Q56: Gene Therapy Persistence Testing

For gene therapy products, testing for the gene therapy may be completed post infusion. Testing for the persistence of the gene therapy includes the following:

• Allelic editing frequency
• Clonal expansion
• Gene therapy globin expression
• Homology directed DNA repair (HAD) frequency
• Indel mutations frequency
• Transgene expression
• Vector copy number (VCN)

Question 56: Were tests performed to detect persistence of gene therapy product infusion?

Indicate if testing for the persistence of the gene therapy product was completed during the reporting period. If Yes, complete the Gene Therapy Persistence (2103) Form.

Section Updates:

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