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European Pharmaceutical Review Issue 2 2024
European Pharmaceutical Review Issue 2 includes articles on cell and gene therapy, from supply chain and manufacturing to microbial testing.
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Gene therapy
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Supporting female leaders to advance cell and gene therapies
At the 2024 Advanced Therapies conference, panellists discussed the lack of women in leadership positions within the cell and gene therapy space and what could be contributing to this.
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AAV gene therapy demonstrates positive activity in Duchenne muscular dystrophy
Initial trial results show the gene therapy enabled up to 85 percent of muscle fibres expressing microdystrophin in boys with Duchenne muscular dystrophy eight weeks post-injection.
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CRISPR technologies fuelling haematological innovations
Greater use of CRISPR-based therapies in clinical trials is expected to drive further advancements in precision medicine, GlobalData states.
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Championing UK ATMP clinical trials
In this Q&A, Jacqueline Barry, Chief Clinical Officer for Cell and Gene Therapy Catapult, elucidates on how the Advanced Therapy Treatment Centre (ATTC) Network can strengthen UK ATMP clinical trials.
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One-time gene therapy could improve wet AMD
A Phase I/IIa trial suggests that a subretinal gene therapy for wet age-related macular degeneration (wet AMD) could reduce the need for patients to receive anti-VEGF injections.
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Moving towards continuous batch RNA production
Here Thibault Jonckheere, CEO, Exothera discusses how continuous RNA production can set a new benchmark in the pharmaceutical industry.
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Comprehensive evaluation of Growth Direct® Rapid Sterility for microorganism detection
22 March 2024 | By Rapid Micro Biosystems
Discover the extensive panel testing done to evaluate the new, innovative sterility testing platform, Growth Direct® Rapid Sterility.
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FDA approves innovative gene therapy for MLD
Following US FDA approval of Lenmeldy™ (atidarsagene autotemcel) for early-onset metachromatic leukodystrophy (MLD), the US wholesale acquisition cost of the gene therapy has been set to $4.25 million.
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ebook: A practical guide to single-use filtration in biopharma
This ebook explains how single-use filtration can support safer, more efficient biopharmaceutical production. Discover more on different filtration stages and unveil key characterisations, like pore size and membrane material, empowering tailored selection to specific biomolecules and processes.
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Pharma Horizons: Environmental Monitoring
This report is dedicated to exploring some of the latest advances in pharmaceutical environmental monitoring, covering topics from contamination control and cleanroom validation through to emerging technologies for bacterial endotoxin testing.
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The trend towards outsourced buffer manufacturing in biopharmaceuticals
12 March 2024 | By Actylis
Join us as we look at how the outsourcing of buffers is helping biopharma drug manufacturers to streamline their processes.
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New data revealed for Novartis SMA gene therapy
Novartis has revealed new data for its one-time gene therapy for spinal muscular atrophy (SMA) in older children.
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2024 Pharmaceutical industry trends – discover the latest pharma quality and manufacturing trends
Some of the most sought-after human experiences are health and happiness. Although pharmaceutical companies cannot determine personal happiness, they are working to improve personal health outcomes that can tip the scale. The demand for personalised medicines, cell and gene therapies, hyperresponsive and patient-centric healthcare, real-world data fuelled by artificial intelligence…
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Process analytical technology: applications and opportunities for pharma
15 February 2024 | By European Pharmaceutical Review, sponsored by Mettler Toledo, Bruker and 908Devices
During this virtual panel, industry experts will discuss the latest advances and applications of process analytical technology in the pharmaceutical sector.
