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Laverock Therapeutics raises £13.5m to accelerate gene silencing platform

BioPharma Reporter

Laverock Therapeutics, the gene editing-induced gene silencing platform for human therapeutic applications, has expanded its seed funding round to Â13.5 million.

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Three-in-one approach boosts the silencing power of CRISPR

Scienmag

The ongoing effort to explore […].

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Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal

pharmaphorum

In 2019, for example it agreed to work with Dicerna on gene-silencing for liver-related cardio-metabolic disease in a deal that involved a $225 million upfront payment. The post Novo Nordisk bulks up in rare diseases with $1.2bn Prothena deal appeared first on.

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

Pharma Marketing Network

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. This relinquishes many of the perceived benefits of RNAi therapies that distinguish them from gene therapy (e.g.,

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Intellia, Regeneron ace first trial with ‘in vivo’ CRISPR drug

pharmaphorum

The reductions matched the efficacy of current therapies for ATTR amyloidosis that require chronic dosing such as Alnylam’s Onpattro (patisiran) and Ionis/Akcea’s Tegsedi (inotersen) – both gene-silencing agents which can cost around $450,000 a year. — Eric Topol (@EricTopol) June 26, 2021.

In-Vivo 105
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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.”.

DNA 98
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The use of base editing in stem-cell based therapies

Drug Discovery World

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. Adult fibroblasts were cultured in a cocktail of transcription factors to reactivate embryonic gene expression patterns 2.