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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 Therapy Definition.

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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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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? Let’s take CAR T cells as an example.

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Delivering on the promise of gene editing

Drug Discovery World

Prime editing, while also employing a single strand nick, expands this scope to all 12 possible changes using a more complex prime editing guide RNA (pegRNA). Improve delivery: Clinical translation of gene therapy is dependent on the efficient and safe delivery of gene editing tools into cells, and this can occur either ex vivo or in vivo.

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Clinical Catch-Up: December 21-25 | BioSpace

The Pharma Data

Galidesivir is a broad-spectrum antiviral, an adenosine nucleoside analog that blocks viral RNA polymerase. STP705 is a small interfering RNA (siRNA) therapy that leverages a dual-targeted inhibitory property and polypeptide nanoparticle (PNP)-enhanced delivery to knock down both TGF-beta1 and COX-2 gene expression.

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