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US FDA grants Fast Track status for Avidity’s AOC 1020 to treat FSHD

Pharmaceutical Technology

AOC 1020 has been designed for the treatment of the underlying cause of FSHD, which is caused by the abnormal expression of a gene known as double homeobox 4 or DUX4. Avidity Biosciences’ AOC 1020 is intended to reduce the DUX4 mRNA and DUX4 protein expression in muscles in these patients.

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Leading innovators in transcription factors for genetically modified cells for the pharmaceutical industry

Pharmaceutical Technology

Adeno-associated virus vectors, alcohol dehydrogenase compositions, and antibody serum stabilisers are some of the accelerating innovation areas, where adoption has been steadily increasing. Among maturing innovation areas are anti-influenza antibody compositions and anti-interleukin-1, which are now well established in the industry.

Genetics 130
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Cell-penetrating peptides as a delivery system for oligonucleotides

Drug Discovery World

Oligonucleotides act on the RNA level through different molecular pathways, but there is a major drawback when using them as drugs: their poor bioavailability and cellular uptake. Over the years, a range of different CPPs have been developed, including natural translocating proteins through to newly-designed computer-prediction sequences 3.

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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

RNA 40
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Synthetic biology tools advancing and accelerating drug discovery efforts

Drug Discovery World

Applications: The ability to synthesise longer, accurate DNA sequences opens up a vast array of application possibilities: generating CRISPR libraries, in situ hybridisation probes, antibody libraries, vaccines, and so on, along with the ability to engineer metabolic pathways, microbes, and plants. .

DNA 98
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What spatial biology can tell us about disease and drug discovery

Drug Discovery World

Today, spatial omics technologies enable imaging-based identification of cells in their native tissue context while simultaneously detecting hundreds of RNAs or tens of protein markers in the same cells. Analysing spatial changes during disease progression or treatment response is key to fully understand pathologies.

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How big data analytics can make personalised care a reality

Drug Discovery World

This approach has led to the development of two software packages for RNA sequencing analysis and reproducible ribosomal profiling 1,2. For example, we recently collaborated with two sequencing laboratories to determine optimal parameters for RNA-Sequencing (RNA-Seq) in the context of a vaccine clinical trial.