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After initial rejection from the National Institute for Health and Care Excellence (NICE) last year, the non-departmental public body of the Department of Health in England has now given the green light to the genesilencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).
The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper. . Epigenetic Editing with CRISPR.
In addition, Sylentis, a biotech company specializing in ophthalmic drugs and RNA interference (RNAi) technology, initiated a Phase III study in 2021. Tivanisiran is a preservative-free eye drop that uses RNAi technology, specifically genesilencing, to target and control the signs and symptoms of dry eye disease.
The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Genes can be switched on with a complementary tool called CRISPRon that has also been described in the paper. . Epigenome Editing with CRISPR.
It has been a year dominated by the pandemic and many lifesciences research projects were put on hold as big pharma turned its attention to vaccines and therapies. It was the third approval from Alnylam’s pipeline of RNA interference therapeutics to make it to market.
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