Sionna Therapeutics targets cystic fibrosis with novel CFTR/NBD1 stabilization; key 2026 readouts and cash runway to 2028.

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.

A tiny antibody component could fundamentally transform the treatment of cystic fibrosis: For the first time, researchers have succeeded in developing a so-called nanobody that penetrates directly ...

Patients with cystic fibrosis often reduce or discontinue respiratory cotherapies after starting CFTR modulators without affecting clinical status.

Chemical modulation of histone deacetylase (HDAC) activity by HDAC inhibitors (HDACi) is an increasingly important approach for modifying the etiology of human disease. Loss-of-function diseases arise ...

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 ...

Around 3 million people worldwide struggle with chronic pancreatitis, a condition in which the pancreas becomes inflamed, ...

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 ...

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 ...

Apical localisation of CFTR protein and its chloride channel activity strictly depend on interactions of its PDZ (for postsynaptic density 95/disclarge/zona occludens)-interacting domain in the C ...

The cystic fibrosis transmembrane conductance regulator has been studied for years but the new efforts have yielded important insights. Scientists at St. Jude Children's Research Hospital and ...