Remove Animal Testing Remove Packaging Remove Protein Expression
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A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

By Greta Friar, Whitehead Institute June 27, 2024 Images of a mouse brain show the effect of a technology called CHARM in turning off the expression of a gene in the brain. The first problem they had to tackle was the editor’s size, because the editor needs to be small enough to be packaged and delivered to specific cells in the body.

Disease 142
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Rapid delivery of toxicological material

Drug Target Review

However, developing an accelerated IND application requires that good laboratory practice (GLP) toxicological data must be generated, collected, interpreted and integrated in the IND-enabling data package. Novel vector design and synthetic promoters have been designed to maximise productivity and enhance gene expression stability.

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FDA Issues “Cliffs Notes”-style Guidance on Cell and Gene Therapy; What Questions Did They Answer? (Part 1)

FDA Law Blog: Drug Discovery

The draft guidance recommends that no more than 15 questions are included in the briefing package. CBER will not commit to reviewing packages greater than 250 pages. Where vector presence is detected, transgene mRNA and/or protein expression levels should also be measured.