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

Vertex to test new class of CF drugs

Vertex to test new class of cystic fibrosis drugs in patients

https://www.bostonglobe.com/business/2016/10/25/vertex-test-new-class-cystic-fibrosis-drugs-patients/IcwjZhIPc2YXyguss2OGSM/story.html

Vertex Pharmaceuticals Inc. said Tuesday it will begin clinical trials for a new class of “triple combination” drug candidates that could nearly double the Continue reading Vertex to test new class of CF drugs

Potential Treatment for Cystic Fibrosis

Catabasis Pharmaceuticals to Present CAT-5571, a Novel Activator of Autophagy, as a Potential Treatment for Cystic Fibrosis at the 30th Annual North American Cystic Fibrosis Conference

http://www.pharmiweb.com/PressReleases/pressrel.asp?ROW_ID=187116#.WA6VFKOZO8U

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that CAT-5571, a novel activator Continue reading Potential Treatment for Cystic Fibrosis

Vertex to get new investment from CFF

Vertex to get new investment from Cystic Fibrosis Foundation

https://www.bostonglobe.com/business/2016/10/14/vertex-get-new-investment-from-cystic-fibrosis-foundation-under-revised-deal/XklbRNebBa1nOhgt4WOqFI/story.html

Vertex Pharmaceuticals Inc. will receive an upfront payment of $75 million and additional payments of up to $6 million a year for the development of new cystic Continue reading Vertex to get new investment from CFF

Vertex ends trial of cystic fibrosis drug combination

https://www.bostonglobe.com/business/2016/08/15/vertex-ends-trial-cystic-fibrosis-drug-combination/6AkHgBOmtKPF9XRgq18IQM/story.html

Vertex Pharmaceuticals Inc. said Monday it is ending a clinical study testing a two-drug combination therapy on a small group of cystic fibrosis patients after an independent board concluded the experimental treatment wasn’t showing Continue reading Vertex ends trial of cystic fibrosis drug combination

Moderna, Vertex to collaborate on cystic fibrosis

https://www.bostonglobe.com/business/2016/07/06/moderna-vertex-collaborate-cystic-fibrosis/rEmPx5Ll746RmQL0oUNIiO/story.html

Moderna Therapeutics Inc. has emerged as a partner of choice for drug makers eager to supplement their own research.

Vertex Pharmaceuticals Inc. became the latest established biopharma company Continue reading Moderna, Vertex to collaborate on cystic fibrosis

CF Meds – Clinical Trials and Off-Label Use

Guest Blog By Jeanie Hanley, MD

The CF therapeutic pipeline has many drugs in development that will become FDA-approved. Some medications may be available only to those who have a specific CFTR mutation; those who have different mutations may be unable to Continue reading CF Meds – Clinical Trials and Off-Label Use

The Paradox of Precision Medicine

http://www.scientificamerican.com/article/the-paradox-of-precision-medicine/

Precision medicine sounds like an inarguably good thing. It begins with the observation that individuals vary in their genetic makeup and that their diseases and responses to medications differ as a result. It then aims to find the right drug, for the right patient, at the Continue reading The Paradox of Precision Medicine

Concert Pharmaceuticals Announces CTP-656 Solid Dose Phase 1 Results Confirmed Superior Pharmacokinetic Profile to Kalydeco®

 LEXINGTON, Mass.–(BUSINESS WIRE)–Concert Pharmaceuticals, Inc. (NASDAQ: CNCE) today announced new results from Part 1 of its multiple ascending dose Phase 1 clinical trial in which a single-dose tablet formulation of CTP-656 was compared to the commercial tablet formulation of Kalydeco®,

Continue reading Concert Pharmaceuticals Announces CTP-656 Solid Dose Phase 1 Results Confirmed Superior Pharmacokinetic Profile to Kalydeco®

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