Vertex buys Concert’s cystic fibrosis drug
Vertex Pharmaceuticals will pay up to $250 million for Concert Pharmaceuticals’ cystic fibrosis drug CTP-656. Currently in Phase II clinical trials, the drug is a deuterated version of Vertex’s own Continue reading News about Concert Pharmaceuticals’ cystic fibrosis drug
Galapagos doses first patient with novel CF corrector GLPG2222
Galapagos NV (Euronext & NASDAQ: GLPG) announces dosing of the first patient with cystic fibrosis (CF) Class III (F508del and a gating mutation like G551D) with novel CF corrector GLPG2222 as an add-on to Kalydeco® in a Phase 2a study. Galapagos further announced the opening of an Investigational New Drug (IND) file with the US Food & Drug Administration for GLPG2222, triggering a $10 million milestone payment.
Continue reading Phase 2a CF corrector study
Nanoparticle-delivered CRISPR tools could treat hemophilia, cystic fibrosis and muscular dystrophy
More and more scientists are using the powerful new gene-editing tool known as CRISPR/Cas9, a technology isolated from bacteria, that holds promise for new treatment of such genetic diseases as cystic Continue reading Nanoparticle-delivered CRISPR tools could treat cystic fibrosis
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
Galapagos initiates Phase 1 study with cystic fibrosis drug GLPG2737
Galapagos NV (Euronext & NASDAQ: GLPG) reports the initiation of a Phase 1 study in healthy volunteers with GLPG2737, a novel C2 corrector drug for cystic fibrosis. Initiation of the Phase 1 study triggers a $10 million milestone payment from AbbVie.
The aim of the Phase 1 study is to evaluate the safety, tolerability and Continue reading Galapagos initiates Phase 1 study
Vertex to test new class of cystic fibrosis drugs in patients
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
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
This GoPro video definitely shows a behind the scenes look at my life with cystic fibrosis. I tried to capture the maintenance that my body requires for it to fully function. Beyond that I really wanted to give a first person point of view of my Continue reading OWN IT: Living with CF GoPro Part II
Cystic fibrosis lung cells were restored to 50 percent of healthy function in work that provides the first evidence that novel therapeutic strategies for human patients can be identified based on yeast studies.
In experiments with isolated cystic fibrosis lung cells, University of Alabama at Continue reading Therapeutically robust correction, in vitro, of the most common cystic fibrosis mutation