Human Gene Therapy for Hemophilia - Guidance for Industry

Titel:
Human Gene Therapy for Hemophilia - Guidance for Industry
Herkunft/Verlag:

FDA Office of Communication, Outreach and Development (OCOD), 10903 New Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM610801.pdf

Dokumentenart:
Guidance for Industry
Inhalt:
This guidance provides recommendations to sponsors developing human gene therapy (GT)1products for the treatment of hemophilia including clinical trial design and related development of coagulation factor VIII (hemophilia A) and IX (hemophilia B) activity assays, including how to address discrepancies in factor VIII and factor IX activity assays. This guidance also includes recommendations regarding preclinical considerations to support development of GT products for the treatment of hemophilia.

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