In the largest clinical trial to date, researchers observed that treatment of the alpha form of erythropoietin protein did not benefit Friedreich’s ataxia (FA) patients. The study, “Long-Term Effect of Epoetin Alfa on Clinical and Biochemical Markers in Friedreich Ataxia,” was published in the…
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The direct and indirect annual costs of care for Friedreich’s ataxia (FA) can be as high as £11,000 to £19,000 per person, according to a study titled “Impact of Friedreich’s Ataxia on health-care resource utilization in the United Kingdom and Germany,” published in BioMed Central’s open access journal…
Scientists believe that delivery of functional frataxin mRNA via RNA transcript therapy (RTT) can become a potential therapy for Friedreich’s ataxia patients. The study, “Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich’s ataxia,” was published in the journal Scientific Reports. Friedreich’s…
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FA Study Finds Deletion of Whole FXN Gene Copy in Patient, and Argues for Expanded Genetic Testing
Mutations in the FXN gene — responsible for Friedreich’s ataxia (FA) — mainly come in the form of repeats of three DNA bases, GAA, at the start of the gene. Cases where one gene copy holds a repeat mutation and the other copy contains another kind of mutation account for only 2 percent to 4 percent…
Agilis Biotherapeutics, LLC, and Waisman Biomanufacturing have signed an exclusive partnership agreement to develop and produce AGIL-FA, Agilis’ potential gene therapy for the treatment of Friedreich’s ataxia (FA). Agilis, a biotechnology company specializing in therapies for rare genetic diseases that affect the central nervous system (CNS), and Waisman, a nonprofit gene…
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Promising Friedreich’s Ataxia Therapy Seen in Synthetic RNA and DNA Agents Used by UT Researchers
University of Texas (UT) Southwestern Medical Center researchers have identified RNA and DNA synthetic agents that increase frataxin levels and alleviate Friedreich’s ataxia (FA), suggesting that synthetic nucleic acids that target the specific disease-causing mutation can be lead compounds for FA therapy. The research paper, “Activating frataxin expression by repeat-targeted nucleic acids,”…
Retrotope announced the completion of a first dosing cohort, and the opening of patient enrollment for a highest dose cohort, in its ongoing, 28-day clinical study of orally dosed RT001 in Friedreich’s ataxia (FA) patients. The treatment to date has been well-tolerated, with no serious adverse effects or dose limiting toxicities, the pharmaceutical company reported. “We…
The Illinois Biotechnology Industry Organization (iBIO) recently announced that Kyle Bryant, founder and director of Ride Ataxia, Friedreich’s Ataxia Research Alliance, will be one of the keynote speakers for the 2016 iBIO Industry Exposition (iBIO, IndEx) at the Hilton Orrington in Evanston, Illinois, April 19-20. The meeting, whose theme is “Converge to…
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In Friedreich’s Ataxia, Neurotrophins Hold Therapeutic Potential to Prevent, Treat Neurodegeneration
Researchers have demonstrated in the lab (in vitro) and in living subjects (in vivo), that gene transfer of brain-derived neurotrophic factor (BDNF) protected models of Friedreich’s ataxia from neuronal degenerative death, providing a proof of principle that neurotrophins might be a therapeutic option to prevent and treat neurodegeneration associated with Friedreich’s ataxia.
RaNA Therapeutics, a Cambridge, Massachusetts-based biotech company that develops next-generation RNA-targeted medicines and which currently has a lead program in Friedreich’s Ataxia, announced that its CEO Ronald Renaud will present a corporate overview on Feb. 8 at the 18th annual BIO CEO & Investor Conference in New York City. RaNA’s management will also…
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