According to an announcement from the Muscular Dystrophy Association, the organization has recently awarded a total of $1,076,232 in MDA Venture Philanthropy (MVP) funding towards the drug company AavantiBio, which is in the process of testing an experimental gene therapy as a treatment for Friedreich’s ataxia, a rare, progressive, genetic disorder. The money more specifically will go towards the company’s phase 2 clinical trial. AavantiBio was created with the goal of creating a more effective treatment for the disease.
About Friedreich’s Ataxia
Friedreich’s ataxia (FRDA) is a neuromuscular genetic disorder which is characterized by abnormal gait due to the degeneration of nerve tissue in the spinal cord. The disorder is linked to a genetic mutation of the FXN gene. The degeneration of the nerves causes the spinal cord to become thinner and neurons begin to lose some of their myelin sheath, a fatty insulating layer that protects these cells and is necessary for normal function. Symptoms of Friedreich’s ataxia include muscle weakness affecting the limbs, heart disorders, poor coordination, diabetes, high foot arches, vision and hearing problems, scoliosis, and slurred speech. Symptoms begin to appear at 7-10 years and the disorder progresses slowly overall. Management strategies for the disorder may include ACE inhibitors, surgery, speech and rehabilitation therapy, and orthoses. Life expectancy is around 50 years. Heart problems are the leading cause of death. To learn more about Friedreich’s ataxia, click here.
MVP and Drug Funding
MVP is a special funding program from the Muscular Dystrophy Association that is specifically allocated towards supporting promising and relevant drug development. The organization seeks out both non-profit and for-profit ventures that are focused on developing therapies for neuromuscular disorders. Gene therapies have gained a lot of notoriety and attention recently. These therapies have the potential to profoundly advance the successful treatment of a diverse array of genetic disorders. The approval of Zolgensma, a gene therapy for spinal muscular atrophy, has spearheaded the arrival of this new class of treatments.
When taking these factors into consideration, it is pretty clear the a gene therapy for Friedreich’s ataxia would completely revolutionize patient outcomes for the disorder. AavantiBio’s clinical trial is expected to begin later this year.
