article thumbnail

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. The post New CEO Mortazavi takes UK biotech e-therapeutics into gene silencing appeared first on.

article thumbnail

NIH renews knockout mouse project for 3rd time

Drug Discovery World

‘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. . Institutions in 15 countries.

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

The trends driving ELRIG Drug Discovery 2022

Drug Discovery World

This year’s event will cover some of the most exciting advancements in areas spanning screening, automation, high content imaging, disease models, cell and gene therapies and how innovation is being driven through partnerships and collaborations. Cell and gene therapies. In 2021, levels of investment hit an all-time high at $22.7

article thumbnail

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Pharma Marketing Network

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.

article thumbnail

Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells through epigenetic editing without altering the genetic sequence itself. It’s a great tool for controlling gene expression.”. pyogenes dCas9.

DNA 98
article thumbnail

UK’s NHS Backs World’s Costliest Drug Libmeldy for the Treatment of Rare Disease MLD

XTalks

Related: Gene Silencing Porphyria Treatment, Givlaari, Finally Wins Over England’s NICE Amid Stellar Long-Term Data. It is an autosomal recessive disorder, which means that if both parents are carriers of the faulty gene that causes the disease, there is a one in four chance that their child will develop the disease.

article thumbnail

Using CRISPR to Edit the Epigenome Might Be Easier Than We Thought

XTalks

Researchers at the University of California San Francisco (UCSF) and the Whitehead Institute have developed a novel CRISPR-based tool called “CRISPRoff” that can switch off genes in human cells without editing the genetic sequence itself. These modifications regulate gene expression without altering the sequence or structure of DNA.

DNA 52