Friedreich’s Ataxia is a rare neurodegenerative disease of genetic origin, which currently has no cure and affects 1 in 30,000 people in the world, usually appearing between 5 and 20 years of age. This condition is a consequence of a functional deficit of the frataxin protein, which leads to cardiac and neurological disorders, which end up sending patients to a wheelchair for life.
Biointaxis is developing the first AAV vector gene therapy for the treatment of Friedreich’s Ataxia. Unlike other solutions, they have demonstrated outstanding preclinical data, managing to provide functional frataxin levels in the affected tissues with a limited number of administrations, thus eliminating the need for chronic treatment, and showing potential to become a genuine cure for the disease.
Orphan diseases have become one of the most active areas of investment in biotechnology; The tax and regulatory facilities enjoyed by drug development projects against these diseases are so powerful that Orphan Drugs were the 4th biopharma sector with the most venture capital investment in 2020.
Since the last round, BIOINTAXIS has manufactured the first scalable batch of the biological drug BTX-101 under GLP quality standards. It has validated it in two mouse animal models, one chronic and one acute of the disease, as well as in non-human primates. With these results, the objective of this round and of 2024 is to validate the regulatory plan with the European Medicines Agency (EMA) and the United States Agency for Drugs (FDA), and the designation of orphan drug will be requested. The objective is to start the IND process in 2026, to evaluate the safety and efficacy of the drug in humans (Phase I / IIa).
