About Event

Welcome to the Digital Gene Therapy for Inherited Metabolic Disorders Summit

Gene Therapy for Inherited Metabolic Disorders is the only summit dedicated to overcoming gene delivery and drug development challenges for inborn errors of metabolism.

This lazer-focussed meeting will arm you with the scientific knowledge, latest data and learning points you will be able to apply in order to progress your own pipeline to the clinic faster.

Bringing together dedicated and experienced professionals across the metabolic space, this event addresses key themes, including:

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Vectors: Selection, design and delivery: Enhance your understanding of how delivery strategy, vector selection and vector design can all contribute to more reliable transgene delivery to target tissues and cells with 4D Molecular Therapeutics & University of Pennsylvania

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How can understanding of animal model system biology enable the identification and validation of novel modelling systems for inherited metabolic disorders? Understand the importance of combining knowledge of model system biology with disease pathophysiology when designing or choosing animal and cell-based models for inherited metabolic disorders with Rocket Pharmaceuticals & the Karolinska Institute

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Dosing and redosing: What does current work mean for the future of this complex topic? Hear from thought leaders on the latest work on dosing, immunological considerations and redosing as it pertains to gene therapy for inherited metabolic disorders with University of Florida

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How can we ensure safety when using viral, non-viral and gene editing approaches? Cover issues ranging from AAV immunogenicity and the safety issues with redosing to considerations when pioneering CRISPR medicines safety assessments with AstraZeneca, The Gene Editing Institute & University of Florida

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Overcoming the challenges in gene therapy clinical trial design for inherited metabolic disorders to fast track the journey to approval: Learn what part natural history studies have to play in this process. Consider how best to design and use your preclinical work to smooth the development of your gene therapy for inborn error candidates with The Clinical for Special Children & University of Pittsburgh