Five months after Sanofi backed away from a proposed deal with Maze Therapeutics on a Pompe disease therapy after an antitrust challenge, Shionogi has snapped up the drug.
Prime Medicine has been given the go-ahead by the FDA for the first human trial of its prime editing technology, which promises to deliver one-shot therapies for a range o
Patients in the US with the ultra-rare disease WHIM syndrome now have their first approved treatment, after X4 Pharma’s Xolremdi was given a green light by the FDA.
For many years, orphan drugs for rare diseases have been a draw for pharma companies, offering high prices, swift regulatory reviews, and often extended marketing exclusiv
Ireland’s SynOx Therapeutics has completed a second-round financing, raising $75 million, as it prepares to take its drug for tenosynovial giant cell tumour (TGCT) into ph
People with the rare disease Cushing’s syndrome can sometimes take years to get a diagnosis, but a new project hopes to shorten that timeline with the help of artificial i