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.
Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biote
Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRe
The FDA has approved Akebia Therapeutics’ vadadustat as a treatment for anaemia caused by chronic kidney disease (CKD) in dialysis patients, becoming the first rival to GS