Biointaxis launches a 0.88M euro crowdfunding campaign to finance a curative therapy for Friedreich's Ataxia

- Innovation, Research

Biointaxis, a spin-off of the IGTP, has the main objective of developing of a unique treatment to cure Friedreich's Ataxia.

Friedreich's ataxia is a hereditary (genetic) progressive neurodegenerative disease that affects adolescents and young adults confining them to a wheelchair for life and causing severe heart deficits. It is caused by decreased levels of the mitochondrial protein frataxin, affecting essential functions in the spinal cord, cerebellum, heart, pancreas, and liver. Currently, there is nocure. In order to demonstrate the biodistribution and safety of BTX-101 in non-human primates, Biointaxis needs to raise 0.8M euros therefore it has launched an equity crowdfunding campaign through the Capital Cell platform.

"The therapy could be implemented in a few years if we reach our goal in this round of funding," explains Dr Matilla-Dueñas CEO and Co-founder of the company, a pioneer in the scientific research into ataxia and a specialist in neurodegenerative disorders. "Biointaxis is developing the gene therapy product, BTX-101, based on adeno-associated virus (AAV) that has demonstrated its effectiveness in two mouse models of the disease by restoring frataxin levels in vivo, preventing, arresting and reversing the neurological and cardiac deficits. associated with Friedreich's ataxia. BTX-101 is currently the only candidate with the potential to cure the disease" says Dr Ivelisse Sánchez, CSO co-founder of Biointaxis and IGTP Researcher.

The neurogenetics research group at the IGTP has been working on this project for more than seven years. Biointaxis' curative therapy consists of providing frataxin to the cells of the nervous system, and other tissues, to correct the deficits caused by the disease. The technology is protected by an international patent.

Friedreich's ataxia affects approximately 2-3 out in 100,000 people, more frequently in populations of European origin (Caucasians). In Spain it is the most frequent form of hereditary ataxias, with an estimated prevalence of 4.7 cases per 100,000 inhabitants.

The Germans Trias i Pujol Research Institute (IGTP)

The IGTP is a public research center accredited as a center of excellence by the Carlos III Health Institute. It is the umbrella for the research activity of the Germans Trias i Pujol Hospital located on the Can Ruti Campus in Badalona.

Until now, Dr Matilla and Dr Sánchez have developed this therapy at the IGTP in collaboration with Dr. Miguel Chillón, a researcher at the Universitat Autònoma Barcelona. The multinational pharmaceutical company Gentec, a shareholder in the company, supports the development of technology as well as the Fundación La Caixa and Caixa Capital Risc through the Caixa impulse program, the Center for Technological and Industrial Development (CDTI) through the Neotec program and the Ministry of Science and Innovation with the Retos-Collaboration project.