Recommended Resources-2025

ISTH - Recommended Resources

National Guidelines and Regulatory Frameworks

US Resources

FDA Gene Therapy for Hemophilia Guidance for Industry »

MASAC Recommendations on Hemophilia Treatment Center Preparedness for Delivering Gene Therapy for Hemophilia »

Gene Therapy Assays: Regulatory Compliance with Unified Immunogenicity »

NIH Regulatory Knowledge Guide for Cell and Gene Therapies »

ASGCT Frameworks »

European Resources

Implementation Guidance (UKHCDO Gene Therapy Taskforce) »

German-Austrian-Swiss Recommendation for Gene Therapy »

Italian Guide (AICE Booklet) »

Gene Therapy Assays: Regulatory Compliance with Unified Immunogenicity »

Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B »

Canadian Resources

Canadian Regulatory Framework and Regulatory Requirements for Cell and Gene Therapy Products »

Global Resources

Chinese Guidelines 2025 »

The WFH Guidelines for the Management of Haemophilia »

Progress in advanced cellular and gene therapies in South Africa and barriers to patient access: A National Consortium paper on behalf of the BloodSA Cell and Gene Therapy working party »

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Supported through educational grants from BioMarin, CSL Behring, Pfizer Inc., Roche, and Spark Therapeutics

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