Digital Conference Agenda

9:00 am - 4:00 pm EDT      |      6:00 am - 1:00 pm PST

* Please note that the following agenda timings are Eastern Time.  

For Pacific Times, please download the full event guide here

8:15 am Online Registration and Coffee Networking Session


Kick start your conference with a virtual networking experience. Grab your morning cup of coffee, sit back and join the conversation

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 Morning Refreshments & Virtual Speed Networking


Grab a quick cup of tea or coffee from the comfort of your own kitchen and jump straight into your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!

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 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

12:00 pm Live Q&A Session


• Have the speakers answer your questions

12:30 pm Lunch

Clinical Endpoints & Biomarkers: Monitoring Disease & Therapeutic Outcomes

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 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?

2:30 pm Deciphering Transcriptional Control for Therapeutic Modulation of Metabolic Diseases

  • Alfica Sehgal Head of Translational Sciences, Camp4 Therapeutics


• Developing a better understanding of transcriptional control and harnessing this for disease modulation
• Which diseases can this approach be applied to?

3:00 pm Live Q&A Session

  • Paul Orchard Professor Pediatric Blood & Marrow Transplantation, University of Minnesota
  • Sonia Poli CSO, Minoryx
  • Alfica Sehgal Head of Translational Sciences, Camp4 Therapeutics


• Have the speakers answer your questions

3:30 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

3:50 pm Live Q&A Session


• Have the speakers answer your questions

4:00 pm Chair’s Closing Remarks

4:10 pm Virtual Networking & Happy Hour


The time of day we’ve all been waiting for! Grab a beverage of your choice from your home bar and use this final networking opportunity to follow up on connections made earlier in the day, ask that burning question to a speaker whose talk got you thinking and forge relations that could lead to your next collaboration

5:00 pm End of Day One