The ”la Caixa” Foundation has selected four projects involving researchers from the Vall d’Hebron Research Institute (VHIR) under its 2026 Health Research and CaixaImpulse Health Innovation calls. The initiatives address some of the main current challenges in biomedical research, with proposals ranging from the development of new therapies for childhood neurological diseases and Parkinson’s disease to new therapeutic approaches for mitochondrial DNA maintenance disorders and cancer.

Taken together, the four projects represent different approaches to biomedical research, from basic and translational research to the development of new therapeutic strategies with the potential to reach clinical practice. These are the four projects that have been selected:

New gene therapies to treat childhood brain disorders

• Project leader: Assumpció Bosch, Universitat Autònoma de Barcelona (UAB).
• VHIR participation: Miguel Chillón, head of VHIR’s Gene Therapy in the Nervous System group.
• Grant received: €1,000,000, under the 2026 Health Research call.
• Consortium project with: CIMA, The Children’s Hospital of Philadelphia Research Institute, Fundación Gen Rebelde and Alliance MLC.

Some rare childhood brain disorders cause epilepsy, movement difficulties and progressive cognitive decline from the earliest stages of life. Currently, no treatments are available that can modify the course of these diseases.

The project aims to develop advanced gene therapies that can restore or correct the affected biological function locally and in a controlled manner. Specifically, it will focus on two severe paediatric encephalopathies and study strategies to deliver therapeutic instructions to the appropriate brain cells without causing unwanted effects.

The researchers will test the therapeutic candidates in disease models and assess their safety, durability and distribution. The goal is to obtain two candidates ready to advance towards clinical development and establish a platform that can contribute to the development of precision gene therapies for other childhood neurological disorders.

An intelligent system to personalise Parkinson’s disease treatment

• Project leader: Jorge Hernández, neurologist in the Movement Disorders Unit of the Neurology Department at Vall d’Hebron University Hospital and researcher in VHIR’s Neurodegenerative Diseases group.
• Grant received: €957,827, under the 2026 Health Research call.
• Consortium project with: Consorci Sanitari de l’Alt Penedès i Garraf, INIBIC-Hospital A Coruña, Barcelona Supercomputing Center-Centro Nacional de Supercomputación (BSC-CNS) and Asociación Parkinson Madrid.

In advanced stages of Parkinson’s disease, patients may experience significant fluctuations in motor symptoms. Continuous infusions can help control these fluctuations, but current systems do not automatically adapt to each person’s changing needs and often require manual adjustments.

The project led by VHIR aims to develop an intelligent system capable of personalising treatment in real time. The proposal will combine wearable sensors to continuously monitor motor symptoms with artificial intelligence systems that analyse this information and recommend dose adjustments, which could subsequently be implemented by the infusion system.

This approach could improve motor symptom control, autonomy and quality of life for people with advanced Parkinson’s disease. At the same time, it could lay the foundations for a more personalised infusion therapy adapted to each patient’s changing needs.

A new therapeutic approach for mitochondrial DNA maintenance disorders

• Project leader: Ramon Martí, head of VHIR’s Neuromuscular and Mitochondrial Pathology group.
• Grant received: €50,000 under the 2026 CaixaImpulse Health Innovation call.
• Consortium project with: Marçal Pastor Anglada, Universitat de Barcelona (UB).

Mitochondrial DNA maintenance disorders (MDDS) constitute a group of severe rare diseases that alter the quantity and quality of mitochondrial DNA, causing very serious impairments, often from childhood. Although the first treatment for a disease in this group (TK2 deficiency) has recently been approved, most of these diseases still lack effective therapeutic alternatives.

The project led by VHIR aims to demonstrate the feasibility of a new metabolic strategy designed to increase the availability of certain nucleotides essential for mitochondrial DNA replication. This approach could pave the way for the development of treatments for other forms of MDDS, such as those associated with mutations in the POLG or DGUOK genes, for which no therapeutic options are currently available.

If the results are positive, this strategy could improve patients’ quality of life, reduce the care burden associated with these diseases and, in the future, expand the number of diseases that can be diagnosed and treated early.

Development of a new therapeutic strategy targeting the Hedgehog pathway in cancer

• Project leader: Josep Roma, principal investigator of VHIR’s Childhood Cancer and Haematological Diseases group.
• Grant received: €50,000 under the 2026 CaixaImpulse Health Innovation call.

The Hedgehog signalling pathway plays a key role in tumour growth, cancer progression and treatment resistance in numerous tumours. Although drugs targeting this pathway exist, their efficacy is limited in most tumours that depend on ligand-mediated activation, which account for more than one-third of human cancers.

The project led by VHIR aims to develop a new molecule capable of inhibiting this form of Hedgehog pathway activation through an innovative strategy targeting the CDO coreceptor. Building on a previously developed compound that had already demonstrated antitumour activity in preclinical models, the researchers will design new variants with improved pharmacokinetic properties to increase their clinical potential.

Initially, the project will focus on paediatric sarcomas, although this approach could be applied in the future to other aggressive tumours that share this biological mechanism.

Support for innovation and knowledge transfer

In addition to financial support, CaixaImpulse Innovation offers selected teams a support programme that includes mentoring, specialised advice and training in technology transfer, business development and research valorisation. The aim is to facilitate the transformation of scientific results into new solutions that can reach the healthcare system and generate a real impact on patients’ health.