Yeda Research and Develpment & Co. Ltd

The Weizmann Institute of Science

Icahn School of Medicine at Mount Sinai

Tel Aviv University

The Medical Research, Infrastructure and Health Services Fund of the Tel Aviv Medical Center

University of Duisburg-essen

Universitaet Duisberg-essen

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

The Medical Research and Infrastructure Fund of the Tel-aviv Sourasky Medical Center

Anthony Futerman

Edward Howard Schuchman

Erich Gulbins

Yael Pewzner-Jung

Naftali Stern

Ari Zimran

Israel Silman

Joel L Sussman

Shengrong Li

Ruth Navon

Michal Harel

Hay Dvir

Etty Osher

Lilly Toker

Stephen W Burgess

Jacques Bodennec

Dori Pelled

Tammar Joseph

Walter A Shaw

Provided herein is a group of sphingosine analogs, useful in the treatment of bacterial infections in mucosal surfaces such as lungs, caused by pathogenic bacteria which are typical to disorders and diseases such as CF and COPD.

Sphingosine analogs and use thereof against bacterial lung infections

The present invention relates in general to the field of enzyme replacement therapy, and specifically to chimeric proteins including protein hormone-therapeutic protein conjugates and fusion proteins, wherein the protein hormone is selected from a protein hormone which is able to cross the blood brain barrier, for the treatment of lysosomal storage diseases, compositions of the same and to methods of use thereof.

Enzyme replacement therapy for treating lysosomal storage diseases

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

A method of identifying a compound capable of correcting an impaired enzymatic activity of a mutant glucocerebrosidase molecule, the method comprising: (a) obtaining a first set of structure coordinates, the first set of structure coordinates defining a 3D structure of a glucocerebrosidase molecule capable of displaying normal enzymatic activity or a portion thereof; (b) computationally generating using the first set of structure coordinates a second set of structure coordinates, the second set of structure coordinates defining a predicted 3D structure of the mutant glucocerebrosidase molecule or a portion thereof; and (c) computationally identifying, using the second set of structure coordinates, a compound capable of interacting with the mutant glucocerebrosidase molecule in such a way as to correct the impaired enzymatic activity thereof, thereby identifying the compound capable of correcting the impaired enzymatic activity of the mutant glucocerebrosidase molecule. A glucocerebrosidase preparation comprising a population of glucocerebrosidase molecules, wherein substantially each of said glucocerebrosidase molecules: (i) has an amino acid sequence at least 95 percent homologous to an amino acid sequence set forth by SEQ ID NO: 1 or 8; (ii) is glycosylated at, or has an aspartatic acid residue at, glycosylation residue 1 of said amino acid sequence; and (iii) is independently unglycosylated at one or more glycosylation residues selected from the group consisting of glycosylation residues 2, 3 and 4 of said amino acid sequence.

Partially deglycosylated glucocerebrosidase polypeptide and crystals thereof

The present invention discloses methods of screening for identification of compounds that inhibit novel targets in the enzymatic pathway of phospholipid synthesis that are related to glycolipid storage diseases, and use of the compounds for treating patients affected with glycolipid storage diseases, particularly Gaucher disease. Specifically, the compounds of the present invention are intended to inhibit the accumulation of phosphatidylcholine (PC), wherein PC accumulation is increased due to the activation of CTP:phosphocholine cytidylyltransferase (CCT) upon glucosylceramide (GlcCer) accumulation.

Methods of screening for inhibitors of phospholipid synthesis related to glycolipid-storage diseases

Growing community of inventors

Rehovot, Israel

Anthony Futerman