news
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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Gene therapy
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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…
webinar
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.
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Vertex secures European approval for CRISPR cell therapy
Now conditionally approved in Europe for sickle cell disease and transfusion-dependent beta thalassemia, the CRISPR therapy offers eligible patients a functional cure.
webinar
Applying ISO/TR 14644-21 – Airborne particle sampling techniques
23 January 2024 | By Particle Measuring Systems
Watch this webinar as GMP expert Mark Hallworth discusses the difference between cleanroom classification and monitoring of the new ISO 14644 Technical Report 21 focuses in section 4.0 and 5.2.
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UK ATMP sector “robust”, research finds
The cell and gene therapy (CGT) sector in the UK is “robust”, with a stable clinical trials landscape, according to research from Cell and Gene Therapy Catapult (CGT Catapult) and the UK BioIndustry Association (BIA).
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Developing micro QC for ATMPs
In this Q&A, Natalie Saunders, Interim Head of Quality Control at CGT Catapult, delves into the advancements and challenges in microbiological QC for cell and gene therapies. She discusses rapid sterility testing methods, regulatory frameworks, and the impact of automation and digitalisation on quality control.
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Streamlining bioprocessing for gene therapy
Adopting technological advances in upstream and downstream processes is vital to the gene therapy space, says Kai Lipinski, CSO at ReciBioPharm. Here he explores emerging technology trends and discusses how they can help to overcome key challenges facing gene therapy manufacturers.
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MeiraGTx sells XLRP gene therapy to Janssen
Janssen will pay up to $415 million for MeiraGTx’s botaretigene sparoparvovec, a gene therapy for treatment of X-linked retinitis pigmentosa (XLRP).
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Bioburden and sterility testing: how to conserve gene therapy product
Here, Kathy Zagaroli, Senior Director of Quality Control at Kiniksa Pharmaceuticals, and Tom Bujold, Senior Director of Quality Control at REGENXBIO, discuss the challenges of bioburden and sterility testing for gene therapies and why industry must come together to drive increased harmonisation.
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Year in review: EPR’s top stories of 2023
EPR wraps up the year with a selection of top stories from 2023, highlighting key trends and topics such as environmental monitoring (EM), quality (QA/QC), manufacturing, regulation and clinical development.
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FDA approves first cell-based gene therapies for sickle cell disease
The US FDA has approved Vertex’s Casgevy and bluebird bio’s Lyfgenia, for one-time treatment of sickle cell disease in patients 12 years and older.
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‘Innovation-first’ mindset vital for advanced therapies sector future
Improving data harmonisation and investing in the advanced therapy sector’s workforce are some of the key recommendations the sector must take to advance the field and increase patient access, a new report highlights.
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Cell and gene therapy: manufacturing and talent trends 2023
In this interview, Stephen Ward, Chief Manufacturing Officer and Jeanette Evans, Chief Business Officer, Cell and Gene Therapy Catapult, highlight key data from the organisation’s recent GMP Manufacturing Survey and Skills Demand reports, and present insight into the sector’s current and future landscape.
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Ferring reveals long-term data for cancer gene therapy
New data from a Phase III gene therapy trial has demonstrated a 90 percent three-year overall survival rate for its participants with a high-risk bladder cancer.
