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Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

JMB: Regulatory RNAs (or regRNAS) are molecules that directly control the expression of nearby protein-coding genes. CAMP4 is exploiting these regulatory interactions to specifically control the expression of genes tied to disease. What diseases are you currently targeting? What are the challenges of drug discovery in this area?

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Searching for answers in rare epilepsy

pharmaphorum

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 86
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Searching for answers in rare epilepsy

pharmaphorum

Geneticist Dr Charles Steward has spent his career studying the human genome – but his work became much more personal when his children were diagnosed with severe neurological diseases. Charlie told pharmaphorum how his search for a genetic cause has led him to straddle the divide between scientist and patient advocate.

Genome 73
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The future outlook for mRNA therapies

Drug Discovery World

Its job is to carry coding information that is essential to the translation and processing of functional proteins. This is essential to its use as a therapeutic agent and gives the technology a vast versatility making it suitable to treating a wide range of diseases – especially those that have high protein expression.

RNA 52
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Unlocking the potential of mRNA for the future treatment of rare diseases 

Drug Discovery World

As we turn our focus to new potential applications and disease areas for the platform, scientists and companies must consider the potential life-saving impact on rare, inherited diseases, says Archana Gupta , PhD, staff scientist in genetic sciences at Thermo Fisher Scientific.

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Epigenetic Editing with CRISPR Might Be Easier Than We Thought

XTalks

The tool could also prove to be safer than conventional CRISPR-based gene therapies as it does not involve DNA editing, and thus would not cause potentially harmful off-target genomic changes. Epigenetic Editing with CRISPR. DNA methylation occurs endogenously in all mammalian cells in response to various stimuli.

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Meet the industry: Who’s at Drug Discovery 2023?

Drug Discovery World

New approaches and thought processes When it comes to diversity in drug discovery, this includes our scientists’ and pharma workforce’s approach to innovation which will help advance the industry. For example, to identify protein structures and even binding pockets from sequence data and modelling potential hits in silico.

Drugs 59