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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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Mitochondrial drugs, with a twist: Pretzel Therapeutics launches with $72.5M in funding

Bio Pharma Dive

Scientists at Pretzel believe fixing mutated mitochondrial DNA with a mix of small molecule therapies and gene editing could be key to solving a number of hard-to-treat diseases.

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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 editing: beyond the hype

pharmaphorum

Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome. That would just slow the whole field down.

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Intellia cleared to start key CRISPR drug study in UK

Bio Pharma Dive

The trial would be the first test of a systemically administered gene editing treatment that uses the Nobel Prize-winning technology to alter DNA.

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CRISPR gene editing ‘cancer shredding’ technique destroys brain tumours

Drug Discovery World

Scientists have used CRISPR gene editing to target and rapidly destroy glioblastoma cells in an approach that could apply to other highly mutated cancers. The post CRISPR gene editing ‘cancer shredding’ technique destroys brain tumours appeared first on Drug Discovery World (DDW).

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Gene editing platform could shape future application of CRISPR-based therapeutics

Drug Discovery World

A team of researchers at Northwestern University in the US has devised a new platform for gene editing that could inform the future application of CRISPR-based therapeutics. They created a system to deliver the cargo required for generating the gene editing machine known as CRISPR-Cas9. High gene editing efficiency.