For new Rhode Island firm, peptides hold hope for disease-altering cystic fibrosis treatments

It’s been nearly two and a half decades since the gene associated with the inherited disease cystic fibrosis was discovered, paving the way for disease-modifying drugs. In that time, only one such drug has been approved in the U.S. and Europe – Vertex’s Kalydeco. And that drug is only Continue reading For new Rhode Island firm, peptides hold hope for disease-altering cystic fibrosis treatments

Cystic Fibrosis: New Assay May Help Individualize Care

A rapid and quantitative assay for assessing function of the CFTR gene using intestinal organoids has been developed, according to a study conducted by Johanna F. Dekkers, MS, from the University Medical Center in Utrecht, the Netherlands, and colleagues. Continue reading Cystic Fibrosis: New Assay May Help Individualize Care

Proteostasis Therapeutics Presents Preclinical Data on Parkinson’s Disease and Cystic Fibrosis Targets at EMBO Conference

Proteostasis Therapeutics, Inc., a company developing novel therapeutics that regulate protein homeostasis to improve outcomes for patients with neurodegenerative and orphan diseases, today presented data on the Company’s preclinical targets for Parkinson’s disease and cystic fibrosis at a Continue reading Proteostasis Therapeutics Presents Preclinical Data on Parkinson’s Disease and Cystic Fibrosis Targets at EMBO Conference

U.S. FDA Grants Fast-Track Designation to Sanofi Pasteur and KaloBios’ Novel Biologic Candidate for Pseudomonas aeruginosa

Sanofi Pasteur, the vaccines division of Sanofi (EURONEXT: SAN and NYSE: SNY), and KaloBios Pharmaceuticals (KBIO) announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to Sanofi Pasteur for the investigation of KB001A, an Continue reading U.S. FDA Grants Fast-Track Designation to Sanofi Pasteur and KaloBios’ Novel Biologic Candidate for Pseudomonas aeruginosa

Savara Pharma begins phase II study of AeroVanc for MRSA lung infection in CF patients

Savara Pharmaceuticals, an emerging specialty pharmaceutical company, has started study drug treatment in a Phase 2 clinical trial evaluating the safety and efficacy of AeroVanc for the treatment of persistent methicillin-resistant Staphylococcus aureus (MRSA) lung infection in cystic fibrosis Continue reading Savara Pharma begins phase II study of AeroVanc for MRSA lung infection in CF patients

N30 Pharma Announces Presentation of Preclinical Data at the Basic Science Meeting of the European Cystic Fibrosis Society

N30 Pharmaceuticals, Inc., a clinical stage, biopharmaceutical company focused on the development of therapies for cystic fibrosis (CF), announced today the presentation of key preclinical data for its novel inhibitors of S-nitrosoglutathione reductase (GSNORi). The data were Continue reading N30 Pharma Announces Presentation of Preclinical Data at the Basic Science Meeting of the European Cystic Fibrosis Society

U.S. FDA OKs Novartis inhaler to treat lung infection in cystic fibrosis patients

U.S. health regulators approved on Friday a hand-held inhaler made by Swiss drug maker Novartis AG to treat a type of bacterial lung infection that often affects cystic fibrosis patients.
Novartis’s TOBI Podhaler contains a dry powder formulation of tobramycin, an antibiotic used to Continue reading U.S. FDA OKs Novartis inhaler to treat lung infection in cystic fibrosis patients

FDA Sends Cystic Fibrosis Treatment Back for More Study

Mar 19, 2013 – The US Food and Drug Administration (FDA) has completed its review of Pharmaxis’ New Drug Application (NDA) for its dry powder formulation of mannitol (Bronchitol, Pharmaxis) and has recommended that the company conduct an additional study to obtain Continue reading FDA Sends Cystic Fibrosis Treatment Back for More Study