Broad Institute

JUNE 27, 2024

Science (2024) Related content New gene delivery vehicle shows promise for human brain gene therapy My Quest to Cure Prion Disease — Before It’s Too Late | Sonia Vallabh | TED Prion diseases lead to rapid neurodegeneration and death and are caused by misshapen versions of the prion protein in the brain. “As

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FDA Law Blog: Drug Discovery

DECEMBER 6, 2024

Valentine On November 19, 2024, FDA released a draft guidance titled Frequently Asked Questions Developing Potential Cellular and Gene Therapy Products. The draft guidance enumerates key considerations for selecting an animal species for both pharmacology and toxicology studies.

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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.

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