Krystal Biotech (NASDAQ:KRYS) said it has generated positive interim clinical results for its inhaled cystic fibrosis (CF) ...

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 ...

Positive interim safety results from the first two dose escalation cohorts, referred to as Cohorts 1 and 2, were reported in 4Q 2024. Today’s update is focused on safety and molecular findings from ...

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 ...

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 ...

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.

The first peer-reviewed journal in the field of human gene therapy, providing all-inclusive coverage of the research, methods, and clinical developments that are driving today's explosion of gene ...

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 ...

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 ...

Cell and gene therapies use the body's building blocks - cells and genetic material - to boost the immune system or correct ...