David Vincent, Process Development Lead at eXmoor Pharma, details some of the critical steps that form the manufacturing strategy for providers of cell and gene therapies.
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Spinal muscular atrophy (SMA)
US Pharmacopeia to collaborate on assessment of analytical methods for adeno-associated viruses (AAVs) to facilitate the development of reference standards.
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.
In this article, experts from Charles River Associates discuss how the introduction of cell and gene therapies will impact upon various aspects of the healthcare system, from provision of care to delivery and supply, and pricing and market access.
The European Commission has granted marketing authorisation to Roche’s Evrysdi™ (risdiplam) as a treatment for spinal muscular atrophy in infants, children and adults.
Zolgensma, a one-off, potentially curative treatment for type 1 spinal muscular atrophy, is set to become the most expensive drug ever approved for use on the UK’s NHS.
The EMA’s committee adopted positive opinions of six medications, including two orphan drugs, and concluded its review of the use of REGN-COV2 – an investigational antibody therapy for COVID-19.
Treatment with Zolgensma® (onasemnogene abeparvovec) resulted in rapid and sustained improvement in motor function in spinal muscular atrophy (SMA) patients.
The European Medicines Agency (EMA) has accepted the marketing authorisation application for Evrysdi™ (risdiplam) as a treatment of spinal muscular atrophy (SMA).
According to researchers, Zolgensma, made by Novartis, will lead the global spinal muscular atrophy market and generate billions in sales.
A summary of the CHMP meeting conclusions, including medicines recommended for approval and indication extension and several safety review findings.
Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of the spinal muscular atrophy...
Clinical trial shows treatment is effective for children with spinal muscular atrophy...
Infants with SMA are more likely to show gains in motor function and survive without assisted support when treated with nusinersen...