Friedreich Ataxia (FRDA) is a devastating rare disease with early onset. FRDA is caused by a mutation in the frataxin gene, causing progressive neurodegeneration, cardiac atrophy and movement problems. The estimated prevalence is 2-4 per 100,000 people, with no treatment or therapeutic approach. Gene therapy vectors based on adeno-associated viruses (AAV) are becoming increasingly popular for their good safety profile, being used in more than 150 clinical trials to treat diverse inherited conditions.
- To develop the first-in-class therapy for FRDA by stopping, preventing or slowing the progression of the neurological symptoms of the disease by restoring the expression of the defective gene in patients with FRDA.
Problem to Solve
Friedreich Ataxia (FRDA) is a devastating rare disease with no available effective therapeutic approach to date.
Gene therapy based on AAV to restore frataxin levels in vivo, thus reversing, halting and preventing the symptoms associated with FRDA.
Level of InnovationThe approach proposed would be a first-in-class therapy to treat the neurological symptoms in FRDA, as no alternative exists at the present time. This gene therapy based on AAV has been pre-clinically tested for FRDA, and optimally targets the CNS.