Developing precision gene therapies safely for severe childhood brain disorders

Assumpció Bosch

Universitat Autònoma de Barcelona (UAB), Spain

Some rare childhood brain disorders cause epilepsy, movement difficulties and progressive cognitive impairment from an early age. No treatment currently exists that can change the course of these diseases. This project seeks to develop advanced gene therapies that can restore or correct the affected biological function locally in a controlled way.

Focusing primarily on two severe paediatric encephalopathies, the researchers will develop and test strategies that allow them to deliver therapeutic instructions to the right brain cells without causing unwanted effects. A team with expertise in gene therapy, neurobiology and translational medicine will first test the candidate therapies in disease models, then assess their safety, durability and distribution and support future regulatory development.

The primary goal is to produce two therapeutic candidates ready to move towards clinical development. Beyond the two targeted disorders, this approach may establish a broader platform for precision gene therapy in neurological disorders affecting children, offering hope to patients and families who currently have very limited treatment options.

• Miguel Chillon, Fundació Hospital Universitari Vall d’Hebron - Institut de Recerca (VHIR), Spain

• Beverly Davidson, The Children's Hospital of Philadelphia Research Institute, United States of America

• José Lanciego, Fundación para la Investigación Médica Aplicada (FIMA/CIMA), Spain

• Fundación Gen Rebelde GNB1, Spain

• Alliance MLC, United States of America

Fine-tuning neuroglial regulation to treat epileptic and motor symptoms in GNB1 and MLC

€1,000,000.00