8:50 am Chair’s Opening Remarks

Reviewing the Clinical Landscape for Rare Metabolic Disease Therapeutics: Where Are We Now?

9:00 am 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

9:30 am Development of an Oral, Non-Systemically Acting Medicinal Product for the Treatment of Phenylketonuria (Pku) to Be Used as a Mono-Therapy


• The product is a nano formulation of phenylalanine-binding particles made of a synthetic polymer
• Animal data confirms the potential for the product to be used as a mono-therapy for all types of PKU
• The technology works as a platform for developing medicinal products for other similar rare, metabolic diseases such as Alkaptonuria (AKU) and Hereditary Tyrosinemia type-1 (HT1)

10:00 am A New Era in Cellular Medicine for Phenylketonuria


• Most phenylketonuria patients rely on a protein-restrictive diet to control their disease that is difficult to adhere to and does not always work
• Rubius Therapeutics is developing an investigational enzyme replacement therapy engineered inside a red blood cell called RTX-134
• RTX-134 is designed to overcome many of the challenges with current treatment options
• RTX-134 is being evaluated in Phase 1 clinical trial for adults with phenylketonuria

10:30 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

11:00 am Morning Break & Refreshments

11:30 am Reviewing the Gene Therapy Landscape for Inherited Metabolic Diseases

12:00 pm A Novel Enzyme Therapy For Patients With Homocystinuria


• An overview of homocystinuria pathology, epidemiology, unmet need, and cost burden
• OT-58’s impact on biochemical and clinical parameters in multiple mouse models of homocystinuria Interim data from Orphan
• Technologies ongoing natural history study and an overview of the ongoing U.S. Randomized Placebo Controlled Phase 1/2 clinical study

Delivering Your Therapeutic to the Area of Action

12:30 pm 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

1:00 pm 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

1:30 pm Lunch & Networking

2:30 pm 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

3:00 pm 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

3:30 pm Afternoon Refreshments & Networking

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

4:00 pm Development of Leriglitazone in Rare Neurodegenerative Diseases


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

4:30 pm Delivering on Hope for Patients with Serious Chronic Diseases


• Optimization of human allogeneic cells shielded by antifibrotic biomaterials as a new platform for the treatment of chronic diseases, including metabolic disorders
• Proof of concept results in animal models of lysosomal diseases
• Applicability of platform to metabolic disorders other than LSDs

5:00 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?

5:30 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 industry-academia collaborations to access and augment novel therapeutic
innovations in the field in order to bring new opportunities to ultra-rare metabolic disease patients

6:00 pm Chair’s Closing Remarks

6:15 pm Scientific Poster Session


After the formal presentations have finished, the learning and networking carries on. The Poster Session allows you to connect with your peers in a relaxed atmosphere and continue to forge new and existing relationships. During this session scientific posters will be presented on the latest advancements in the Antigen Specific Immune Tolerance field.