University of California10d
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 ...
pharmaphorum5d
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.
pharmaphorum3d
New Vertex drugs could help 90% of cystic fibrosis patients
Boehringer's cystic fibrosis gene therapy is poised to start clinical testing, hoping to offer a one-shot alternative to costly CFTR modulator drugs.
sciencex12d
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 ...
Rolling Out5d
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 ...
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 one-a-day pill could make it vanish.
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 ...
McGill University Health Centre16d
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 ...
Interesting Engineering on MSN6d
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 ...
The Motley Fool on MSN19d
Where Will Vertex Pharmaceuticals Be in 10 Years?
The biotech won approval for its first cystic fibrosis (CF) drug -- Kalydeco -- back in 2012 ... collecting approvals for a gene-editing therapy for blood disorders a little over a year ago and a pain ...
PMLiVE20d
Vertex’s triple combination cystic fibrosis therapy Alyftrek approved by MHRA
Vertex Pharmaceuticals’ triple combination cystic fibrosis (CF) treatment has been approved ... by correcting the malfunctioning protein made by the CFTR gene. The MHRA’s approval of the therapy was ...