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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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Casting aside CRISPR scissors and making a point with base editors

pharmaphorum

Dr Jennifer Harbottle, senior scientist in the R&D Base Editing team of PerkinElmer’s Horizon Discovery business, looks at progress made in the realms of biotechnology and next-generation diagnostics, vaccines and therapeutics, including the application of CRISPR-Cas9 gene editing in developing and refining cell therapies.

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Regulatory Trends in Cell and Gene Therapies

Advarra

Bottlenecks in the manufacturing process, supply chain issues such as accessibility of good manufacturing practice (GMP)-grade reagents, and shortages of qualified scientists and engineers have caused many therapies to fail at critical stages of the clinical development pipeline. But review issues are not the only problems.

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CRISPR breakthroughs: New solutions for common diseases

Drug Discovery World

Incorporating in vivo pooled CRISPR screens has allowed for the interrogation of thousands of genetic perturbations in cells, leading to the determination of the involvement of molecules (PTPN2, ADAR1, or DHX37) in immune checkpoint inhibitor (ICI) responsiveness and T cell activation.

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