Bridget Bax

66 Anos

en

Sobre mim

I am a researcher in rare diseases at St George's, University of London. My research focuses on two objectives: i) to improve the understanding of the underlying pathogenic molecular mechanisms of rare inherited diseases and ii) to develop cell-based therapies for diseases with unmet needs. Currently my team are focusing on the fatal, ultra-rare disease, mitochondrial neurogastrointestinal encephalomyopathy (MNGIE).

Contato

PhD (Medicine) Royal Postgraduate Medical School, UK

BSc (Biochemistry) Royal Holloway College, UK

I have  over 20 years of experience in developing the autologous erythrocyte as a vehicle for delivering enzyme replacement therapies and other therapeutic proteins.  Our team have employed this therapeutic approach in the clinical setting for over 18 years in the treatment of an adult patient with adenosine deaminase deficiency. For the past 7 years, this platform technology has been applied to the compassionate treatment of patients with MNGIE, in the form of erythrocyte encapsulated thymidine phosphorylase  (EE-TP).  Orphan Drug Designation was granted for EE-TP by both the Food and Drug Administration (USA ) and European Medicines Agency, and a European clinical trial of EE-TP (funded by the MRC) is due to commence in the second quarter of 2018.  The technology was exclusively licensed to Orphan Technologies by St. George’s University of London in 2012 and three patents for the treatment of MNGIE have been filed in the UK, Europe and the USA.