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Taking cell and gene therapies from idea to commercialisation
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)
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Collaboration to enable standardisation of AAV quality measurement
US Pharmacopeia to collaborate on assessment of analytical methods for adeno-associated viruses (AAVs) to facilitate the development of reference standards.
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Zolgensma® meaningfully improves spinal muscular atrophy in patients
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.
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Curative cell and gene therapies and healthcare system disruption
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.
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Evrysdi™ approved for treatment of spinal muscular atrophy in Europe
The European Commission has granted marketing authorisation to Roche’s Evrysdi™ (risdiplam) as a treatment for spinal muscular atrophy in infants, children and adults.
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NICE approves gene therapy for spinal muscular atrophy
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.
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EMA recommends six medications for approval in February meeting
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.
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EMA’s transformative treatments of 2020
In this article, we summarise the trial findings for the European Medicines Agency’s 13 most transformative therapies of 2020.
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SMA drug Zolgensma® has “significant therapeutic benefit”, according to interim data
Treatment with Zolgensma® (onasemnogene abeparvovec) resulted in rapid and sustained improvement in motor function in spinal muscular atrophy (SMA) patients.
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EMA accepts marketing application for Spinal Muscular Atrophy treatment
The European Medicines Agency (EMA) has accepted the marketing authorisation application for Evrysdi™ (risdiplam) as a treatment of spinal muscular atrophy (SMA).
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Study suggests Zolgensma will generate $2.5bn of global sales by 2025
According to researchers, Zolgensma, made by Novartis, will lead the global spinal muscular atrophy market and generate billions in sales.
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Committee for Medicinal Products for Human Use (CHMP) meeting highlights
A summary of the CHMP meeting conclusions, including medicines recommended for approval and indication extension and several safety review findings.
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Gene therapy medication provides improvements in severe SMA
Spinraza, the gene therapy medication, also provides significant improvements in cases with the next most severe form of the spinal muscular atrophy...
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Nusinersen improves motor function of children with SMA
Clinical trial shows treatment is effective for children with spinal muscular atrophy...
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Nusinersen enables infants with SMA to regain motor function
Infants with SMA are more likely to show gains in motor function and survive without assisted support when treated with nusinersen...