Genzyme Corporation

Placon Therapeutics, Inc.

University of Iowa Research Foundation

Tarveda Therapeutics, Inc.

Edward R Lee

David J Harris

Craig S Siegel

Seng H Cheng

John Marshall

Simon J Eastman

Ronald K Scheule

Cecilia M Bastos

Richard A Silva

Yibin Xiang

Cassandra Celatka

Frederic Vinick

Lisa M Cuff

Junkai Liao

Angeles Dios

John Kane

Mikaela Levine

Mark T Bilodeau

Thomas Henry Jozefiak

Nelson S Yew

Shirley C Hubbard

Mathieu B Lane

Benoît Moreau

Chau-Dung Chang

Rossitza Gueorguieva Alargova

Craig Antony Dunbar

Timothy E Barder

Rebecca G Bagley

Eric A Rowe

Rajesh R Shinde

Patrick Lim Soo

Adam H Brockman

Danielle N Rockwood

S Catherine Hubbard

H Michael Shepard

Michael J Welsh

Richard J Gregory

Daniel C Maneval

Canwen Jiang

Allen J Fasbender

Duane E Johnson

Mikaela N Keynes

Danille N Rockwood

Thomas H Jozafiak

Cassandra A Celatka

Yabin Xiang

The present disclosure relates to novel platinum compositions having a heterocycle ligand and nanoparticles and methods using such compositions, e.g. for treating cancer.

Platinum compounds having a heterocycle ligand, nanoparticles, and uses thereof

The present teachings relate to compounds and compositions for treatment of cancers. In some embodiments, the composition comprises a platinum (IV) complex having at least one carboxylate or carbamate ligand.

Compounds, compositions, and methods for the treatment of cancers

A compound for use in treating polycystic kidney disease is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating polycystic kidney disease in a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. Methods of treating in polycystic kidney disease in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

2-acylaminopropoanol-type glucosylceramide synthase inhibitors

A compound is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. The subject has type 2 diabetes; renal hypertrophy or hyperplasia associated with diabetic nephropathy; Tay-Sachs; Gaucher's; or Fabry's disease. Methods of decreasing plasma TNF-&#x3b1;, lowering blood glucose levels, decreasing glycated hemoglobin levels, inhibiting glucosylceramide synthase, and lowering glycosphingolipid concentrations in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

The present teachings relate to compounds and compositions for treatment of cancers. In some embodiments, the composition comprises a platinum compound having a heterocycle ligand. Cabazitaxel-platinum nanoparticles are disclosed herein, as well as methods of making and using the same.

Cabazitaxel-platinum nanoparticles and methods of using same

A compound for use in treating polycystic kidney disease is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating polycystic kidney disease in a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. Methods of treating in polycystic kidney disease in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. 

A compound is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. The subject has type 2 diabetes; renal hypertrophy or hyperplasia associated with diabetic nephropathy; Tay-Sachs; Gaucher's; or Fabry's disease. Methods of decreasing plasma TNF-&#x3b1;, lowering blood glucose levels, decreasing glycated hemoglobin levels, inhibiting glucosylceramide synthase, and lowering glycosphingolipid concentrations in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. 

Cationic amphiphiles for intracellular delivery of therapeutic molecules

Novel cationic amphiphiles are provided that facilitate transport of biologically active (therapeutic) molecules into cells. The amphiphiles contain lipophilic groups derived from steroids, from mono or dialkylamines, or from alkyl or acyl groups; and cationic groups, protonatable at physiological pH, derived from amines, alkylamines or polyalkylamines. There are provided also therapeutic compositions prepared typically by contacting a dispersion of one or more cationic amphiphiles with the therapeutic molecules. Therapeutic molecules that can be delivered into cells according to the practice of the invention include DNA, RNA, and polypeptides. Representative uses of the therapeutic compositions of the invention include providing gene therapy, and delivery of antisense polynucleotides or biologically active polypeptides to cells. With respect to therapeutic compositions for gene therapy, the DNA is provided typically in the form of a plasmid for complexing with the cationic amphiphile. Novel and highly effective plasmid constructs are also disclosed, including those that are particularly effective at providing gene therapy for clinical conditions complicated by inflammation. Additionally, targeting of organs for gene therapy by intravenous administration of therapeutic compositions is described.

Organ-specific targeting of cationic amphiphile / DNA complexes for gene therapy

Cystic fibrosis transmembrane conductance regulator (CFTR) is a chloride (Cl,) channel regulated by phosphorylation and intracellular nucleotides. CFTR is the major Cl,transport pathway in airway epithelial cells. The abnormal transepithelial Cl,transport and subsequent defective fluid transport caused by CF is a result of the genetic mutations of the gene coding for the CFTR protein. The present invention is directed to the novel use of ionophores as artificial Cl,transport pathways into CF epithelia to treat the defective Cl,and fluid transport lonophores and in particular, small molecule ionophores, represent a potential novel means of treating CF. The invention also includes using an ionophore to generate chloride secretion on intact monolayers of airway epithelia cells and other epithelia cells by administering an ionophore to a mammal.

Small molecule chloride transport

Organ-specific targeting of cationic amphiphile/DNA complexes for gene

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Sudbury, MA, United States of America

Edward R Lee