Not long after I posted this in August last year, a single-patient trial commenced in a patient with Duchenne, using a CRISPR therapeutic delivered by a high-dose rAAV9 vector.
The patient died 8 days after dosing.
A paper has now been published in The New England Journal of Medicine attributing the likely cause of death to an innate immune reaction triggered by the AAV vector.
This case highlights once more the safety challenges of “one and done” therapies.
https://www.nejm.org/doi/full/10.1056/NEJMoa2307798
https://www.biospace.com/article/pa...deliver-gene-editing-therapy-nejm-study/?s=89
https://www.cgtlive.com/view/crispr-therapeutic-ready-to-dose-in-dmd
https://www.insideprecisionmedicine...g-the-clinical-outlook-for-multiple-diseases/
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