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Gene Therapy and Pharmacokinetics

Camargo

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Expression Considerations.

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How transcriptomics is driving drug discovery

Drug Discovery World

SB: Transcriptomics is the study of genomic RNA transcripts in a biological system. RNA transcript expression levels can provide insight into underlying biology – eg. which genes are upregulated, and which ones are downregulated – which is very useful when studying different disease states.

RNA 52
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Cash round sets up trial of InteRNA’s microRNA for cancer

pharmaphorum

InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

Trials 58
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Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies. Here are 5 considerations when developing a communications strategy for a novel genetic medicine. #1.

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How big data analytics can make personalised care a reality

Drug Discovery World

A key breakthrough area where big data analytics has shown tremendous scope and application is in systems biology, which has bolstered holistic drug research and development, ultimately to provide patients with access to the right therapies. Figure 2: Advanced data visualisation examples for transcriptomics data.

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What spatial biology can tell us about disease and drug discovery

Drug Discovery World

BN : Our bodies consist of an incredibly complex network of about 35 trillion cells divided into as many as 300 cell types that fulfil distinct roles in development, homeostasis, and disease. Analysing spatial changes during disease progression or treatment response is key to fully understand pathologies.

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Delivering on the promise of gene editing

Drug Discovery World

Possible solutions: A new generation of gene editing tools is being developed to avoid double stranded breaks altogether and to be potentially less destructive than traditional nuclease systems. DNA targeting specificity of RNA-guided Cas9 nucleases . In one example, Choi et al. References.