- Global Pharma News & Resources
14-Sep-2021 - 16-Sep-2021

Gene Therapy for Metabolic Disorders

More companies and institutions than ever before are embarking on development of gene therapies in metabolic disease, and they are encountering unique challenges, from fine-tuning dosing to defining clinically relevant biomarkers. Gene Therapy for Metabolic Disorders will unite large pharma and innovative biotechs to collectively overcome translational drug development barriers to deliver on the transformative promise of gene therapy approaches for patients in need.

Incorporating in-depth insights into the leading metabolic disease programs, this event will ensure that you can optimize dosing to achieve the required enzyme levels without over-expression, define the necessary parameters for transitioning to first-in-human trials, and overcome unique challenges encountered in multi-organ systemic gene therapy delivery for complex metabolic indications.

Don’t get left behind. Join colleagues from Preclinical, Translational and Clinical departments of leading companies including AVROBIO, Freeline Therapeutics and LogicBio to optimize your gene therapy approach and revolutionize the treatment of metabolic diseases.