Insights on Discovery
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Comparing Second Strand Synthesis Processes To Establish rAAV Batch Quality
2/5/2025
In this study, we present a comprehensive analysis of various published and newly developed dsDNA conversion processes that were tested using the same sequencing chemistry.
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Targeted CRISPR/Cas9 Screen Identifies Superior HEK293 Cell Lines For AAV9 Production
2/5/2025
In this study, we developed a proprietary clonal suspension-adapted HEK293 cell line (AC001.230) through clonal isolation and high-throughput screening, resulting in improved productivity for 7 out of 10 AAV serotypes compared to 293F cells.
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A High-Throughput Small Molecule Screen Identifies Targets That Increase AAV9 Production In Suspension HEK293 Cells
2/5/2025
Consider the potential of this small molecule screening to enhance AAV9 production and improve gene therapy manufacturing efficiency by leveraging novel compounds.
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Plasmid Impurity Sizing Of The nptII Kanamycin Resistance Gene In Recombinant Adeno-Associated Virus By Droplet Digital Polymerase Chain Reaction
2/5/2025
Explore our novel ddPCR method for assessing plasmid-derived antibiotic resistance gene impurities in rAAV products to enhance regulatory compliance and ensure the safety and quality of gene therapy vectors.
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Method Qualification Of Mass Photometry For The Determination Of Full-To-Empty Capsid Ratio For rAAVs Using The Refeyn System
2/5/2025
Leverage mass photometry to quickly and accurately monitor the full-to-empty ratio of rAAV samples, ensuring consistent product quality and enhanced safety and efficacy in gene therapy applications.
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Design Of Experiment (DoE) Based High Throughput Process Optimization For rAAV Manufacturing Development
2/5/2025
This study presents the results of a Design of Experiment (DoE) approach to assess how these process parameters impact rAAV yield and quality.
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Quality Over Quantity: Where Is The Trade Off?
2/5/2025
Our study aims to compare two widely used transfection reagents to our standard process, evaluating their impact on final product quantity and quality as part of our ongoing platform innovation efforts.
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New Hope For Neuronal Diseases With Polymer Nanoparticle-Based Gene Therapy
1/28/2025
A new polymer nanoparticle (PNP) system enables targeted delivery of large genetic payloads to neuronal cells, advancing gene therapies for NF1 and other nervous system disorders.
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Fit-For-Purpose Biomarker Development
1/17/2025
Here, we highlight the essential framework of a “fit-for-purpose” biomarker development strategy, emphasizing strategic and transactional engagements with clinical research organizations.
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Biomarkers In Oncology Studies
1/17/2025
Here we provide an overview of oncology biomarker development and strategic considerations.