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How to advance AAV-based gene therapies

Drug Discovery World

in Cell Biology and Biochemistry from University College London and was a post-doctoral fellow at Albert Einstein College of Medicine in New York City. Food & Drug Administration (2017) FDA approves novel gene therapy to treat patients with a rare form of inherited vision loss. She received a Ph.D. References 1: U.S.

Therapies 162
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Who are the AACR Scientific Achievement Award winners?

Drug Discovery World

He is being recognised for his fundamental discoveries dedicated to characterising the genetic drivers of human B-cell lymphomas and for uncovering new avenues for cancer prevention and therapy that have been routinely exploited clinically to diagnose and determine novel therapeutic strategies for B-cell lymphoma.

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Breakthrough drug candidates and cancer treatment innovations

Drug Target Review

As such, our team remains on track to complete our 5×5 strategy – laid out in October 2022 – by harnessing our technology and resources to continue efficiently growing Zymeworks’ pipeline of in-house and partnered candidates. Author Bio: Paul Moore, PhD Dr Moore joined Zymeworks in July 2022 as Chief Scientific Officer.

Treatment 100
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Optimising CRIPSR gene editing of hard-to-transfect cells

Drug Target Review

Starting from a single cell rather than bulk cells eliminates the need for post-transfection cell sorting, saving hands-on time and ensuring 100 percent monoclonality. This holds particular significance, as it is a prerequisite for FDA approval in biotechnology that any cell clones must originate from a single-cell progenitor.

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Optimising CRISPR gene editing of hard-to-transfect cells

Drug Target Review

Starting from a single cell rather than bulk cells eliminates the need for post-transfection cell sorting, saving hands-on time and ensuring 100 percent monoclonality. This holds particular significance, as it is a prerequisite for FDA approval in biotechnology that any cell clones must originate from a single-cell progenitor.

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Advanced 3D cell-based technologies

Drug Target Review

In spite of current success and possibility to be a successful cell technology model, the integration of organ-on-chips into drug development process needs more optimisation to be validated for FDA approval. From 3D cell culture to organs-on-chips. Trends in Cell Biology. 2010;328(5986):1662-8. Ingber DE.

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Codon Digest: Injected Gene Editors

Codon

An open-source FACS automation system for high-throughput cell biology. Read Eisai and Biogen — companies behind the controversial, FDA-approved drug for Alzheimer’s, called Aduhelm — have published three additional papers with “detailed analyses” from their phase 2 trials. Might be useful!

DNA 52