Agency IQ

APRIL 12, 2024

Draft guidance on potency assays for CGT products garners extensive stakeholder input Late last year, the FDA published a draft update to its 2011 guidance on potency assays for cell and gene therapy products, unveiling a major shift in approach to the issue.

Production 40

Production FDA Therapies Biochemical Assays 40

Broad Institute

JANUARY 14, 2025

When I received my genetic test report in 2011, the world had never heard of base editing. His lab had just developed base editing, a gene-editing approach that makes single-letter changes in DNA and can shut down protein production using strategies including installing a stop signal in the genetic code. Nature Medicine.

Disease 144

Disease DNA Protein Production Treatment 144

The Pharma Data

DECEMBER 3, 2020

An initial analysis of data from 17/22 patients who completed one of these assessments showed substantially decreased neurocognitive abilities compared to children of the same age level despite the use of multiple anti-epileptic therapies. In addition, a gap in adaptive functioning was noted using the Vineland Adaptive Behavior Scales.

Clinical Trials 52

Clinical Trials Protein Expression Pharmacokinetics Trials 52

Codon

AUGUST 10, 2023

Nature (2011). Link Gene Expression & Regulation →Genetic regulatory mechanisms in the synthesis of proteins , by Jacob F. & Link The PURE system for the cell-free synthesis of membrane proteins , by Kuruma Y. & Link The Elaboration of the Central Dogma. Scitable, by Nature Education. & Xie X.S.

DNA 98

DNA Cell Biology Molecular Biology Engineering 98

Codon

AUGUST 10, 2023

Nature (2011). Link Gene Expression & Regulation →Genetic regulatory mechanisms in the synthesis of proteins , by Jacob F. & Link The PURE system for the cell-free synthesis of membrane proteins , by Kuruma Y. & Link The Elaboration of the Central Dogma. Scitable, by Nature Education. & Xie X.S.

DNA 52

DNA Cell Biology Engineering Molecular Biology 52