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Gene Therapy and Pharmacokinetics

Camargo

Gene therapy is a new therapeutic approach in which genes are used to treat or prevent diseases. It is a comprehensive term which encompasses a large variety of therapy products including viral and bacterial vectors, plasmid DNA, human gene editing technology, and patient-specific cellular gene therapy.

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Bayer trumpets $1bn CRISPR deal with Mammoth Bio

pharmaphorum

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells. Casgevy’s approval by the FDA is momentous: it is the first CRISPR-based gene-editing therapy to be approved in the US.

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Beyond the pandemic: the potential of mRNA technology

pharmaphorum

The company has also stated, at the same time as the study was announced, that it will look at developing mRNA technology in oncology and genetic diseases. At present, the biotech has 10 vaccine candidates at various stages of the preclinical and discovery phase, focused across three different areas of infectious diseases.

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Key Trends in the Life Sciences to Look Forward to in 2022

XTalks

The RNA Revolution: From mRNA Vaccines to RNA Editing. RNA, and its protein-generating form messenger RNA (mRNA) discovered in 1961 , has quickly transitioned from being an obscure, finicky molecule that is difficult to work with, to becoming a significant cornerstone of therapeutic innovation in pharma and biotech.

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Advances in neuroscience drug discovery

Drug Discovery World

For example, it can take up to it three years to recruit enough participants for a dementia clinical trial. years it takes to complete an entire cancer clinical trial. The organisation also notes that only 1% of the people that can take part in dementia clinical trials actually do so. This compares to the 2.3

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