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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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New CEO Mortazavi takes UK biotech e-therapeutics into gene silencing

pharmaphorum

UK biotech e-therapeutics has a new CEO, with executive chairman and former Silence Therapeutics chief Ali Mortazavi chosen to spearhead the next stage in the company’s development into gene silencing and other areas. fundraiser in July for the next stage of its development. million compared with £3.8

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Novo Nordisk’s gene silencing alliance with Dicerna bears first fruit

pharmaphorum

Two years after starting to work together, Novo Nordisk and Dicerna have selected the first candidate from a joint project to find new, gene-silencing drugs for liver-related cardiometabolic diseases. The nomination of a first candidate in the collaboration that will advance into development triggers a $2.5

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Innovate UK funding for new precision cancer treatment technology

pharmaphorum

Programmable Oligonucleotide Delivery System (PODS), developed by Sixfold Bioscience, is a versatile system which delivers short interfering RNA (siRNA) gene silencing cargo to specific cancer cells.

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A new strategy for siRNA stabilization by an artificial cationic oligosaccharide

Scienmag

Credit: TMIMS RNA interference is a gene regulatory mechanism in which the expression of specific genes is downregulated by endogenous microRNAs or by small interfering RNAs (siRNAs). To improve their stability, most of the developed siRNA-based […].

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Alnylam gets NICE backing for porphyria therapy Givlaari

pharmaphorum

UK cost-effectiveness watchdog NICE is set to recommend NHS use of Alnylam’s gene-silencing therapy Givlaari in England and Wales for the rare disease acute hepatic porphyria (AHP), after its advisors issued a positive verdict on the drug. This is a huge development for patients, more info at: [link] !

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RNA Therapeutics: A Novel Approach to Treating Diseases

Roots Analysis

RNA therapeutics are a novel class of biopharmaceuticals that harness the power of RNA molecules for the treatment and prevention of a wide range of disorders, including oncological, and genetic disorders as well as infectious diseases. Non-coding RNAs include antisense oligonucleotides (ASOs) and RNA aptamers.

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