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Baldwidx I have previously discussed here gene therapies (DNA...

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    Baldwidx

    I have previously discussed here gene therapies (DNA therapies) and raised specific concerns about the viral vectors that are used for delivering gene therapies, which are meant to be a “one and done” treatment. I’ve also mentioned adverse events that can result from the immunosuppressant drugs that are used prophylactically with viral vector delivered gene therapy.

    PYC Therapeutics uses antisense oligonucleotides, which are one type of RNA therapy. Antisense therapies don’t have a permanent effect like gene therapies and typically are dosed every couple of months. PYC is employing peptide conjugation to enhance cellular delivery of its antisense therapies. This allows lower doses that are less likely to cause serious adverse events.

    As my grasp of genetic medicine is limited, I've included a PDF link below to an information sheet on the different gene therapy approaches.

    https://hotcopper.com.au/threads/competitors-to-daybue.7335954/page-19?post_id=74384394

    https://hotcopper.com.au/threads/neurogene-for-rett.7195113/page-7?post_id=65892502

    ASGCT-Gene-Therapy-Approaches-12-22-22-update.pdf
 
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