Develop More Clinically Efficacious, Durable & Safe Gene Therapies for Inherited Metabolic Disorders

Biopharma are beginning to realize the potential of gene therapies as a truly curative therapeutic for inherited metabolic disorders. However, despite promising early clinical data the journey through the clinic and beyond will not be without its challenges.

Addressing these challenges, the inaugural Gene Therapy for Inherited Metabolic Disorders Summit will put the spotlight on the understanding of topics from the physiopathology of disease through to the nuances of dosing and delivery methods and designing a trial with endpoints and outcome measures that are meaningful both clinically and to patients.

Join 60+ industry leaders across the metabolic disorders space to overcome the specific delivery and drug development challenges to accelerate the development of more clinically efficacious, durable and safe gene therapies for metabolic disorders.

Don’t miss this opportunity to join pioneers from AstraZeneca, Amicus Therapeutics and Rocket Pharmaceuticals on this journey to revolutionize the treatment of inherited metabolic disorders.

Expert Speaker Faculty

Hung Do

Chief Scientific Officer

Amicus Therapeutics

Jonathan Schwartz

Chief Medical Officer

Rocket Pharmaceuticals

Paolo Martini

Chief Scientific Officer Rare Diseases

Moderna Therapeutics

Roberto Nitsch

Associate Director of Clinical Pharmacology & Safety Sciences for CRISPR Medicines


Barry Byrne

Professor & Associate Chair of Department of Paediatrics

University of Florida

Charles Vite


University of Pennsylvania

Eric Kmiec


Gene Editing Institute

Gabriel Brooks

Vice President of Translational & Clinical Research

4D Molecular Therapeutics

Hear What Past Attendees Have To Say

“I really enjoyed engaging in conversations with leaders in the field of gene therapy. The passion to help those plagued by rare diseases was infectious.”

Sarepta Therapeutics

“This is the place to go to if you want to connect to experts in the field of gene therapy.”


“The congress was very well organized and the educational content and topics was of a very high quality. I left very inspired about what’s to come in gene therapy!”

Emotive Agency

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