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Overcoming diverging regulatory expectations to bring CGTs to market

Drug Discovery World

While CGT is still an emerging market, its growth has accelerated over the last five years, resulting in a wave of products approved by the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Driven by the rapid pace of innovation, the market for CGTs is expected to grow from $4.4 billion in 2020 to $15.5

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Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. The company’s nuclease collection includes several Protospacer Adjacent Motifs (PAMs), short sequences that help determine the genome’s DNA segments.

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Leading innovators in gene splicing using nucleases for the pharmaceutical industry

Pharmaceutical Technology

Within the emerging innovation stage, cell therapy for ocular disorders, coronavirus vaccine components, and DNA polymerase compositions are disruptive technologies that are in the early stages of application and should be tracked closely. Nucleases are enzymes that hydrolytically cleave the phosphodiester backbone of DNA.

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Synthetic biology tools advancing and accelerating drug discovery efforts

Drug Discovery World

Rapid and accurate DNA synthesis . Conventional DNA synthesis, typically used for making sequences such as primers, employs phosphoramidite chemistry, a method that was developed in the 1980s and is still considered the gold standard for DNA synthesis. .

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Verve starts trials of cholesterol drug in test of base editing technique

pharmaphorum

Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment. Through base editing, drugs replace single nucleotide in the DNA strand with another, without making double-strand breaks (DSBs) in the gene.

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Genotoxicity Testing: Unlocking the Future Safety Assessment Opportunities

Roots Analysis

Disruption of the integrity and function of DNA at the gene or chromosomal level can lead to heritable mutations, resulting in genetic disorders, birth abnormalities, or cancer. Mechanism of Genotoxicity / Mutagenicity The interaction of genotoxins / mutagens with the structure of DNA causes damage to the genetic material.

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Advances in cell line development

Drug Discovery World

The market for cell line development is currently rising due to an increased focus by the life sciences sector on biopharmaceuticals and targeted therapies. According to Grand View Research, the global cell line development market was valued at $4.81 Bacterial cell lines such as E. coli have their own set of benefits however.