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14th Annual RNA Therapeutics Conference

pharmaphorum

14th Annual RNA Therapeutics. Investigating the next generation of genetic medicine through RNA based therapies. RNA therapeutics is a rapidly expanding industry with increasingly growing potential for immunotherapy, personalised medicines, and treatment of genetic, infectious, and chronic diseases. Date: 8 – 9 February 2023.

RNA 52
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Lipid nanoparticles and mRNA used to treat hereditary blindness

Drug Discovery World

Oregon State University College of Pharmacy scientists have shown that lipid nanoparticles and messenger RNA could treat blindness associated with a rare genetic condition. . Limitations of AAV gene therapy . We’re hoping to use what we’ve learned so far about LNPs to develop an improved gene editor delivery system.” .

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AI-designed protein awakens silenced genes, one by one

The Pharma Data

By combining CRISPR technology with a protein designed with artificial intelligence (AI), it is possible to awaken individual dormant genes by disabling the chemical “off switches” that silence them. The chemical modifications that regulate gene activity are called epigenetic markers. it can be reawakened.

Protein 52
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New CRISPR-ready neurons could democratise genomics

Drug Discovery World

bit.bio has launched the first offering from its new product range ioCRISPR-Ready Cells, which allow research and drug discovery scientists to knockout any gene of interest in glutamatergic neurons. With ioCRISPR-Ready Cells, we’re putting the future of genomics in the hands of every scientist.

Genome 52
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New CRISPR-Based Tool Called PASTE Gene Editing Inserts Large DNA Sequences at Desired Sites

XTalks

Expanding upon the CRISPR-Cas9 gene editing system, researchers at MIT have designed a new technique called PASTE gene editing that can cut out defective genes and replace them with new genes in a safer and more efficient way. The PASTE gene editing technique was recently published in Nature Biotechnology.

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Why genomic healthcare data matters in the development of new therapies 

Drug Discovery World

In April 2022, PacBio expanded a research collaboration with Children’s Mercy Kansas City in an effort to improve scientists’ understanding of the genetic underpinnings of rare diseases. The scientists said the results support the use of WGS as a standard diagnostic tool within routine clinical practice and the study 3. .

Genome 98
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Where is the drug discovery expertise happening in the UK?

Drug Discovery World

A company within this ecosystem is STORM Therapeutics, a clinical stage biotechnology company creating novel small molecule therapies that inhibit RNA modifying enzymes (RME) for use in oncology and other diseases. billion in funding.

Drugs 75