Please provide your email address to receive an email when new articles are posted on . Patients given aerosolized 4D-710 had elevated CFTR protein levels that went over normal levels. Over 12 months, ...

SPL84 is administered directly into the lungs where it is taken up by the cells and is expected to drive the production of fully functional CFTR proteins. The Food and Drug Administration (FDA) has ...

Please provide your email address to receive an email when new articles are posted on . Triple therapy improved lung function, BMI and other factors. Patients naïve to cystic fibrosis transmembrane ...

BETHESDA, Md.--(BUSINESS WIRE)--The Cystic Fibrosis Foundation today announced its investment of $5 million in Sionna Therapeutics, a company founded in 2019. The company is focused exclusively on ...

Thanks to simultaneous achievements in gene therapy and medicine delivery platforms, new solutions to intractable problems are bringing us to a real watershed moment in the treatment of respiratory ...

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 ...

A study published in Nature Communications revealed a new antisense oligonucleotide (ASO) therapy applicable to the W1282X mutation of the cystic fibrosis transmembrane conductance regulator gene ...

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 ...

Detailed price information for Krystal Biotech Inc (KRYS-Q) from The Globe and Mail including charting and trades.

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 ...