University of California7d
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
pharmaphorum2d
Trial of Boehringer's cystic fibrosis gene therapy imminent
An inhaled gene therapy for cystic fibrosis developed by Boehringer Ingelheim is due to start clinical testing shortly, hoping to become a first-in-class treatment for the genetic disorder.
Rolling Out2d
Cystic fibrosis care made easier for parents
Learn how to navigate cystic fibrosis care with confidence, from treatments and school plans to building a supportive medical ...
ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT2100 for the Treatment of Cystic Fibrosis
ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA ...
sciencex9d
Cystic fibrosis gene therapy programme gets green light with public funding
A groundbreaking gene therapy trial for cystic fibrosis (CF) will begin in March, thanks to a new grant from a government funding body. One hundred and thirty adults and children with CF will take ...
Science Daily12d
Newly uncovered mechanism could drive next-gen cystic fibrosis treatments
The Hospital for Sick Children. "Newly uncovered mechanism could drive next-gen cystic fibrosis treatments." ScienceDaily. ScienceDaily, 13 March 2025. <www.sciencedaily.com / releases / 2025 / 03 / ...
Cystic Fibrosis Clinical Trials and Studies: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies
In March 2025, Vertex Pharmaceuticals Incorporated announced a study is to evaluate the safety, and tolerability and efficacy ...
NHS hope as new miracle drug greatly reduces some cystic fibrosis symptoms
The genetic condition cystic fibrosis had no treatments on the NHS before 2019 and a Daily Express campaign - now a miracle ...
Business Wire1mon
FDA Grants Orphan Drug Designation to Revolutionary Cystic Fibrosis Therapy
Cystic fibrosis is a genetic disorder that adversely affects ... Inc. announced the Orphan Drug Designation of its cystic fibrosis therapy by the Food and Drug Administration (FDA).
Interesting Engineering on MSN3d
Scientists train dog detectives to sniff deadly bacteria from skin, urine, clothes
In collaboration with Medical Detection Dogs, a team at Imperial College London has discovered dogs can detect bacteria like ...
McGill University Health Centre13d
Major funding for research project on cystic fibrosis
A new research project focusing on rare genetic mutations that are linked to cystic fibrosis just received $600,000 in ...
Morningstar3d
ReCode Therapeutics Receives U.S. FDA Orphan Drug Designation for RCT2100 for the Treatment of Cystic Fibrosis
investigational mRNA therapy being developed to treat cystic fibrosis (CF). CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Although recent ...