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Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data

XTalks

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 gene silencing treatment Givlaari (givosiran) for the treatment of the rare metabolic disorder, acute intermittent porphyria (AIP).

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

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.

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Vevye: A New Cyclosporine Solution for Dry Eye Disease

XTalks

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 gene silencing, to target and control the signs and symptoms of dry eye disease.

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Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

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.

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2020 review – Pharma’s progress outside of COVID-19

pharmaphorum

It has been a year dominated by the pandemic and many life sciences 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.