News

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…

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…

Avalanche Biotechnologies, Inc., announced that it has entered into a “definitive agreement” to acquire the French company Annapurna Therapeutics SAS. The merger, expected to close by mid-2016, has been approved by the boards of directors of both companies but is subject to approval by Avalanche stockholders. Under the agreement’s terms, Avalanche will acquire the totality…

Chondrial Therapeutics recently announced that further development of its lead drug candidate for the treatment of Friedreich’s ataxia, TAT-Frataxin (TAT-FXN), will be aided by researchers with the Therapeutics for Rare and Neglected Diseases (TRND) program, run through the National Institutes of Health (NIH), with the aim of filing an Investigational New Drug (IND) application for the therapy and…

Catabasis Pharmaceuticals, Inc., recently announced that it will receive the Kyle Bryant Translational Research Award, given by the Friedreich’s Ataxia Research Alliance (FARA), to further evaluate CAT-4001, a potential Friedreich’s ataxia (FA) drug. CAT-4001 is being developed by Catabasis as a potential treatment for neurodegenerative diseases, such as FA and amyotrophic lateral…

Corticosteroid therapy is a potential therapeutic treatment for patients with Friedreich ataxia (FRDA) and nephrotic syndrome, according to a study titled “Friedreich Ataxia and nephrotic syndrome: a series of two patients,” published in the journal BMC Neurology. FRDA is a genetic, progressive, neurodegenerative disorder that causes…

Bamboo Therapeutics, Inc., a biotechnology company focused on developing gene therapies for rare central nervous system and neuromuscular diseases, including Friedrich’s ataxia, announced that it has acquired the University of North Carolina (UNC) at Chapel Hill’s viral Vector Core gene therapy manufacturing facility. Financial details of the deal…