Browsing Tag
Cystic fibrosis
9 posts
BillionToOne’s Unity Fetal Risk screen reshapes cystic fibrosis prenatal testing with early therapeutic pathways
BillionToOne’s Unity Fetal Risk Screen delivers 100% sensitivity in cystic fibrosis prenatal detection, unlocking earlier treatment options for families.
Vertex Pharmaceuticals bags UK approval for ALYFTREK, a next-generation cystic fibrosis treatment
The United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted approval for ALYFTREK (deutivacaftor/tezacaftor/vanzacaftor), a next-generation…
Arcturus Therapeutics’ ARCT-032 gets EU’s orphan medicinal product designation for cystic fibrosis
Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT), a leading global clinical-stage messenger RNA medicines company, has achieved a significant…
Sionna Therapeutics showcases novel CFTR stabilizers at NACFC 2023
At the forefront of cystic fibrosis research, Sionna Therapeutics recently unveiled critical preclinical findings on their innovative Series…
Idera Pharmaceuticals acquires rare orphan disease company Aceragen
Idera Pharmaceuticals, a US-based biopharma company, has acquired Aceragen, a biotechnology company focused on developing drugs for rare,…
Gene therapy company 4D Molecular Therapeutics raises $70m in Series C round
California-based gene therapy company 4D Molecular Therapeutics (4DMT), which is focused on adeno-associated virus (AAV) gene therapy vector…
Cystic fibrosis drug KALYDECO to be launched in New Zealand by Vertex Pharmaceuticals
Vertex Pharmaceuticals is set to launch its cystic fibrosis drug KALYDECO (ivacaftor) in New Zealand, which will be…
Allergan to sell brazikumab to AstraZeneca and Zenpep to Nestle
Allergan has agreed to sell IL-23 inhibitor brazikumab to AstraZeneca and gastrointestinal medication Zenpep (pancrelipase) to Nestle in…
Synspira’s PAAG15A receives orphan drug designation for cystic fibrosis treatment
Synspira Therapeutics’ investigational inhaled treatment, PAAG15A, has been granted orphan drug designation by the U.S. Food and Drug…