Animal Testing and Protein Expression

A therapy candidate for fatal prion diseases turns off disease-causing gene

Broad Institute

JUNE 27, 2024

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. Credit: Neumann EN, Bertozzi TM, et al.

Disease

Disease Therapies Engineering Clinical Trials

Rapid delivery of toxicological material

Drug Target Review

SEPTEMBER 10, 2024

For example, advances in media and feeds have contributed to higher cell protein expression by optimising nutrient availability and reducing accumulation of waste products within a culture. Novel vector design and synthetic promoters have been designed to maximise productivity and enhance gene expression stability.

Drug Development

Drug Development Clinical Supply Production Protein Expression

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

FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Instead, this guidance highlights questions about species, animal model, and product selection for nonclinical programs as well as several aimed at helping to understand the purpose and importance of POC, toxicity, and biodistribution studies.

Therapies

Therapies FDA Production Disease

Drug Discovery Digest

A therapy candidate for fatal prion diseases turns off disease-causing gene

Rapid delivery of toxicological material

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

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