For a long time there have been no treatments for Friedreich’s ataxia. With no FDA approved therapies, people with this disease have only been receiving symptomatic treatment. Exicure has changed this with their new technology, which uses spherical nucleic acid (SNA). With this new treatment they have also created a neurological development program. They have partnered with the Friedreich’s Ataxia Research Alliance (FARA), and both organizations hope to improve the lives of those with this disease. Along with their work on this new program, Exicure is also expanding their Scientific Advisory Board in the hopes that new minds will allow their company to improve further.
About Friedreich’s Ataxia (FRDA)
Friedreich’s ataxia (FRDA) is a rare, genetic, neurological disease that is characterized by movement and neurological issues. It affects one of every 40,000 people in the United States, and it affects males and females equally. Symptoms typically begin to present themselves between the ages of five and 15. Those who do not experience symptoms until 25 or after have late-onset FRDA, and people who do not experience symptoms until 40 have very late-onset FRDA. The latter tend to see a slower progression of the disease.
A mutation in the FXN gene causes FRDA. It produces frataxin, which is necessary for normal function of the mitochondria. This mutated gene is inherited in an autosomal recessive pattern, meaning that both parents must pass down the mutation in order for their child to have Friedreich’s ataxia.
FRDA is a progressive disease. People may experience difficulties with moving and slurred speech at first, but may eventually require a wheelchair. Other symptoms include fatigue, abnormalities in the feet, cardiac arrhythmia, chest pain, shortness of breath, scoliosis, issues with hearing and vision, diabetes mellitus, poor movement coordination, and slurred speech.
After these symptoms are noticed, a diagnosis is obtained through genetic testing. Doctors will examine the FXN gene to find the mutation that results in FRDA. After one is diagnosed, treatment will be symptomatic. It includes genetic counseling, aids for walking and mobility, surgical or non-surgical therapies for the spine, heart medications, aids or medicine for hearing and vision, psychotherapy, and speech therapy.
About Exicure’s New Program
Exicure has partnered with the Friedreich’s Ataxia Research Alliance (FARA) in order to have some of the best minds work on their neurological development program. They will use Exicure’s new spherical nucleic acid (SNA) technology, which is supported by preclinical data. This technology works to target the mutated gene, and has been tested in rodents and primates. In these models the SNA technology was shown to distribute to all parts of the brain, including the regions associated with FRDA.
Along with creating this new program, Exicure is also expanding their Scientific Advisory Board to include new experts in neurology: Dr. Susan Perlman, who is the Medical Director of the National Ataxia Foundation, and Dr. Hank Paulson, who is a professor in the Department of Neurology at the University of Michigan. They hope that their innovations, including the SNA technology, will continue to improve with the help of these new brilliant minds.
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