4DMT Receives Up to $14M from CFF for Development of Customized Gene Carrier
After being given more than $3 million from the Cystic Fibrosis Foundation (CFF) in 2017 to expand the development of viral vectors for...
Are intestinal organoids the key to developing personalized therapy for cystic fibrosis?
Intestinal organoids have been utilized to develop personalized therapy for cystic fibrosis patients. Cystic fibrosis (CF) is a rare...
The Future of Cell and Gene Therapy
Each passing year, the success of cell and gene therapy (CGT) becomes clearer, more widely covered in the media, and is increasingly the...
Developing a first-in-class regenerative gene therapy for cystic fibrosis
OmniSpirant Limited is an Irish company developing a regenerative gene therapy with the potential to transform respiratory medicine. The...
Symdeko Approved in Australia for CF Patients Age 12 or Older With Certain Gene Mutations
Cystic fibrosis (CF) patients in Australia age 12 and older, who have two copies of the F508del mutation in the CFTR gene (a copy...
Gene Therapy
Cystic fibrosis (CF) is a rare, heritable disease caused by mutations in the CFTR gene, which provides instructions for making the CFTR...
Personalized Nutrition Programs May Help Improve Outcomes for CF Patients, Survey Suggests
New personalized strategies that can address the nutritional needs of cystic fibrosis patients may provide the best care and improve...
Gene Expression Patterns in Blood May Lead to Patient-tailored CF Treatment, Study Finds
Researchers tested blood samples from cystic fibrosis (CF) patients and identified distinct gene expression patterns that could lead to...
Specific Sweat Test Better Detects Kalydeco’s Effects on CFTR Function, Study Suggests
The effects of CFTR modulator therapies such as Kalydeco (ivacaftor) can be more reliably assessed using a test that specifically...
New Gene Therapy Could Treat Cystic Fibrosis With One Dose
A new partnership in the UK will develop a gene therapy for cystic fibrosis that could treat the disease with a single dose. Cystic...
