Author Archives: Lindsey Shapiro PhD

Zolgensma gene therapy for SMA now routinely accessible in UK

The one-time gene therapy Zolgensma (onasemnogene abeparvovec) will now be routinely accessible at low or no cost to presymptomatic infants in England who are genetically predisposed to spinal muscular atrophy (SMA). Per updated recommendations from the country’s National Institute for Health and Care Excellence (NICE), this…

Apitegromab Clinical Trial Updates Expected This Year

The ongoing SAPPHIRE clinical trial testing apitegromab, Scholar Rock’s investigational muscle-directed therapy, in people with spinal muscular atrophy (SMA) is expected to complete patient enrollment later this year, the company announced. Top-line data from the trial are anticipated in 2024. Should results be positive, Scholar Rock…

CANbridge Acquires Possibly Safer, More Effective SMA Gene Therapy

CANbridge Pharmaceuticals has acquired exclusive global rights to develop, manufacture, and commercialize a second-generation gene therapy for spinal muscular atrophy (SMA). The company had completed preclinical studies of the investigational gene therapy in collaboration with the Horae Gene Therapy Center at the University of Massachusetts (UMass)…