“For families affected by Leigh syndrome, every research advance matters. SynLeigh represents hope — hope that innovative science, combined with a strong patient voice, can lead to meaningful treatments where none currently exist,” said Paula Morandi Chair of IMP, Board Member of Mitocon and patient representative for mitochondrial eye diseases.

The consortium brings together leading international experts in mitochondrial disease research and translational science, alongside patient organisations, reflecting a strong commitment to collaboration across borders and disciplines.

“It is a very exciting time for mitochondrial diseases with recent approvals for different drugs. For Leigh syndrome however we still do not have treatments. In this consortium, scientists and patient representatives will work on consolidating two drugs for clinical trial readiness. We hope that our efforts can result into practical therapeutic options for patients affected by Leigh syndrome”, commented Professor Alessandro Prigione, Project Coordinator of SynLeigh.

The SynLeigh Consortium includes:

• Ole Pless (Fraunhofer Hamburg, Germany)
• Emma Puighermanal (I3PT Parc Taulí Institute, Barcelona, Spain)
• Antonio Del Sol (University of Luxembourg)
• Raquel Cuella Martin (McGill University, Montreal, Canada)
• Una Riekstina (University of Latvia)
• Dario Brunetti (Istituto Neurologico Carlo Besta, Milan, Italy)
• International Mito Patients 
• Cure Mito
• Mitocon

The consortium also acknowledges the previous and ongoing funding that has laid the groundwork for SynLeigh. This includes support from the European Joint Programme on Rare Diseases (EJPRD) for the CureMILS consortium, AFM-Téléthon for the SlideMITO consortium, and the European Commission for the Horizon Europe SIMPATHIC project. IMP has been an active partner in both the CureMILS and SIMPATHIC projects, ensuring that the patient voice has been embedded throughout these initiatives. Together, these programmes have enabled the development of strong collaborative networks, innovative research methodologies and essential preliminary data.

Collectively, these efforts strengthen confidence that SynLeigh will deliver meaningful advances with tangible benefits for individuals and families living with Leigh syndrome, bringing the mitochondrial disease community closer to urgently needed therapeutic options.

IMP, Mitocon and Cure Mito strongly believe that as patient organisations we must work together on an international basis to progress the development of research and urgently needed therapies for everyone affected by mitochondrial diseases. 

Contacts: 

Jo de Bry, Director of Development and Operations, IMP, jodebry@mitopatients.org

Serena Massucci, Chief Scientific Officer, Mitocon, s.massucci@mitocon.it

Kasey Woleben, Executive Director and President, Co-Founder, kasey@curemito.org