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
SparingVision has raised €75 million in second-round funding that will be used to fund clinical trials of gene therapies for ocular diseases retinitis pigmentosa (RP) and dry age-related ma
Sarepta is pressing forward with a bold plan to file with the FDA for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD) in the next few months, with a
Acadia Pharma's trofinetide has hit its efficacy targets in a phase 3 trial involving girls with the devastating neurological disorder Rett syndrome, although safety has dampened enthusiasm