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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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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. The start of dosing in the phase 1/1b trial of the drug has been delayed by the COVID-19 pandemic, but recruitment of the target 80 participants with advanced solid tumours is reported to be ongoing.

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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.

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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. In addition to encoding the target antigens, mRNA vaccines also provide adjuvant properties that amplify the immune response.

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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. greater safety, transient and reversible effect on gene expression).

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How big data analytics can make personalised care a reality

Drug Discovery World

It’s a rapid growth area for clinical trials in the next few years as we seek to understand the underlying biology behind many of the targets and diseases we study. This approach has led to the development of two software packages for RNA sequencing analysis and reproducible ribosomal profiling 1,2.

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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?