{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T13:34:07Z","timestamp":1790775247033,"version":"4.1.0"},"reference-count":20,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2019,2,11]],"date-time":"2019-02-11T00:00:00Z","timestamp":1549843200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Greenovation Biotech GmbH"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2019,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Moss\u2010aGalactosidase A (moss\u2010aGal) is a moss\u2010derived version of human \u03b1\u2010galactosidase developed for enzyme replacement therapy in patients with Fabry disease. It exhibits a homogenous N\u2010glycosylation profile with &gt;90% mannose\u2010terminated glycans. In contrast to mammalian cell produced \u03b1\u2010galactosidase, moss\u2010aGal does not rely on mannose\u20106\u2010phosphate receptor mediated endocytosis but targets the mannose receptor for tissue uptake. We conducted a phase 1 clinical trial with moss\u2010aGal in six patients with confirmed diagnosis of Fabry disease during a 28\u2010day schedule. All patients received a single dose of 0.2 mg\/kg moss\u2010aGal by i.v.\u2010infusion. Primary endpoints of the trial were safety and pharmacokinetics; secondary endpoints were pharmacodynamics by analyzing urine and plasma Gb3 and lyso\u2010Gb3 concentrations. In all patients, the administered single dose was well tolerated. No safety issues were observed. Pharmacokinetic data revealed a stable nonlinear profile with a short plasma half\u2010life of moss\u2010aGal of 14\u2009minutes. After one single dose of moss\u2010aGal, urinary Gb3 concentrations decreased up to 23% 7 days and up to 60% 28\u2009days post\u2010dose. Plasma concentrations of lyso\u2010Gb3 decreased by 3.8% and of Gb3 by 11% 28\u2009days post\u2010dose. These data reveal that a single dose of moss\u2010aGal was safe, well tolerated, and led to a prolonged reduction of Gb3 excretion. As previously shown, moss\u2010aGal is taken up via the mannose receptor, which is expressed on macrophages but also on endothelial and kidney cells. Thus, these data indicate that moss\u2010aGal may target kidney cells. After these promising results, phase 2\/3 clinical trials are in preparation.<\/jats:p>","DOI":"10.1002\/jimd.12052","type":"journal-article","created":{"date-parts":[[2019,2,12]],"date-time":"2019-02-12T05:06:22Z","timestamp":1549947982000},"page":"527-533","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":63,"title":["Pharmacokinetics, pharmacodynamics, and safety of moss\u2010aGalactosidase A in patients with Fabry disease"],"prefix":"10.1002","volume":"42","author":[{"given":"Julia B.","family":"Hennermann","sequence":"first","affiliation":[{"name":"Villa Metabolica, Department of Pediatric and Adolescent Medicine University Medical Center Mainz  Mainz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laila","family":"Arash\u2010Kaps","sequence":"additional","affiliation":[{"name":"Villa Metabolica, Department of Pediatric and Adolescent Medicine University Medical Center Mainz  Mainz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gy\u00f6rgy","family":"Fekete","sequence":"additional","affiliation":[{"name":"II. 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