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Bridget Bax
64 years
en
About me
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).
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Education
PhD (Medicine) Royal Postgraduate Medical School, UK
BSc (Biochemistry) Royal Holloway College, UK
Experience
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.
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Publications
- Bax, B.E (2017) Drug Development for Rare Diseases: Challenges and Regulatory Initiatives. Archives of Science, 1:2 - mitochondrial-myopathy - https://www.omicsonline.org/open-access/drug-development-for-rare-diseases-challenges-and-regulatory-initiatives.pdf
- Chapman, T.P., Hadley, G., Fratter, C., Cullen, S., Bax, B.E., Bain, M.D., Sapsford, A., Poulton J., and Travis, S.P.L. (2014) Unexplained gastrointestinal symptoms: Think mitochondrial myopathy. Digestive and Liver Disease. 46, 1-8. - mitochondrial-myopathy - https://www.ncbi.nlm.nih.gov/pubmed/23768727
- Bax, B.E., Bain, M.D., Scarpelli, M., Filosto, M., Tonin P., and Moran, N. F. (2013) Clinical and biochemical improvements in a patient with MNGIE following enzyme replacement. Neurology. 8, 1269-1271. - mitochondrial-myopathy - https://www.ncbi.nlm.nih.gov/pubmed/23966250
- Levene, M., Coleman, D., Kilpatrick, H., Fairbanks, L., Gangadharan, B., Gasson, C., and Bax, B.E. (2013) Preclinical toxicity evaluation of erythrocyte-encapsulated thymidine phosphorylase in BALB/c mice and Beagle dogs: an enzyme replacement therapy for - mitochondrial-myopathy - https://www.ncbi.nlm.nih.gov/pubmed/22977166
- Scarpelli, M., Russignan, A., Zombor, M., Bereczki, C., Zappini, F., Buono, R., Bax, B.E., Padovani, A., Tonin P., and Filosto, M (2012) . Poor outcome in a MNGIE Patient with a novel TYMP mutation: the need for early diagnosis. Case Reports in Neurolo - mitochondrial-myopathy - https://www.ncbi.nlm.nih.gov/pubmed/23341816
- Bourgeaux, V., Lanao, J.M., Bax, B.E., and Godfrin, Y. (2016) Drug-loaded erythrocytes: on the road toward marketing approval. Drug design Development and Therapy. 10, 665-676. - mitochondrial-myopathy - https://www.ncbi.nlm.nih.gov/pubmed/26929599