news
Gene therapy for rare muscular dystrophy presents preliminary promise
Initial clinical results for a gene therapy being evaluated in limb-girdle muscular dystrophy has shown evidence of transgene expression in the three-month muscle biopsy.
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Gene therapy
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Regulatory-first for gene editing therapy
The world’s first investigational in vivo CRISPR-based gene editing therapy cleared for late-stage clinical development is expected to enter Phase III in late 2023.
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Key developments in pharmaceutical manufacturing – September 2023
This article outlines the recent progress of several major new manufacturing facilities that will be central in supporting the production of innovative therapies in Europe.
webinar
How to implement single temperature incubation in your environmental monitoring routine
11 October 2023 | By bioMérieux
Watch this webinar to hear Laurent Leblanc explore the challenges and benefits of implementing the “one media / one temperature” approach for routine environmental monitoring.
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Exploring the gene therapy biomanufacturing landscape
In this exclusive Q&A with European Pharmaceutical Review, Sheila Ann Mikhail, co-founder of AskBio and keynote speaker at CPHI Barcelona delves into the current landscape and future potential of the gene therapy sector. She explores strategies for making gene therapies more affordable and accessible, innovations in drug delivery, as well…
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CPHI Barcelona 2023 – Event preview
CPHI Barcelona takes place in October 2023, covering a wide range of topics from next generation biomanufacturing and formulation to sustainability.
whitepaper
ebook: Accelerate the development of your biologics
Altasciences’ Proactive Drug Development can accelerate the development of biologics by up to 40%, from lead identification to clinical POC.
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Gene therapy: a radical pharmaceutical revolution
Karen Pinachyan, Head of Medical Affairs Europe at CSL Behring provides an overview of past and present gene therapy development, plus the progress of clinical trials and key challenges in the sector.
news
Advanced therapy collaboration network launched in Scotland
Advanced therapy (ATMP) manufacturers and developers in Scotland are set to be supported through a new collaboration initiative from the Cell and Gene Therapy Catapult (CGT Catapult).
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UK ATMP facility granted cGMP approval
A UK CDMO is now permitted through MHRA certification to produce clinical supply of gene therapy AAV, including bioprocess through to cGMP manufacturing.
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Pioneering PAT to improve gene therapy AAV manufacture
The CGT Catapult and two other collaborators have agreed to work together and use advanced process analytical technologies (PAT) to improve the process and efficiency of gene therapy AAV manufacturing.
whitepaper
Technical document: Introducing: Ami Polymer’s Sterile Sampling Systems
Next-Gen Sterile Sampling System: Pinnacle Compliance - USP Standards 87, 88, 85, 661.1, 788, 71, and ISO 11737-1 for Unparalleled Pharma & Biopharma
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Stem cell therapy delivers potential for sickle cell disease
A Novartis’ Phase I/II study for sickle cell disease trialling the gene therapy OTQ92 is the first treatment to target a new genetic area and use cryopreserved stem cells, according to new paper published in NEMJ.
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CGT manufacturing QC market to value $2.8b by 2031
Advanced analytical technologies in quality control (QC) processes in cell and gene therapy manufacturing is anticipated to help deliver a 24.6 percent market CAGR between 2023 and 2031.
whitepaper
Report: Accelerate the RMM validation process
To address the perceived barrier to entry for rapid microbiological methods, new validation packages are now available for Celsis® rapid microbial detection.
