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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

Most recently, these companies include: Fibrocor Therapeutics; Encyte Therapeutics (acquired by Zealand Pharma A/S); and Notch Therapeutics, a biotechnology company focused on gene-edited T cell therapies, which has a strategic partnership with Allogene Therapeutics.

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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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Leading innovators in CRISPR nucleases for the pharmaceutical industry

Pharmaceutical Technology

The eukaryotic genome can be cut at any desired position by introducing plasmids containing Cas genes and specifically constructed CRISPRs into eukaryotic cells. CRISPR nucleases serve as an important genome editing tool. The company is largely involved in genome editing using CRISPR/Cas9 and CRISPR/Cas12a systems.

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