All Gene therapy articles
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Industry newsGene therapy restores hearing in five children
Five children with hereditary deafness have had their hearing restored in both ears through gene therapy in a clinical trial.
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PodcastsRegRapPod − May 2024
Vol.21 #5: In May 2024’s RegRapPod, host Alan Booth talks to Issue Editor Harriet ‘Podcast’ Edwards about her specialist subject ATMPs.
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InterviewDr Peter Marks on reflecting innovation through FDA regulation
Harriet Edwards and Dr Katherine Bowen spoke to Dr Peter Marks, Director of Center for Biologics Evaluation and Research at the Food and Drug Administration (FDA), about his career and the continued evolvement of regulation to support innovative medicines development
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Industry newsAngioedemas caused by rare gene fault curable by CRISPR
A single dose of a CRISPR gene therapy has been shown to almost eliminate a rare genetic, inflammatory and potentially life-threatening condition.
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JournalCell and gene therapy – navigating complexities in CMC development for optimisation of global regulatory strategies
Cell and gene therapy (CGT) products represent a frontier in modern medicine, offering precision treatments. Yet their development is affected by multifaceted challenges including chemistry, manufacturing, and controls (CMC). This article provides a comprehensive exploration of the complexities involved in CMC development and considers its role in establishing quality, safety ...
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JournalTOPRA Annual lecture
Dr Miguel Forte started his lecture by describing the fantastic cell and gene therapies (C>) opportunity for patients and other stakeholders in the pharmaceutical industry; he described a complex business with significant investment and value to patient in oncology and rare disease.
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Industry newsCRISPR tool used to treat sickle-cell and β-thalassemia
Casgevy is the first gene therapy that uses the CRISPR/Cas9 gene-editing tool to be approved by the UK MHRA.
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Industry newsGene therapy gives hope to patients with ‘butterfly skin’
FDA approves first gene therapy to treat dystrophic epidermolysis
bullosa patients – a condition sometimes called ‘butterfly skin’. -
JournalG-E-T-ting there – how gene editing technologies could change more than genes
The field of genome editing technologies (GETs) is rapidly advancing, marking a new and exciting era of curative medicines. These products can alter genetic material in patients, highlighting the crucial need to introduce dedicated legislation to ensure that GETs are developed and maintained in a safe and effective manner.
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JournalHM8: The present and future of developing and commercialising cell and gene therapies in a global world
Gene therapies are highly innovative products which have been approved only for the treatment of rare diseases so far. Changes in manufacturing processes during development as well as difficulties in gathering large clinical data sets explain the common challenges encountered by gene therapy developers to demonstrate the benefit of their ATMP.
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JournalUS FDA CDER and CBER guidance of interest
The US Food and Drug Administration (FDA) has been steadily increasing the number of guidance documents produced on a yearly basis over the past decade. However, even with this increased pace, there are numerous topics that industry is looking to the FDA for clarity, especially newer topics like gene therapy, ...
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FocusGene therapy products: regulatory expectations and challenges for industry
As the technology behind gene therapy products continues to evolve, applicants face the challenge of adapting their programmes to increasingly demanding and detailed regulation. This article provides an overview of current regulatory expectations and the challenges they may present to industry.
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JournalNavigating the regulatory landscape for regenerative medicines in Japan
This is the final article in a series of three that critically analyse the differences and similarities between the regulation of advanced therapy medicinal products (ATMPs), also known as regenerative medicines…[1],[2]
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FocusTime is running out for the rare pediatric disease designation and priority review voucher programs
Since 2012, the rare paediatric disease priority review voucher (RPD PRV) program in the US, provides incentives to promote research in leftover orphan paediatric disease...
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JournalGene therapy: regional assessment comparison
Axicabtagene ciloleucel (tradename YESCARTA) is an autologous chimeric antigen receptor T-cell (CAR-T) therapy that targets CD19 and is approved for the treatment of aggressive, relapsed or refractory forms of B-cell non-Hodgkins lymphoma (NHL). The active substance is composed of a patient’s cells that have undergone ex vivo modification, which causes ...
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JournalGlobal regulatory frameworks for the development of gene therapy products
Gene therapies have the potential to revolutionise the global healthcare system. Some of these products may offer curative benefits to patients with devastating conditions. This continuing professional development (CPD) supplement provides an overview of the regulatory framework for the development of gene therapy products in key markets.