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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Drug Discovery World

OCTOBER 4, 2022

There’s no question that cell and gene therapies have emerged as some of the most promising and innovative medicines on the market. . billion in 2020, according to the Alliance for Regenerative Medicine’s (ARM’s): Disrupting the Status Quo report. In 2021, levels of investment hit an all-time high at $22.7

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pharmaphorum

DECEMBER 23, 2020

But there was some considerable progress in other fields of medicine even though research efforts were diverted away, reports Richard Staines. Calliditas said part A of the global phase 3 NeflgArd clinical trial has met its target, measuring the effect of Nefecon versus placebo in patients with the disease known as IgAN for short.

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XTalks

FEBRUARY 14, 2022

The UK’s National Health Service (NHS) has recommended the use of Libmeldy for the treatment of the rare genetic disease metachromatic leukodystrophy (MLD). Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. million (approximately $3.8 million USD).

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Drug Discovery World

AUGUST 1, 2023

The NIH states: “This natural mechanism for sequence-specific gene silencing promises to revolutionise experimental biology and may have important practical applications in functional genomics, therapeutic intervention, agriculture and other areas.” There are many examples of ways in which RNAi is impacting drug discovery.

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The Pharma Data

NOVEMBER 24, 2020

PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. Oxlumo marks our third FDA approval in less than three years, positioning us to meet or exceed our Alnylam 2020 strategy and goals, and further highlighting the productivity of our RNAi platform and the speed at which we can bring innovative medicines to patients.

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The Pharma Data

APRIL 27, 2023

These early results establish the first human translation of Alnylam’s proprietary C16-siRNA conjugate platform for central nervous system (CNS) delivery and are the first clinical demonstration of gene silencing in the human brain using an RNAi therapeutic.

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