cystic fibrosis
ReCode Therapeutics Doses First Cystic Fibrosis Patient in Phase Ib Trial of mRNA Drug
RCT2100 is an experimental treatment that patients inhale and is designed to deliver CFTR mRNA directly to lung cells.
FDA to Review Vertex Pharma's NDA for 'Vanza Triple' Cystic Fibrosis Regimen
Vertex's regulatory application for the triplet combination has priority review from the agency and has been validated by the European Medicines Agency.
EC Approves Vertex's Kalydeco for Cystic Fibrosis Patients as Young as 1 Month
Kalydeco, already approved for patients as young as 1 month old in the US and UK, is now available to babies that young throughout the European Union.
SpliSense to Start Phase II Trial of Cystic Fibrosis Antisense Oligonucleotide Drug in US
The firm is developing SPL84 as a treatment for patients with cystic fibrosis caused by a rare splicing mutation.
EMA's CHMP Issues Positive Opinions on Biogen's Qalsody, Vertex's Kalydeco
The committee recommended marketing authorization under exceptional circumstances for Qalsody as well as expanding Kalydeco's indication to younger patients.