December 23, 2018 mcfadyena

$150,000 Awarded to Dr. Brian Bigger for MPS II Gene Therapy Research!

Our MPS II Research Fund and the entire Isaac Foundation is always on the lookout for hopeful research and researchers in MPS II. Dr. Brian Bigger’s work is just that. We are THRILLED to announce that we just granted $150,000 to Bigger’s Lab in the UK and we are hopeful that this will lead to anther gene therapy option for boys and men with MPS II. Please read below for details of Biggers’ work:

“The Bigger lab have developed several gene therapies for neurodegenerative lysosomal storage diseases including most recently Mucopolysaccharidosis (MPS) II (Hunter), MPSIIIA, MPSIIIB and MPSIIIC. Most patients have no treatments, and where treatments do exist, they are ineffectual in the brain.  Delivery of replacement enzyme via haematopoietic stem cell gene therapy (HSCGT) using a lentiviral vector can effectively target the brain, correcting disease.

We recently developed a stem cell gene therapy that can target the brain in Hunter disease, via a lentiviral vector expressing a blood brain barrier crossing peptide coupled to the IDS gene (LV-IDS-APOEII). We have successfully performed a proof-of-concept study in MPSII mice demonstrating correction of disease (Gleitz 2018 Embo Mol Med) and superiority of the LV-IDS-APOEII vector over the normal enzyme.

We obtained funding for an InnovateUK Manchester Advanced Therapies Centre Hub (iMATCH), a consortium of academic and industry partners with a co-ordinated strategy to scale up gene therapies for patients in Manchester. Our remit on iMATCH is to develop scaled up GMP transduction of haematopoietic stem cells, but vector production was excluded from the call.

The grant from the Isaac foundation will allow us to purchase GLP grade IDS-APOEII (Hunter disease genome) plasmid from plasmid factory to enable production of a large-scale GMP-like lentiviral vector for Hunter disease, which would both validate our large-scale transduction procedure in CD34+ cells for iMATCH and provide data for a potential filing for phase I/II clinical trial.”

 If you have questions, please reach don’t hesitate to contact us!


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