Shire Human Genetic Therapies, Inc.

Takeda Pharmaceutical Company Limited

Transkaryotic Therapies, Inc.

Advanced Technologies and Regenerative Medicine, LLC

Peter Francis Daniel

Marianne Borowski

Carol M Kinoshita

Mishra Prashsant

Michael W Heartlein

Douglas A Treco

Richard F Selden

Dongling Su

Melanie D Williams

Prashant Mishra

Thomas J Schuetz

Asok C Sen

Carol A Kinoshita

Methods and compositions for treating Gaucher disease are described.

Compositions and methods for treating Gaucher disease

The invention features a method of producing a high mannose glucocerebrosidase (hmGCB) which includes: providing a cell which is capable of expressing glucocerebrosidase (GCB), and allowing production of GCB having a precursor oligosaccharide under conditions which prevent the removal of at least one mannose residue distal to the pentasaccharide core of the precursor oligosaccharide of GCB, to thereby produce an hmGCB preparation. Preferably, the condition which prevents the removal of at least one mannose residue distal to the pentasaccharide core is inhibition of a class 1 processing mannosidase and/or a class 2 processing mannosidase. The invention also features an hmGCB preparation and methods of using an hmGCB preparation.

High mannose proteins and methods of making high mannose proteins

Improved compositions and methods are provided for stabilizing a solution of bone morphogenetic protein. The compositions comprise an aqueous solution of GDF-5 and a biocompatible acid, such as hydrochloric, acetic, phosphoric, or trifluoroacetic acid, wherein the solution has a pH of from about 3.0 to about 3.6, thereby providing for improved stability of the GDF-5 protein during handling and prolonged storage at reduced temperatures.

Stable composition of GDF-5 and method of storage

The invention provides highly purified &agr;-Gal A, and various methods for purifying it; &agr;-Gal A preparations with altered charge and methods for making those preparations; &agr;-Gal A preparations that have an extended circulating half-life in a mammalian host, and methods for making same; and methods and dosages for administering an &agr;-Gal A preparation to a subject.

Treatment of a &agr;-galactosidase a deficiency

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Natick, MA, United States of America

Peter Francis Daniel