eturbonews

New Mechanism Could Fully Restore CFTR Function in Cystic Fibrosis

Sionna Therapeutics today announced the official launch of the company and the closing of a $111 million Series B financing. The round was led by OrbiMed with participation from funds advised by T.

Nanoparticle-based gene editing could expand treatment options for cystic fibrosis

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene into human airway cells, restoring key biological function in a laboratory ...
News Medical on MSN

New gene editing approach offers hope for cystic fibrosis patients

UCLA researchers have developed a lipid nanoparticle-based gene-editing approach capable of inserting an entire healthy gene ...
Healio

‘CFTR dysfunction may be a lot more common’ in healthy young adults

Please provide your email address to receive an email when new articles are posted on . Sweat chloride values ranging from 30 mEq/L to 59 mEq/L were observed in “a surprisingly high proportion of ...
Healio

Novel biomarker shows promise in assessing CFTR function in patients with cystic fibrosis

Please provide your email address to receive an email when new articles are posted on . Challenged urine bicarbonate excretion provided a simple and safe quantification of cystic fibrosis ...

Triple therapy shown to help with cystic fibrosis in the long term

Cystic fibrosis (CF) is the most common inherited metabolic disease in Europe. More than 8,000 people in Germany are affected ...
EurekAlert!

Integrated structural biology provides new clues for cystic fibrosis treatment

(Memphis, Tenn.—March 22, 2023) Scientists at St. Jude Children’s Research Hospital and Rockefeller University have combined their expertise to gain a better understanding of the cystic fibrosis ...
News Medical

Understanding the structure of CFTR could help design more effective therapies for cystic fibrosis

Scientists at St. Jude Children's Research Hospital and Rockefeller University have combined their expertise to gain a better understanding of the cystic fibrosis transmembrane conductance regulator ...
Kaleido Scope

A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations

The drug is a small molecule with a novel mechanism of action, say David Bedwell, Ph.D., and Steven Rowe, M.D., MSPH, co-senior authors. Bedwell is professor and chair of the University of Alabama at ...
Nasdaq

Sionna Therapeutics Presents Preclinical Data on Novel CFTR Modulators at the 48th European Cystic Fibrosis Conference

Sionna Therapeutics, Inc. has announced the presentation of promising preclinical data at the 48th European Cystic Fibrosis Conference in Milan, showing that its nucleotide-binding domain 1 (NBD1) ...
Business Wire

Vertex to Present Long-Term Data Demonstrating Significant Benefits of Treatment With CFTR Modulators at North American Cystic Fibrosis Conference (NACFC)

BOSTON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that five scientific abstracts about the company’s portfolio of cystic fibrosis (CF) medicines will be ...