Discoverybiomed, Inc.

Johns Hopkins University

The UAB Research Foundation (The University of Alabama at Birmingham)

Erik Schwiebert

John Dixon

John Streiff

Hongwu Gao

William B Guggino

Joseph P Ritchie

Eric C Seales

Deborah Mai

Lihua Liang

Akos Zsembery

Coumarin derivative compounds and methods for the treatment of hyperproliferative diseases, such as cancer, polycystic kidney disease, and fibrosis of different tissues (e.g., idiopathic pulmonary fibrosis), are provided. The methods include administering to a subject a compound as described herein. Also provided are methods for inhibiting the interaction between two or more heat shock protein chaperones in a cell.

Coumarin derivatives and methods of use in treating hyperproliferative diseases

Novel CFTR corrector compounds that are effective in rescuing halide efflux, delF508-CFTR protein processing, and apical functional chloride ion transport in a cell are provided. Also provided are methods for treating protein folding disorders (e.g., cystic fibrosis and chronic obstructive pulmonary diseases). The methods include administering a CFTR corrector compound or pharmaceutically acceptable salt or prodrug thereof. Methods of rescuing halide efflux in a cell, correcting a processing defect of a delF508-CFTR protein in a cell, and correcting functional delF508-CFTR chloride channels in a cell are also provided.

Coumarin derivatives and methods of use in treating cystic fibrosis, chronic obstructive pulmonary disease, and misfolded protein disorders

Novel CFTR corrector compounds that are effective in rescuing halide efflux, delF508-CFTR protein processing, and apical functional chloride ion transport in a cell are provided. Also provided are methods for treating protein folding disorders (e.g., cystic fibrosis). The methods include administering a CFTR corrector compound or pharmaceutically acceptable salt or prodrug thereof. Methods of rescuing halide efflux in a cell, correcting a processing defect of a delF508-CFTR protein in a cell, and correcting functional delF508-CFTR chloride channels in a cell are also provided.

Small molecule CFTR correctors

Small molecule bicyclic and tricyclic CFTR correctors

Novel CFTR corrector compounds that are effective in rescuing halide efflux in a cell are provided. Also provided are methods for treating protein folding disorders (e.g., cystic fibrosis). The methods include administering a CFTR corrector compound or pharmaceutically acceptable salt or prodrug thereof. Methods of screening for CFTR corrector compounds are also described herein. The methods of screening include contacting a cell that endogenously expresses a CFTR mutation with the compound to be screened and detecting a rescue of halide efflux from the cell.

Treating protein folding disorders with small molecule CFTR correctors

Described herein are spiperone derivative compounds which have been found to be useful in methods of stimulating CaCC and treating disorders (or symptoms thereof) associated with CaCC including cystic fibrosis, wherein a compound of the invention is administered to a subject.

Spiperone derivatives and methods of treating disorders

This invention relates generally to a method of increasing cytosolic Calevels in mammalian cells comprising contacting P2X receptor Caentry channels or any and all other Caentry channels or mechanisms on the cell with an effective amount of a small molecule, and a composition comprising the small molecule in a delivery system. The invention has broad applicability in the pharmaceutical industry as a method of treating airway diseases (such as cystic fibrosis and asthma), ailments of the lung and airways (such as those caused by common cold pathogens or allergens in allergy), kidney diseases and renal hypertensive disorders (such as polycystic kidney disease and salt-sensitive hypertension syndromes), and endocrine disorders (such as diabetes).

Growing community of inventors

Birmingham, AL, United States of America

Erik Schwiebert