Icahn School of Medicine at Mount Sinai

Genzyme Corporation

Mount Sinai School of Medicine

Other

The Weizmann Institute of Science

Wylder Nation Foundation

University of Pennsylvania

The Mount Sinai Medical Center

Universitaet Duisberg-essen

Yeda Research and Development Co. Ltd. at the Weizmann Institute of Science

Edward Howard Schuchman

Robert J Desnick

Gerald F Cox

Laura P Andrews

James M Murray

Richard N Kolesnick

Vladimir R Muzykantov

Anthony Futerman

Erich Gulbins

Silvia Muro Galindo

Adrian Bartoll

Maria Dolores Ledesma

Yael Pewzner-Jung

Calogera M Simonaro

Helen Vlassara

Gary E Striker

Efrat Eliyahu

Nataly Shtraizent

Xingxuan He

The present invention provides, inter alia, compositions and methods for using CB1 cannabinoid receptor agonists, or other compounds capable of increasing endocannabinoids or endocannabinoid signaling, for treating and preventing lysosomal storage disorders in which lipid storage occurs (including, e.g., disorders associated with sphingomyelin accumulation). In particular embodiments, the present invention provides compositions and methods for treating such lysosomal storage disorders with one or more fatty acid amide hydrolase inhibitor alone or in combination with one or more additional agent.

Compositions and methods for activating signaling through the CB1 cannabinoid receptor for treating and preventing diseases and disorders characterized by abnormal cellular accumulation of sphingolipids such as sphingomyelin

The present invention relates to a therapeutic composition including a ceramidase mixture and a pharmaceutically acceptable carrier, where the ceramidase mixture includes an inactive acid ceramidase precursor and an active acid ceramidase. The invention also relates to a method of acid ceramidase treatment, including formulating the acid ceramidase used in said treatment as a ceramidase mixture, where the ceramidase mixture includes an inactive acid ceramidase precursor and an active acid ceramidase. The invention further relates to a method of producing a therapeutic composition including providing a medium containing an inactive acid ceramidase precursor; incubating the medium under conditions effective to transform a portion of the inactive acid ceramidase precursor to active acid ceramidase; and recovering the incubated medium as a ceramidase mixture comprising the inactive acid ceramidase precursor and an active acid ceramidase. The present invention also relates to preparation of a therapeutic composition of a ceramidase lacking acid sphingomyelinase.

Therapeutic acid ceramidase compositions and methods of making and using them

The present invention relates to methods of producing chondrocytes, improving the phenotype of a chondrocyte population, promoting chondrogenesis, maintaining a cell population in a differentiated state or increasing the number of cells of a population in a differentiated state, producing a population of differentiated cells, and treating certain diseases or disorders. Each of these methods involves treating a cell population with, and/or culturing a cell population in, a ceramidase.

Ceramidase and cell differentiation

The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.

Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency

The present invention relates to a method for treating or preventing pathogenic infections in a subject having Cystic Fibrosis, COPD, and/or an open wound. This method involves selecting a subject having Cystic Fibrosis, COPD, and/or an open wound and administering to the selected subject a ceramidase under conditions effective to reduce ceramide and to treat or prevent the pathogenic infection. The method also involves the use of a ceramidase in combination with other drugs to reduce infection, reduce ceramide, or improve lung function in Cystic Fibrosis, COPD, and/or open wound patients.

Ceramide levels in the treatment and prevention of infections

The present invention relates to compositions and methods for delivering lysosomal proteins. The compositions and methods described herein permit the targeted delivery of exogenous lysosomal proteins to cell surface proteins that allow their internalization via non-clathrin pathways. The present invention further relates to the use of the compositions and methods for enzyme replacement therapy of lysosomal storage diseases. Nucleic acids, recombinant cells and kits useful for making and using the compositions of the invention are also provided.

Targeted protein replacement for the treatment of lysosomal storage disorders

The present invention relates to methods of treating a subject with a lysosomal disorder, by administering an agent for enzyme replacement therapy and an agent for anti-TNF-&#x3b1; therapy; by administering a pentosan polysulfate therapy; or by administering a substrate reduction therapy and an anti-TNF-&#x3b1; therapy. The invention further relates to a method of reducing inflammatory cytokines in a subject with a lysosomal disorder that is being treated by enzyme replacement therapy, by administering an agent for anti-TNF-&#x3b1; therapy.

Anti-TNF-&#x3b1; therapy for the mucopolysaccharidoses and other lysosomal disorders

The present invention relates to methods of promoting the survival of cells by treating the cells with acid ceramidase. A kit for promoting ex vivo cell survival is also disclosed, as is a method of predicting in vitro fertilization outcome of a female subject.

Acid ceramidase and mammalian cell survival

Growing community of inventors

Haworth, NJ, United States of America

Edward Howard Schuchman