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Study in mice shows potential for gene-editing to tackle mitochondrial disorders

Scienmag

Defective mitochondria – the ‘batteries’ that power the cells of our bodies – could in future be repaired using gene-editing techniques. Our cells contain mitochondria, […].

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How Potential Changes to the NIH Guidelines Could Impact IBC Review

WCG Clinical

Although the NIH Guidelines were originally written with non-clinical laboratory research in mind, they also apply to human gene transfer (HGT) research, wherein rsNA or rsNA-containing products are administered to research participants. Since then, however, certain genetic engineering technologies (e.g.,

Genome 52
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LogicBio Therapeutics Receives FDA Fast Track Designation for LB-001 for the Treatment of Methylmalonic Acidemia (MMA)

The Pharma Data

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

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Scopus BioPharma Announces Appointment of Additional Directors

The Pharma Data

Scopus is a biopharmaceutical company whose lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. The company’s lead drug candidate is a novel, targeted immuno-oncology gene therapy for the treatment of multiple cancers. Scopus BioPharma Inc.

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Using bispecific antibodies to advance cancer immunotherapy

pharmaphorum

Droplet digital PCR (ddPCR) technology provides a sensitive, accurate, and absolute quantification of gene edit confirmation in cell line development, and the gene copy number can be reproducibly determined to assure safety and stability.

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Lineage Cell Therapeutics Provides End of Year Shareholder Letter

The Pharma Data

Notably, we never modify the DNA of these cells, which avoids some of the safety concerns that have been reported with certain gene-editing technologies. Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements.

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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.”.

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