Ionis has committed to a phase 3 programme for its antisense therapy for rare genetic disorder Angelman syndrome, just weeks after Biogen decided against exercising an option to license the
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
Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be u
Buoyed by encouraging data for its lead drug for autosomal dominant polycystic kidney disease, Regulus Therapeutics has moved swiftly to file a financing that will give it
Gene-editing biotech Prime Medicine has priced a follow-on public offering, hoping to raise $140 million to advance its pipeline of one-shot therapies for severe genetic d
Novartis' programme providing free access to its spinal muscular atrophy (SMA) gene therapy Zolgensma is being scaled back to a dozen countries worldwide, according to the company.
It has been five years since Lexicon Pharma's type 1 diabetes therapy, Zynquista, was rejected by the FDA, and it may struggle to have a better outcome at its second attempt.