Contract Manufacturing FDA Approval Gene Therapy Genetic Disease 
XTalks
MAY 17, 2023
The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.
Pharmaceutical Technology
JULY 29, 2022
In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression.
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