The €25,000 contribution is allocated to the team led by Miguel Chillón, head of the Gene Therapy in the Nervous System group at the Vall d’Hebron Research Institute (VHIR).

The Gen Rebelde GNB1 Foundation is a non-profit organization driven by families affected by GNB1 encephalopathy and created with the aim of promoting scientific research, fostering collaboration among researchers, clinicians, and patients, and supporting families. Its activity is particularly focused on advancing research projects such as gene therapy and on generating initiatives that help accelerate the development of new therapeutic options for this rare disease.

The project, titled “Gene therapy for GNB1 encephalopathy,” is currently in an intermediate stage of development and focuses on the generation and validation of adeno-associated viral (AAV) vectors to address this ultra-rare neurodegenerative disease. The research team has already developed patient-derived cellular models and is currently working on validating an animal model that will allow the evaluation of safety, efficacy, and the molecular mechanisms involved in this potential therapeutic strategy.

The contribution from the Gen Rebelde Foundation allows further progress in developing this line of research, with the goal of laying the groundwork for a future therapy that can be applied to patients.

A donation driven by solidarity

“This contribution has been made possible thanks to the commitment of numerous individuals, companies, and organizations that have collaborated in various charitable initiatives organized over the past year,” adds Marc León, founder of the foundation.

Among them, the involvement of Levante Street Food and its community in Caños de Meca stands out, as organizers and volunteers of the third edition of the Levante Aid Fest. Contributors also include the promoters and collaborators of charity markets held in Valencia, Mallorca, and Cádiz, as well as various support activities for the foundation organized in Albacete and Murcia.

The Gen Rebelde Foundation expressed its gratitude to all those who have helped make this donation possible. “Every gesture counts. Every initiative drives research forward. And every step brings us closer to the future our children deserve,” the organization emphasized.

Collaboration between patient associations, civil society, and scientific institutions is key to advancing research into ultra-rare diseases such as GNB1 encephalopathy, where progress in knowledge depends largely on social support and solidarity initiatives like this one.