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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. Zinc fingers. That would just slow the whole field down.

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ElevateBio raises funds to advance cell and gene therapies

Pharmaceutical Technology

ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.

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Intellia Gets FDA Clearance to Start First Ever Phase III Trial for an In Vivo CRISPR Drug

XTalks

Clinical-stage genome editing company Intellia Therapeutics has received clearance from the US Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to start a pivotal phase III trial of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis with cardiomyopathy.

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ElevateBio and Pittsburgh University to establish biomanufacturing centre

Pharmaceutical Technology

This centre will be equipped with its supporting technologies, such as gene editing induced pluripotent stem cell (iPSC) and cell, vector and protein engineering expertise.

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Advancements in upstream CLD for enhanced biotherapeutics production

Drug Discovery World

Upstream cell line development has seen notable progress with recent advances in cell line engineering, particularly in enhancing gene of interest integration specificity, clone selection, and production efficiency. Next-generation engineering tools, such as gene editing have enabled knock-out cell lines with enhanced characteristics.

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Double knockout strategy could boost CAR-T in solid tumours

Drug Discovery World

The research team, including lead author David Mai, a Bioengineering graduate student in the School of Engineering and Applied Science, hypothesised that targeting the related, but independent Roquin-1 regulator at the same time could boost responses further. In some mice, it also led to overproduction of lymphocytes, causing toxicity.

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Five UK TechBio companies driving the AI revolution

Drug Discovery World

A BioIndustry Association (BIA) report lists the widespread adoption of artificial intelligence (AI) and machine learning (ML), the advancement of gene editing technologies, the power of engineering biology, and a growing interest from major pharmaceutical players as key trends driving the sector.