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. .

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Drug Discovery World

OCTOBER 4, 2022

DDW Editor Reece Armstrong explores some of the trends that will be featured in sessions throughout ELRIG Drug Discovery 2022 and the news behind them. 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. Automation.

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pharmaphorum

DECEMBER 13, 2020

The deal – the largest in the pharma sector since the start of the pandemic – bolsters AZ’s immunology franchise with $4 billion blockbuster Soliris (eculizumab) and longer acting follow-up Ultomiris (ravulizumab), plus a pipeline of 11 drugs for rare and autoimmune diseases.

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pharmaphorum

DECEMBER 23, 2020

Originally developed to treat late-stage EGFR-mutated disease, the ADAURA study tested Tagrisso as an adjuvant treatment in patients with early disease following surgery. The results also suggest further uses for the drug in early disease and more revenues to come from one of the company’s biggest success stories from the last decade.

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pharmaphorum

OCTOBER 29, 2020

Already a major player in gene therapy, Novartis has swooped on US startup Vedere Bio in a $280 million deal that builds its position in inherited eye diseases that can lead to blindness. It also gets two preclinical-stage development projects. IRDs affect around 2 million people worldwide.

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Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

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Drug Discovery World

JANUARY 23, 2023

Kevin Hemphill, R&D Manager at PerkinElmer’s Horizon Discovery explores how base editing has emerged as an attractive gene editing option for researchers wanting to develop stem cell-based therapies. In addition, iPSC lines, despite their pluripotency, have skewed suitability towards certain cell types 12,13.

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The Pharma Data

NOVEMBER 24, 2020

Food and Drug Administration (FDA) approved Oxlumo (lumasiran) injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction.

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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. The post Alnylam finds belly fat gene and drug target in UK Biobank study appeared first on.

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Roots Analysis

OCTOBER 22, 2023

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. These therapeutics are broadly classified into two categories, namely coding RNAs and non-coding RNAs.

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