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Conference Briefings

Highlights from the NHF 15th Workshop on Novel Technologies and Gene Transfer for Hemophilia

Highlights from the NHF 15th Workshop on Novel Technologies and Gene Transfer for Hemophilia

Presented by: David Lillicrap, MD and Glenn F. Pierce, MD, PhD
September 13-14, 2019 • Washington, DC

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Introduction
Presented by Dr. Lillicrap

Challenges Associated with AAV as a Tool for Delivering Therapeutic Transgenes
Presented by Dr. Pierce

FDA Guidance for Hemophilia Gene Therapy Clinical Development
Presented by Dr. Pierce

Factor IX Gene Therapy Clinical Trials Update
Presented by Dr. Lillicrap


Gene Editing and Cell-Based Therapy for Hemophilia
Presented by Dr. Pierce



Looking Ahead with Optimism
Presented by Dr. Pierce

Factor VIII Gene Therapy Clinical Trials Update
Presented by Dr. Lillicrap


Next Generation Gene Therapies for Hemophilia
Presented by Dr. Lillicrap

Response to AAV Vector Delivery
Presented by Dr. Lillicrap


Extending Gene Therapy to Children with Hemophilia
Presented by Dr. Lillicrap

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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.

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