Inborn Errors of Metabolism Drug Development Summit 2020

The Inborn Errors of Metabolism Drug Development Summit 2020 is a conference focused on key patient access, regulatory, clinical and commercial aspects of gene therapy.

Topics

• A New Era in Cellular Medicine for Phenylketonuria
• Development of an Oral, Non-Systemically Acting Medicinal Product for the Treatment of Phenylketonuria (Pku) to Be Used as a Mono-Therapy
• Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases
• Treating PKU by Lentiviral Vector Gene Delivery to Liver
• Delivering on Hope for Patients with Serious Chronic Diseases
• Development of Leriglitazone in Rare Neurodegenerative Diseases
• Early Commercial Strategy for Rare Indications
• Patient Engagement in Rare Metabolic Disease Studies