What are biomarkers?
Biomarkers are measurable indicators of a biological state or condition. They reveal the molecular and genetic mechanisms underlying disease, helping researchers identify precise therapeutic targets. Because many biomarkers arise directly from affected organs, patient samples can uncover valuable diagnostic and treatment targets. This has accelerated the development of advanced diagnostics and targeted therapies, such as gene-editing therapies tailored to correct specific genetic mutations or cell-based therapies engineered to enhance immune responses. By enabling patient stratification, predicting treatment response, and monitoring safety and efficacy, biomarkers are essential to the development and optimization of modern therapies.
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Charting a path with biomarker discovery tools
What is a control strategy?
Control strategy is important for any drug to achieve the appropriate product quality, and essential for novel products/class of products to define and achieve critical quality attributes (CQA). Per ICHQ10, control strategy is defined as “A planned set of controls, derived from current product and process understanding that assures process performance and product quality. The controls can include parameters and attributes related to drug substance and drug product materials and components, facility and equipment operating conditions, in-process controls, finished product specifications, and the associated methods and frequency of monitoring and control.”
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A standardized approach to CGT quality management
Orchestration platforms refer to software infrastructure that coordinates the extremely complex operational workflow required to deliver CGTs to a patient. These technology platforms help treatment centers and companies manage ordering, scheduling, delivery, and infusion workflows safely and efficiently, ensuring safe and timely delivery of personalized therapies.
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Orchestrating cell and gene therapies
What are adeno-associated viruses (AAV)?
AAVs (adeno-associated viruses) are small, engineered viruses used as delivery vehicles to carry therapeutic DNA into human cells. Because they do not cause disease and are highly efficient at delivering genetic material, they are popular tools for treating genetic diseases by replacing or silencing faulty genes, particularly in the CNS and liver. AAV gene therapy has emerged as a frontrunner in the CGT landscape, due to its favorable safety profile, long-term transgene expression, minimal immunogenicity, and a lifetime single-dose administration for the treatment of a majority of disease indications.
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Advancing AAV production
What is a seed train?
A seed train is the sequential scale-up process used to expand producer cells from a small starting culture into the large cell population required for manufacturing viral vectors or advanced therapies. The expansion process ensures sufficient cell numbers and viability for manufacturing, while maintaining product quality.
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Optimizing seed train processes