ProQR Therapeutics announced that it plans to announce proof-of-concept results from tests of its lead candidate to treat cystic fibrosis (CF), QR-010, at a scientific conference this year, and is continuing to advance the development of this and other transformative RNA medicines for severe orphan diseases like CF and Leber’s…
Pulmatrix‘s inhaled PUR1900 for treating pulmonary fungal infections in patients with cystic fibrosis(CF) has received orphan drug status by the U.S. Food and Drug Administration (FDA). The designation was granted “based on a plausible hypothesis that (PUR1900) may be clinically superior to the same drug that is already approved for the same…
Young adults with cystic fibrosis (CF) are particularly vulnerable to depression. As a consequence, these patients may have low rates of adherence to treatments and a severely impaired quality of life, researchers report. The study, “Associations between adherence, depressive symptoms and health-related quality of life in young adults with…
Prenatal testing of people of reproductive age using an expanded carrier screening may increase the detection of hypothetical future fetuses at risk for up to 94 severe or profound conditions including cystic fibrosis, compared to the current recommendations by professional organizations, according to a recent study. Severe conditions are defined as…
Vertex Pharmaceuticals recently gave an update on its Phase 3 development program, which consists of four studies, on the investigational compound VX-661 in combination with Kalydeco (ivacaftor) as a treatment of patients with cystic fibrosis (CF). The company plans to terminate one study, which is investigating VX-661 plus ivacaftor in people with one copy of…
Burkholderia contaminans, a bacteria belonging to the Burkholderia cepacia complex (Bcc) is indeed an opportunistic pathogen in cystic fibrosis according to a study conducted by a team of international researchers.
Proteostasis Therapeutics announced it is now enrolling cystic fibrosis patients and healthy adults in two Phase 1 clinical trials evaluating its lead drug candidate PTI-428. PTI-428 is an oral amplifier of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Amplifiers are CFTR modulators that selectively increase the amount of an…
Arch Biopartners and Catalent Inhalation, a division of Catalent Pharma Solutions, have entered agreements to start manufacturing AB569 for human trials in patients with chronic and antibiotic resistant bacterial lung infections. Representing a large part of the group affected, patients with cystic fibrosis (CF) stand to benefit most. AB569, invented by Daniel Hassett,…
The Japanese Patent Office has granted AmpliPhi Biosciences a patent for the “Beneficial effects of bacteriophage treatments” targeting Pseudomonas aeruginosa infections. A similar patent has already been granted in the United States and a request for one has been filed in Canada. In Australia and in the European…
New four-dimensional computed tomography (4DCT) for lung scans, developed by Professor Andreas Fouras at Monash University, offers the potential to revolutionize treatment for the millions of patients worldwide with lung diseases like cystic fibrosis. The 4DxV X-ray imaging technology, developed and commercialized by Professor Fouras’ technology company 4Dx, provides a state-of-the-art, noninvasive…
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