Scientists from Spirovant Sciences describe SP-101, a novel adeno-associated virus (AAV) gene therapy for the treatment of cystic fibrosis (CF) in the peer-reviewed journal Human Gene Therapy. They ...

BETHESDA, Md.--(BUSINESS WIRE)--The Cystic Fibrosis Foundation today announced its investment of $6 million in Carbon Biosciences to support the company‘s preclinical research into an innovative gene ...

LONDON, July 3 (Reuters) - Scientists conducting a major trial of a therapy that replaces the faulty gene responsible for cystic fibrosis say the treatment has showed significant benefit for the first ...

Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most common fatal ...

BETHESDA, Md.--(BUSINESS WIRE)--Today, the Cystic Fibrosis Foundation announced an additional investment of up to $24 million in Prime Medicine to continue the development of a gene editing therapy ...

An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their mutation type, is being tested in human trials in the UK and Europe. Cystic ...

Hundreds of people with the most common type of cystic fibrosis could be offered a new triple therapy after the National Institute for Health and Care Excellence (NICE) recommended ...

CINCINNATI, July 30, 2024 /PRNewswire/ -- Genexys BioMed, a pre-clinical stage biotechnology company developing a novel non-viral, non-lipid genetic medicine platform for the treatment of cystic ...

An inhalable medicine with the potential to improve lung disease in people with cystic fibrosis, irrespective of their mutation type, is being tested in human trials in the UK and Europe. Cystic ...

Monthly administration of liposome-delivered gene therapy for cystic fibrosis (CF) improved forced expiratory volume (FEV) in a randomized, double-blinded, placebo-controlled phase 2b clinical trial.

4D Molecular Therapeutics (FDMT) slipped ~15% on Wednesday after the company announced updated data from an early-stage trial for 4D-710, its experimental therapy for a genetic disorder known as ...

NATIONWIDE — Emily Kramer-Golinkoff can't get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most ...