Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Drug Discovery World

SEPTEMBER 5, 2022

‘Knockout’ mice are bred in a laboratory with specific genes silenced or ‘knocked out’. The goal of this final phase is to produce and phenotype hundreds of knockout mouse models for genes with little to no known function, to better understand the genetic bases for diseases in humans and animals. .

Gene Silencing 52

Gene Silencing Gene Genetics Genome 52

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).

Gene Therapy 98

Gene Therapy Drugs Gene Gene Silencing 98

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.

RNA 52

RNA Drugs Gene Silencing Vaccination 52

Drug Discovery World

JANUARY 23, 2023

Increasing interest in CRISPR-Cas gene editing systems over recent years has delivered innovative developments in precision genome engineering technologies, holding enormous potential for applications across the healthcare sector. Dis Model Mech. 2020;13:dmm042317. doi:10.1242/dmm.042317. 13: Cuomo A, Seaton D, McCarthy D, et al.

Gene Editing 52

Gene Editing Gene Engineer DNA 52

Drug Discovery World

OCTOBER 4, 2022

Cell and gene therapies. There’s no question that cell and gene therapies have emerged as some of the most promising and innovative medicines on the market. . With 3,633 advanced therapeutical medicinal products (ATMPs) currently in some form of clinical or pre- clinical development, the sector shows no signs of slowing down.

Gene Therapy 52

Gene Therapy Drugs Gene Manufacturing 52

pharmaphorum

JUNE 28, 2021

The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening rare disease. . — Eric Topol (@EricTopol) June 26, 2021. . Regeneron has partnered the company since 2016.

In-Vivo 105

In-Vivo Gene Editing Trials Drugs 105