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First Patient Enrolled in Phase 1 Gene Therapy Study in Ireland

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  • Guy
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    It's certainly one of the most promising approaches at the moment. This follows on from the SOD1 study and is similar (in part) to the successful work done to treat SMA. You may have seen the recent paper on the SOD1 study; summary in https://investors.biogen.com/news-re...sults-phase-12 Hopefully there will be a more detailed write up later including the results of the additional data held but even the published data suggests about a five fold reduction in ALS FRS-R rate of decline. If they can apply it using viral vectors, rather than lumbar puncture, then it might be a single treatment.

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  • Graham
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    Thanks JD. I suspect this is the only way to cure MND.

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  • First Patient Enrolled in Phase 1 Gene Therapy Study in Ireland

    A Phase 1 clinical trial of a gene-based therapy for amyotrophic lateral sclerosis (ALS) is now recruiting patients in one of its European centers.

    The Clinical Research Facility at St James’s Hospital Dublin, in Ireland, has enrolled its first patient and is one of four European centers to participate in this study.

    Sponsored by the U.S. pharmaceutical company Biogen, the trial involves a new gene therapy, called BIIB078, that targets a mutation in the ALS-associated gene C90rf72. Known as an expansion, this mutation occurs when a sequence of DNA “letters” — which stand for the chemicals that make up DNA — is repeated within the gene.

    The therapy, developed in collaboration with Ionis Pharmaceuticals, prevents the production of abnormal forms of the protein encoded by the C90rf72 gene while preserving normal forms of this protein.

    “Up to 10% of people in Ireland with MND [motor neuron disease] carry an abnormal expansion of the C9orf72 gene,” Orla Hardiman, the principal investigator for the Irish part of the study, said in a press release.

    https://alsnewstoday.com/news-posts/...dy-in-ireland/

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