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Vertex tiptoes further into Duchenne research with Tevard deal

Bio Pharma Dive

An alliance with the startup brings Vertex an RNA-based medicine for the disease, adding to a portfolio that already includes a gene editing treatment.

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Moderna partners with Life Edit for mRNA gene editing therapies

Pharmaceutical Technology

Moderna has entered a strategic research and development partnership with ElevateBio’s Life Edit Therapeutics to discover and develop new in-vivo mRNA gene editing therapies. Under the deal, both companies will partner on the research and preclinical studies, which will be funded by Moderna.

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Delivering on the promise of gene editing

Drug Discovery World

As gene editing technologies like CRISPR progress toward clinical study, researchers must continue to advance new approaches and address inherent challenges, explains Jon Chesnut, PhD, Senior Director, Cell Biology R&D, Thermo Fisher Scientific. Early phase clinical trials for gene editing therapies.

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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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Discovery paves the way to ‘off-the-shelf’ cervical cancer therapy

Drug Discovery World

Researchers from Japan have used CRISPR gene editing to enhance the effectiveness of rejuvenated cytotoxic T lymphocytes (rejTs) for targeted immunotherapy. To overcome this issue, the team used CRISPR-Cas9 two-step ‘scarless’ gene editing on iPSCs derived from an HPV-specific cytotoxic T lymphocyte clone.

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This week in drug discovery (10-14 October)

Drug Discovery World

Meanwhile, Ochre Bio successfully raised $30 million to advance its pipeline of RNA therapies for liver disease, and researchers found that combining CRISPR gene editing with Spherical Nucleic Acids could expand the potential of the technology. . Partnership seeks to transform Cystic Fibrosis research .

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Johns Hopkins Researchers Identify CRISPR Dimmer

The Pharma Data

A naturally occurring system for tuning CRISPR-Cas9 expressing in bacteria, identified in a study published in Cell , could have implications for gene editing therapies as well. A CRISPR-Cas9 system has two components: the Cas9 guide RNA that directs the system to edit a particular gene, and the CRISPR “scissors” that make the cut.