XTalks

OCTOBER 26, 2021

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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Gene Silencing Gene Clinical Trials Clinical Trials 98

XTalks

OCTOBER 27, 2021

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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Gene Silencing Life Science FDA Approval Gene 40

XTalks

MAY 4, 2021

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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DNA Gene Gene Silencing Genomics 98

XTalks

JUNE 14, 2023

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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Gene Silencing Drug Delivery FDA Approval RNA 97

XTalks

FEBRUARY 14, 2022

Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. It is an autosomal recessive disorder, which means that if both parents are carriers of the faulty gene that causes the disease, there is a one in four chance that their child will develop the disease.

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pharmaphorum

JUNE 8, 2022

Zilebesiran (formerly ALN-AGT) is one of a new breed of gene-silencing drugs that are intended to treat common, chronic diseases with infrequent dosing to boost compliance with treatment.

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XTalks

MAY 4, 2021

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. .

DNA 52

DNA Gene Gene Silencing Genomics 52