Pharmaceutical Recombinant Human Acid Sphingomyelinase Compositions and Methods

Disclosed here are compositions comprising recombinant acid sphingomyelinase (rASM) having desired purity, specific activity, and/or rASM isoforms. Also provided are methods for making and purifying such compositions, comprising chromatography steps. Further provided are methods of modulating rASM s...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Brower, Kevin, Wasylenko, Thomas M, Jin, Xiaoying
Format: Patent
Sprache:eng
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Beschreibung
Zusammenfassung:Disclosed here are compositions comprising recombinant acid sphingomyelinase (rASM) having desired purity, specific activity, and/or rASM isoforms. Also provided are methods for making and purifying such compositions, comprising chromatography steps. Further provided are methods of modulating rASM specific activity in a composition, and methods of modulating rASM isoforms in a composition. The methods disclosed here can be particularly useful for manufacturing pharmaceutical compositions comprising rASM for treating acid sphingomyelinase deficiency (ASMD).