FDA Approves Kalydeco for Cystic Fibrosis Patients Ages 2 and Up

http://www.raredr.com/

The U.S. Food and Drug Administration (FDA) continues to provide new indications for Vertex’s Kalydeco (ivacaftor) for Cystic Fibrosis (CF) patients. Earlier today, it was approved for use in more than 600 patients ages 2 and older who have 1 of 5 residual function mutations that results in a splicing Continue reading FDA Approves Kalydeco for Cystic Fibrosis Patients Ages 2 and Up

Support of Clinical Candidate for Cystic Fibrosis from Sprynx Biosciences

Spyryx Biosciences Presents Data and Corporate Update at Two Recent Conferences in Support of Clinical Candidate for Cystic Fibrosis

– Provides corporate update at Needham Healthcare Conference

– Highlights positive data & mechanism of action of SPX-101 at
ECFS Basic Science Conference Continue reading Support of Clinical Candidate for Cystic Fibrosis from Sprynx Biosciences

AbbVie, Galapagos’ Kalydeco challenger clears phase 2

http://www.fiercebiotech.com/biotech/abbvie-galapagos-kalydeco-challenger-clears-phase-2
Galapagos has posted phase 2 data on the cystic fibrosis drug it is developing with AbbVie. The data suggest the potentiator can play a role in challenging Vertex’s Kalydeco and its follow-up programs, although its place in a planned triple-combination therapy may be taken by a competing Continue reading AbbVie, Galapagos’ Kalydeco challenger clears phase 2

My Five Takeaways from #NACFC2016

I had a dream last night, and in that dream the Limp Bizkit classic song, Nookie was on repeat for some reason. I have no idea why; I haven’t heard that song in about 12 years. Needless to say I woke up pretty jacked up and motivated to get sh*t done before the Packers game this afternoon. Continue reading My Five Takeaways from #NACFC2016

Gene therapy for treating CF lung problems

Gene therapy may be viable approach for treating CF lung problems

http://www.news-medical.net/news/20160920/Gene-therapy-may-be-viable-approach-for-treating-CF-lung-problems.aspx

Two new studies from the University of Iowa suggest that gene therapy may be a viable approach for treating or preventing lung disease caused by cystic fibrosis (CF).

Working with CF pigs, the researchers, based in the UI Pappajohn Biomedical Continue reading Gene therapy for treating CF lung problems

Cystic fibrosis patients may benefit from re-purposed approved drug

http://tinyurl.com/gvvmojk

New research suggests that a drug already approved for the treatment of metabolic acidosis – where the body has too much acid – could be repurposed and used to treat airway disease in patients with cystic fibrosis. The drug showed Continue reading Cystic fibrosis patients may benefit from re-purposed approved drug

CF Researchers Identify Way to Move Key Mutant Protein to Its Proper Spot in Cells

By Malika Ammam, PhD
Originally posted in Cystic Fibrosis News Today, 2/19/16

Collaborative research between Eindhoven University of Technology (Netherlands), McGill University (Canada), and the University of Duisburg-Essen (Germany) has identified a promising approach that could Continue reading CF Researchers Identify Way to Move Key Mutant Protein to Its Proper Spot in Cells

Galapagos starts SAPHIRA Phase 2 study with GLPG1837 in cystic fibrosis patients

Galapagos NV (Euronext & NASDAQ: GLPG) announced today the first dosing in its Phase 2 exploratory program of GLPG1837 in patients with cystic fibrosis (CF).

GLPG1837 is a candidate CFTR potentiator drug in clinical development for the treatment of Class III mutations in cystic fibrosis. The SAPHIRA Phase 2 Continue reading Galapagos starts SAPHIRA Phase 2 study with GLPG1837 in cystic fibrosis patients

Galapagos advances CF clinical development

  • Initiation of Phase 1 study with corrector GLPG2222, earning Galapagos a $10 million milestone payment from AbbVie
  • Expansion of CF portfolio with three novel candidate drugs, with aim to create the most effective combo

Mechelen, Belgium; 19 January 2016: Galapagos NV (Euronext & NASDAQ: GLPG) provides an update on progress made in the cystic fibrosis (CF) programs. Galapagos and AbbVie (NYSE: ABBV) aim to develop a triple CFTR combination therapy to address 90% of patients with CF. In order to bring a more effective therapy to patients, the companies plan to develop multiple candidates and backups for each of the three components (1st generation correctors, next generation correctors, and potentiators) with the goal of identifying a potential triple combination. In addition to our potentiator and corrector that are being tested in the clinic, a total of four additional compounds have the potential to reach clinical development in the CF program this year. Continue reading Galapagos advances CF clinical development

Vertex Outlines 2016 Business Priorities to Support the Discovery and Development of New Transformative Medicines for the Treatment of Cystic Fibrosis and Other Serious Diseases

http://investors.vrtx.com/releasedetail.cfm?ReleaseID=949601

-Approximately 25,000 people with cystic fibrosis worldwide currently eligible for treatment with ORKAMBI® (lumacaftor/ivacaftor) or KALYDECO® (ivacaftor); Vertex Continue reading Vertex Outlines 2016 Business Priorities to Support the Discovery and Development of New Transformative Medicines for the Treatment of Cystic Fibrosis and Other Serious Diseases