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Gene Therapy and Pharmacokinetics

Camargo

Additionally, gene editing allows us either to remove or to modify harmful genes. While most gene therapy clinical studies are ongoing, a number of products are in advanced clinical development, and several are approved by FDA. Gene Expression Considerations. Route of Administration 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. SB: Certainly.

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

pharmaphorum

million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. 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 57
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Personalised mRNA cancer vaccine prolongs survival in melanoma

Drug Discovery World

The clinical benefit was observed regardless of the tumour mutational burden (TMB) status, according to results from the Phase IIb KEYNOTE-942 clinical trial presented at the AACR Annual Meeting 2023, held April 14-19. The association between this treatment approach and TMB will be further explored in upcoming studies.

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How big data analytics can make personalised care a reality

Drug Discovery World

Typically, the data obtained from such studies is large and high dimensional with thousands of molecules being measured for each individual sample requiring special statistical, data science, and bioinformatics skills to integrate the information and correlate it with the clinical outcome of interest.

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What spatial biology can tell us about disease and drug discovery

Drug Discovery World

Analysing spatial changes during disease progression or treatment response is key to fully understand pathologies. RA: With spatial biology being cross-disciplinary – in that it uses techniques in both gene expression analysis and immunofluorescence – is expertise in the field limited?

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Delivering on the promise of gene editing

Drug Discovery World

Now, after nearly 35 years of research and more than a decade of preclinical progress, several different gene editing modalities are being tested in early phase clinical trials. Early phase clinical trials for gene editing therapies. Gene editing challenges and potential solutions. References.