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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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FDA Approves First Interchangeable Humira Biosimilar + Gene Silencing Treatment for Porphyria Gets Nod From England’s NICE – Xtalks Life Science Podcast Ep. 34

XTalks

Ayesha also talked about a new gene silencing treatment for porphyria called Givlaari that received recommendation from England’s NICE after having been initially rejected by the health watchdog last year. Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data.

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Vevye: A New Cyclosporine Solution for Dry Eye Disease

XTalks

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. It is a small synthetic double-stranded RNA oligonucleotide (siRNA) designed for this purpose, representing the state-of-the-art in gene silencing technology.

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The 3rd Annual RNAi – Based Therapeutics Summit Returns!

pharmaphorum

With 28 pioneering speakers from large pharma, innovative biotech and KOLs of academia who are ready to discuss the full and comprehensive range of RNAi drugs from discovery to development and beyond, join us in Boston to hear how they address the major challenges facing the industry.

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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. But there was some considerable progress in other fields of medicine even though research efforts were diverted away, reports Richard Staines. Rare disease progress.

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The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

Taking place at the ExCel in London from 4-5 October, ELRIG Drug Discovery 2022 is set to provide visitors with access to the latest trends, technologies and expertise driving the life sciences sector. . Knockout’ mice are bred in a laboratory with specific genes silenced or ‘knocked out’.

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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. .

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