Cincinnati Children's Hospital Medical Center

Alexion Pharmaceuticals, Inc.

Children's Hospital Research Foundation

Inozyme Pharma, Inc.

Gregory A Grabowski

Anthony Quinn

Tayeba Khan

Kim Lynette Askew

Nelson Hsia

Benjamin Liou

Zhiliang Cheng

W Charles O'Neill

Hong Du

The present invention provides a method of treating NPP1 deficiency or NPP1-associated disease such as idiopathic infantile arterial calcification (IIAC), pseudoxanthoma elasticum, vascular calcification in chronic kidney disease (VCCKD), insulin resistance, hypophosphatemic rickets, myocardial ischemia, joint calcification, angioid streaks, and ossification of the posterior longitudinal ligament of the spine. The present invention provides a method for treating tissue calcification by administering soluble NPP1 to produce a transient increase in serum pyrophosphate levels.

Methods of treating tissue calcification

The present invention comprises a method to diminish and/or eliminate atherosclerotic plaques, in mammals, through direct and indirect treatment of these plaques, in situ, using suitable substances which are capable of lipid removal, primarily through hydrolysis, either by a catalytic or stoichiometric process, wherein the substance targets receptors in and/or on the cell which lead to uptake into the lysosome. Such substances used to diminish and/or eliminate atherosclerotic plaques are generally comprised of lipid hydrolyzing proteins and/or polypeptides.

Lipid hydrolysis therapy for atherosclerosis and related diseases

The present disclosure relates to methods of treating diseases states such as lysosomal storage diseases and/or neurodegenerative diseases. Also disclosed are one or more compositions that may be useful for one or more of the disclosed methods, including compositions that may comprise acid &#x3b2;-glucosidase (GCase) protein comprising one or more mutations, peptides of acid &#x3b2;-glucosidase, and DNA vectors and cell lines related to acid &#x3b2;-glucosidase peptides or proteins.

Lysosomal protein targeting sequence and therapeutic applications of same

Growing community of inventors

Wheat Ridge, CO, United States of America

Gregory A Grabowski