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Message: LSD enzyme fusion proteins

Armagen has four lysosomal storage disease (LSD) enzyme therapies in their pipeline for their LSD enzyme/insulin receptor monoclonal antibody protein fusions. These have all been shown in Rhesus studies to achieve either ~1% of injected dose per brain or ~1% of injected dose per 100 mg of brain. Rhesus brain weight varies between ~85 and 100 g according to this reference. So is this our benchmark for the PET imaging that biOasis plans to perform to confirm biodistribution in non-human primate models?

AGT-181 Hurler syndrome (MPS I), a-L-iduronidase (IDUA) enzyme, currently in Phase 2 trial

AGT-182 Hunter syndrome (MPS II), idursulfase (IDS) enzyme, currently in Phase 1 trial

AGT-183 Metachromatic leukodystrophy, arylsulfatase A (ASA) enzyme, pre-IND status

AGT-184 Sanfilippo A, N-sulfoglucosamine sulfohydrolase (SGSH) enzyme, also called sulfamidase, pre-IND status

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4137762/

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3919501/

https://www.ncbi.nlm.nih.gov/pubmed/24059813

https://www.ncbi.nlm.nih.gov/pubmed/17680664

 

 

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