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The development of a gene therapy for Friedreich’s ataxia receives €2.17 million in new public funding

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The Spanish Ministry of Science, Innovation and Universities (MICIU), through the Public-Private Collaboration R&D&I program, has granted €1.7 million for the development of a gene therapy aimed at Friedreich's ataxia. Biointaxis -a spin-off of the Germans Trias i Pujol Research Institute (IGTP)- is collaborating with IGTP and the research institutes of Hospital Universitario La Paz (IDIPAZ) and Hospital Universitari de Girona Dr. Josep Trueta (IDIBGI) on this project to bring the therapy to a cohort of patients, including paediatric patients.

The project aims to develop and validate a gene therapy vector based on a new generation of adeno-associated virus, with the goal of offering a curative treatment for patients affected by Friedreich's ataxia, a neurodegenerative disease that severely impacts mobility and personal autonomy. Since its founding in 2018 as a biotech spin-off, Biointaxis has focused its efforts on developing FRATAXAV®, a gene therapy drug that can reach the nervous system and peripheral organs, including the heart, with the aim of halting the progression of the disease and restoring movement abilities in patients.

In this regard, the first candidate vector to be developed, FRATAXAV®, has successfully passed the initial in vivo preclinical proof-of-concept tests in mice, and has shown excellent safety and biodistribution profiles in non-human primates, using doses equivalent to clinical efficacy levels. These results have enabled Biointaxis to move forward with the drug's validation, with the goal of advancing it to the clinical trial phase in patients. "Our aim is to prevent the onset, halt the progression of the disease, and restore movement abilities in affected patients, with the ultimate goal of alleviating the ataxia and cardiomyopathy, the primary symptoms of this condition", explained Antoni Matilla, leader of the Neurogenetics research group at IGTP and CEO of Biointaxis. "The financial support from the Spanish Ministry will allow us to consolidate the drug's development and prepare its transition to clinical trials in patients. I am confident that we will begin the clinical trial in 2026".

Additionally, the European Commission has granted Biointaxis €466,000 to evaluate its gene therapy in induced pluripotent stem cells (iPSCs) and 3D organoids, both neural and cardiac, from patients with Friedreich's ataxia. This funding will enable the validation of proprietary biomarkers, which will strengthen the evaluation of the therapy in clinical phases.

With the recent results obtained in preclinical animal studies, both in mice and primates, Biointaxis will soon initiate regulatory procedures with the European (EMA) and American (FDA) agencies to apply for orphan drug designation and authorisation to begin clinical trials. The company plans to start evaluating the gene therapy in clinical trials with Friedreich's ataxia patients in 2026.