MANUFACTURING ARTICLES
Peter Marks: An Early Preview of mRNA, Gene Therapy Platforms
Though Marks emphasized that this concept/designation at the regulatory level is still somewhat of a “we’ll-know-it-when-we-see-it” phenomenon, his remarks this summer — as well as a few additional discussions that have occurred throughout 2023 — add a bit more clarity to our understanding of what a gene therapy platform can be.
MANUFACTURING VIDEOS
Hosted by Cell & Gene Collaborative Director Anna Rose Welch, ARW on RNA puts a creative spin on the emerging mRNA + RNA therapeutics industry. Here, in Episode 5, Welch dives into the separate — but strikingly similar — worlds of fly fishing and mRNA R&D. In particular, she explains how a fly fisherman’s strategies to catch a fish relate to the work of mRNA scientists as they strive to identify the most appropriate immunogenicity profiles for their mRNA vaccines or therapeutics.
As CRISPR use continues to grow, so has the demand for clinical-grade guide RNA. Learn about GMP sgRNA and their stringent process and manufacturing controls.
Is your mRNA production process ready for scale up? Learn how to choose a capping method to optimize your mRNA yield and process costs.
Akhilesh Bhambhani, Sr. Director, Biologics Drug Product Development at Ultragenyx Pharmaceuticals and Roberta Duncan, VP and mRNA Program Lead at Seqirus offer insight into CMC considerations and entertain audience questions on the topic.
MANUFACTURING RESOURCES
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![GettyImages-1194781834-cell]( "mRNA-Lipid Nanoparticles Circumvent Viral Vector Limitations")
Both physical and chemical nonviral gene delivery systems for ex vivo genetic modification offer advantages over viral vectors, including smaller scale production and the low risk of immunogenicity.
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![Doctor Preparing Injection Site GettyImages-1379313214]( "How LNP Technology Meets The Delivery Need Of saRNA Vaccines")
Explore challenges to the successful development of RNA-based drugs and vaccines as well as the advantages of utilizing lipid nanoparticle technology as a delivery platform for saRNA.
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![Scientist lab cell and gene dna GettyImages-1305079941]( "Ultra-Sensitive Assay For Anti-Sense Oligonucleotide Quantification")
Learn about a new level in sensitivity for challenging analytes in matrix that opens doors for accurate bioanalysis and metabolism studies with specificity not achievable with orthogonal methods.
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![GettyImages-919410014-dna-gene-therapy-future-technology]( "Purification Of DNA Oligonucleotides Using An Anion Exchange Resin")
Learn how a strong anion exchange resin can be used for the efficient purification of single-stranded DNA oligonucleotides used in clinical trials.
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![iStock-879831370-plasmid-lab-cell]( "Pre-Banking And Avoiding Manufacturing Challenges For GMP Plasmids Containing Unstable Sequence Regions")
Ensuring consistent replication and proper retention of plasmid DNA sequence is vital to the economics and efficiency in generating starting materials used for production of AAV delivered gene therapy and mRNA vaccines and therapeutics.
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![GettyImages-1269045566-rna]( "Ensure High Quantity And Quality Of Isolated Bacterial Ribonucleic Acid (RNA)")
In this scientific brief we evaluate the performance of the the Pall Nucleic acid binding (NAB) Nanosep centrifugal device for nucleic acid isolation.
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![Scientist In Lab GettyImages-1354172647]( "Boost LNP Drug Potency With Optimized Delivery Formulations")
Lipid nanoparticles (LNPs) function as a drug delivery system for nucleic acids. Learn about the biomedical applications of LNPs as well as the anatomy, formulation optimization, and safety of LNPs.
