Tag: Cystic fibrosis
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 milestone in the development of treatments for ... Read More
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 2 NBD1 stabilizers at the 2023 North ... Read More
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, orphan pulmonary, and rheumatic diseases. The acquisition ... Read More
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 discovery and product development, has raised $70 ... Read More
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 available to eligible patients from next month. ... Read More
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 separate agreements, as per the latest pharma ... Read More
Synspira’s cystic fibrosis treatment PAAG15A gets FDA orphan designation
Synspira has been given the orphan designation status by the US Food and Drug Administration (FDA) for poly (acetyl, arginyl) glucosamine (PAAG15A) for cystic fibrosis ... Read More