Arcturus Therapeutics’ ARCT-032 gets EU’s orphan medicinal product designation for cystic fibrosis

Pallavi Madhiraju- February 22, 2024 0

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

Pallavi Madhiraju- November 4, 2023 0

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

Pallavi Madhiraju- October 2, 2022 0

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

pharmanewsdaily- June 17, 2020 0

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

pharmanewsdaily- February 26, 2020 0

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

pharmanewsdaily- January 27, 2020 0

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 PAAG15A receives orphan drug designation for cystic fibrosis treatment

pharmanewsdaily- October 14, 2018 0

Synspira Therapeutics’ investigational inhaled treatment, PAAG15A, has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of cystic ... Read More