Biotech company ProQR start a research programme in Friedreich’s ataxia

Ataxia UK welcomes the news that Leiden-based biotech company ProQR are expanding their research interests and are now working on Friedreich’s ataxia. Dr Julie Greenfield, Ataxia UK’s Research Manager says ‘We first met with Sarah Hafith, ProQR’s business and operations manager, at the World Orphan drug conference in November 2014 and discussed the ataxias as a potential research area for companies to engage in. Having encouraged ProQR to attend the International Ataxia Research conference last year, we are delighted that they participated and indeed that the company is now dedicating their resources to working on Friedreich’s ataxia. ’ Dr Sarah Hafith says ‘‘We believe that RNA therapeutics hold a great opportunity for genetic diseases like Friedreich’s Ataxia, we are very excited about exploring applying our technology for Friedreich’s Ataxia patients as well’

Posted: 23/02/2016

A partnership between a biotech company and a manufacturing group working on gene therapies opens new avenues for the development of treatments for Friedreich's ataxia 

Agilis Biotherapeutics, LLC (Agilis), a biotechnology company advancing innovative gene therapies for rare genetic diseases that affect the central nervous system (CNS), and Waisman Biomanufacturing, a non-profit gene and cell therapy development and manufacturing group located at the UW-Madison Waisman Center, (Waisman) announced that the companies have entered into an exclusive partnership agreement for the production of Agilis’ novel gene therapy product, AGIL-FA, for the treatment of Friedreich’s ataxia. 

Posted: 22/02/2016