pharmaphorum9d
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, ...
Vertex Pharma wins EU backing to expand label for Kaftrio cystic fibrosis drug
Vertex Pharmaceuticals (NASDAQ:VRTX) announced Friday that an expert panel of the EU drug regulator, the European Medicines ...
pulmonologyadvisor2d
Cystic Fibrosis Lung Transplant Data Suggest Racial Disparities in HEMT Benefits
Racial disparities in lung transplantation rates in CF suggest that HEMT has benefitted White patients more than Black and Hispanic patients.
Medscape21d
Cystic Fibrosis Transmembrane Conductance Regulator–Modifying Medications
[1] These mutations are called gating mutations. The CFTR protein reaches the cell membrane but the channel does not open properly and chloride transport cannot occur (Figure 1d). Class IV ...
Business Insider10d
Boehringer Ingelheim and partners start clinical development of a first-in-class, inhaled gene therapy for people with cystic fibrosis
2 CF is caused by mutations in the CFTR gene. These mutations cause the CFTR protein to become dysfunctional, which leads to the development of sticky, thick mucus clogging the airways, which causes ...
Inhalable Gene Therapy Trial Begins for Cystic Fibrosis Patients
An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis is being tested in human ...
precisionmedicineonline2d
CHMP Recommends Vertex's Kaftrio, Kalydeco Combo for Additional Cystic Fibrosis Patients
The committee supports approving the CFTR modulator for patients 2 years and older with at least one non-class I CFTR gene mutation.
Oxford Biomedica to support Boehringer Ingelheim’s LENTICLAIR 1 trial
OXB announces that its proprietary lentiviral vector manufacturing technology will be used in Boehringer Ingelheim’s newly initiated LENTICLAIR ...
Medindia7d
Inhalable Gene Therapy Trialed for Cystic Fibrosis Patients
Researchers trial inhalable gene therapy for cystic fibrosis, offering a potential breakthrough in treating the genetic lung ...
Yahoo Finance10d
Boehringer Ingelheim and partners start clinical development of a first-in-class, inhaled gene therapy for people with cystic fibrosis
The aim is to improve lung function and reduce exacerbations for people with CF irrespective of their mutations, including those who genetically cannot benefit from CFTR modulators. “We are very ...