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.
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.
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