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

Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. This includes Denali Therapeutics whose treatment TAK-594/DNL593 is in a Phase I/II trial. The trial features four cohorts. This protein promotes lysosomal function.

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Development Gene Therapy Protein Gene 246

pharmaphorum

JULY 28, 2022

Alnylam Pharma has made a name for itself, developing gene-silencing therapies for rare disorders, but its latest discovery could take it into a much larger category – metabolic and cardiovascular disease. Transgenic mice in which the gene has been deleted have improved control of blood glucose and insulin sensitivity.

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Biobanking Gene Gene Silencing Drugs 52

XTalks

JUNE 14, 2023

The clinical trials consistently showed significant therapeutic improvements in ocular surface damage and associated symptoms, while pioneering visual function improvement analysis”, said John D. Additionally, the clinical trials underscored the favorable tolerability profile of Vevye.

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pharmaphorum

JULY 13, 2021

Novo Nordisk also gets rights to the remainder of Dublin-based Prothena’s ATTR amyloidosis programme under the terms of the deal, although PRX004 is the only candidate that has reached the clinical trials stage. All told, the deal could be worth up to $1.2

Gene Silencing 98

Gene Silencing Sales Gene Editing Clinical Trials 98

pharmaphorum

FEBRUARY 9, 2021

million ($22 million) in second-round financing as it prepares to start clinical trials of its lead drug candidate INT-1B3 for solid tumours. InteRNA is focusing on the development of gene-silencing drugs based on microRNA, naturally occurring, non-coding strands of RNA that are thought to regulate gene expression in cells.

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Trials Gene Silencing RNA Gene Expression 57

XTalks

FEBRUARY 14, 2022

The drug’s approval in the EU was based on analysis of clinical trial data including both pre- and early-symptomatic, and early-onset MLD patients, which showed that a single-dose treatment is effective in modulating disease progression in most early-onset MLD patients. The incidence of MLD is approximately one in 100,000 worldwide.

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Gene Therapy Drugs Gene Gene Silencing 98