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STAT+: After blazing trails in RNA-targeted drugs, Ionis jumps into gene editing

STAT News

Ionis Pharmaceuticals, the California company that turned RNA-targeting medicines from an unproven idea into blockbuster drugs, is now expanding into a new class of therapeutics that treat disease by editing DNA.    On Monday, the company announced a partnership with Metagenomi to kickstart these efforts.

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Ionis teams with Metagenomi and dives into gene editing

Bio Pharma Dive

The RNA drug developer is paying the richly-funded startup $80 million in a wide-ranging alliance that marks its first foray into DNA editing medicines.

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Gene Therapy and Pharmacokinetics

Camargo

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19. Gene Therapy Definition.

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Junk DNA: How the dark genome is changing RNA therapies

Drug Discovery World

Samir Ounzain , PhD, CEO & Co-Founder of HAYA Therapeutics, looks at how a better understanding of our DNA can lead to increased activity for RNA therapeutics. The whole world realised the power of RNA when the Covid-19 pandemic brought us the first mRNA-based vaccines.

RNA 52
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Is single-cell gene expression the next trend in next generation sequencing? 

Drug Discovery World

By Dr Dina Finan, PhD, Product Manager at 10x Genomics and Dr Nick Downey, PhD, NGS Collaborations Lead at Integrated DNA Technologies. RNA sequencing has become a widely used tool that can explore the transcriptome in a seemingly endless number of applications. appeared first on Drug Discovery World (DDW). Username or E-mail.

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RNA interference method could treat muscular dystrophy

Drug Discovery World

Japanese researchers propose using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama muscular dystrophy. . One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene. One form of FCMD is caused by a genetic abnormality in the ‘fukutin’ (FKTN) gene.

RNA 52
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Using gene therapy to live longer, healthier lives

Drug Discovery World

She discusses with DDW’s Megan Thomas the company’s lead programme; a gene therapy called ER-100. Life Biosciences’ gene therapy approach differs from traditional gene replacement and gene editing approaches in that the company are not altering the DNA sequence.