Redenlab selected to support a pivotal Phase 2a gene therapy trial in Friedreich ataxia
Redenlab Inc, the speech neuroscience company delivering insights in speech and language testing, announces that it has been selected by medium sized USA pharmaceutical company to support their upcoming gene therapy study in Friedreich ataxia (FA). Conducted across 5 sites in 3 countries over 2 years, the Phase 2a study will investigate the safety, tolerability and efficacy of gene therapy on adolescents with FA. For the study, Redenlab will provide advanced speech recording and analysis solutions.
Friedreich ataxia is a multisystem neurodegenerative disorder and the most common of the hereditary ataxia syndromes, with a prevalence of 0.5–3/100 000 individuals of western European lineage. It is caused by a triplet repeat expansion in the FXN gene, which leads to reduced frataxin, a mitochondrial protein important for iron metabolism. There is currently no treatment proven to alter its natural course. Symptoms typically present at 10–15 years of age but can be earlier or considerably later, and patients are usually wheelchair dependent within 10–15 years of disease onset. Other deficits include dysarthria and dysphagia, progressive limb and gait ataxia, optic and auditory neuropathy, scoliosis, and cardiomyopathy. For a patient perspective, read here : https://www.bmj.com/content/bmj/347/bmj.f7062.full.pdf
Adam Vogel PhD, Chief Science Officer of Redenlab, commented:
“Redenlab are excited to be part of the sponsors efforts to innovate and drive the field forward in this frontier clinical trials space. This recent award reflects the confidence our partners place in Redenlab as a trusted speech neuroscience company. With very limited efficacy data in FA for gene therapy, this first in human study presents a significant challenge and a huge advancement for trials in this disease group. We are proud to support the sponsors in their tireless efforts to bring cutting edge treatments to this devastating disease, and this recent award reflects the confidence our partners place in Redenlab as the trusted speech neuroscience company for their ataxia innovations.”