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 infectious diseases and rare liver and respiratory conditions. The European Commission (EC), influenced by a favorable review from the European Medicines Agency (EMA), has recently granted orphan medicinal product designation […]
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 American Cystic Fibrosis Conference. The company’s stabilizers, SION-719 and SION-451, have demonstrated the potential to fully restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in preclinical […]
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 was carried out through an all-stock deal in a ratio under which original Idera Pharmaceuticals’ shareholders will own a stake of around 33% in the combined company, while Aceragen’s shareholders […]
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 million through a Series C financing round. The Series C financing round was led by Viking Global Investors. It also saw participation from new investors such as Amzak Health, Casdin […]
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. The US biopharma company said that clinicians can apply for patient access using the exceptional circumstances framework under the Pharmaceutical Management Agency (PHARMAC). Ludovic Fenaux – Senior Vice President of […]
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 acquisition news. Financial details of the two deals have not been disclosed. The divestments are being taken up in an effort to wrap up the $63 billion AbbVie acquisition of […]
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 fibrosis. PAAG15A, also known as SNSP113, is under development by Synspira Therapeutics as an innovative inhaled therapy designed to enhance lung function in patients with cystic fibrosis. This drug targets […]
