8:50 am Chair’s Opening Remarks

Delivering Your Therapeutic to the Area of Action

9:00 am Treating PKU by Lentiviral Vector Gene Delivery to Liver

  • David Pauza Chief Scientific Officer, American Gene Technologies


• Exploiting natural tropism of lentivirus vectors
• Permanent genetic modification suitable for infants
• New strategies to improve hepatocyte transduction

9:30 am Next-Generation Antibody-Guided Enzyme Replacement Therapy for Lysosomal Diseases


• Discuss design considerations for antibody-guided enzymes
• Share proof-of-concept data on the use of antibody-guided enzymes for the treatment of lysosomal diseases
• Discuss delivery methods for antibody-guided enzymes, including gene therapy

10:00 am
Speed Networking


This session is the ideal opportunity to get face-to-face time with many of the brightest minds working in the Antigen Specific Immune Tolerance field and establish meaningful business relationships to pursue for the rest of the conference

10:30 am
Morning Break & Refreshments

11:00 am Valanafusp-Alpha: The Development Path of a Brain Penetrating Molecular Enzyme Replacement Therapy for the Treatment of Neurological & Somatic Symptoms pf MPS I


• The trojan horse platform of antibody-enzyme fusion proteins to transport therapeutics across the blood-brain-barrier
• Discovery, validation and preclinical development of Valanafusp alpha, aka AGT- 181
• Phase II clinical proof of concept of Valanafusp alpha and its effect on pediatric patients with severe untransplanted MPS I
• Regulatory pathway and clinical endpoints discussed with FDA and EMA for a pivotal trial with Valanafusp alpha

11:30 am A Cell-Penetrating & Degradative Antibody-Enzyme Fusion (AEF) Platform for the Treatment of Lafora Disease & Other Aggregate-Based Genetic Disorders


• The Valerion delivery platform allows intracellular delivery of active proteins and enzymes to the cytoplasmic compartment of systemic muscle and brain tissues
• This platform allows delivery of functional enzymes capable of clearing toxic intracellular polyglucosan or protein aggregates
• Clearance of these aggregates in multiple animal models and in a Phase I/II clinical study in Pompe disease demonstrates novel metabolic and functional efficacy

12:00 pm
Lunch & Networking

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

1:00 pm MRNA Therapy for Inherited Metabolic Disorders


• mRNA and lipid nanoparticles science and technology
• Preclinical and non-clinical studies in inherited metabolic disorders
• Future and applications of mRNA

1:30 pm Development of Leriglitazone in Rare Neurodegenerative Diseases


• Development in X-ALD and Friedreich ataxia
• Translational models
• Clinical endpoints and biomarkers: challenges and opportunities

2:00 pm
Afternoon Break & Refreshments

2:30 pm Business Development in Rare Disease


• Evaluating Orphan Assets
• Evaluating drug candidates for partnering & investment
• Finding rare disease assets to match development goals
• Assessing asset suitability for deal-making

2:40 pm Transplantation for MPS I & ALD: Implications of Newborn Screening

  • Paul Orchard Professor Pediatric Blood & Marrow Transplantation, University of Minnesota


• What is the current status of allogeneic transplantation for MPS I and ALD?
• At the time of diagnosis, can we predict phenotypes? Why is this important?
• How does disease status at the time of transplantation influence outcomes?

3:10 pm

Panel Discussion: Tackling Ultra-Rare Inborn Errors of Metabolism: Addressing a Huge Unmet Medical Need


This panel discussion will set to map out the future for ultra-rare inherited metabolic therapeutics. Analyze current and future treatment options and explore how a patient-centric approach to drug development can be utilized through collaborations with advocacy groups. This panel will also address the role of industryacademia collaborations to access and augment novel therapeutic innovations in the field in order to bring new opportunities to ultra-rare metabolic disease patients

3:40 pm Chair’s Closing Remarks