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 Virtual Networking

8:50 am Chair’s Opening Remarks

Optimizing Your Clinical Trial Design

9:00 am Phase 2-3 Gene Therapy Trial Using Adeno-Associated Virus Vector for Patients With Mucopolysaccharidosis Type Iiia


• A gene therapy approach for MPSIIIA is promising when delivered directly to the brain enabling transduction of neurons, which in turn can lead to sufficient levels of active enzyme to cross-correct neighboring cells.
• AAVance is a Phase 2-3, single-arm, international study of AAV serotype rh.10 carrying the human SGSH (AAVrh.10-SGSH) for the treatment of MPS IIIA (NCT03612869).
• Description of the natural history data to serve as an external control group and status of the Phase 2-3 study will be presented

Overcoming Barriers to Patient Access for Rare Populations

9:30 am Industry-Academia Collaborations for Accelerating Therapeutic Innovations in Rare IEM Disorders

  • Reena Kartha Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota


• Importance of industry-academia collaborations to address an unmet need in ultra-rare IEM disorders
• Discuss potential avenues for collaboration with examples
• Provide overview on how patient advocacy groups can initiate such interactions

10:00 am Live Q&A Session

  • Sophie Olivier CSO, Lysogene
  • Reena Kartha Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota


• Have the speakers answer your questions

10:30 am Morning Break & Virtual 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 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

11:30 am Patient Engagement Throughout the Drug Development Process in Genomic Medicines


• Emphasizing the importance of early engagement for clinical trial recruitment
• How to design a compelling engagement process in order to maximize patient trial retention
• A case study of patient engagement for rare metabolic disease clinical trials

12:00 pm Lunch

12:59 pm

Optimizing Patient Centric Drug Development Approaches

1:00 pm Effective Collaborations Between Industry and Patient Organizations

  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation


• Highlighting the importance of collaboration between industry and patient organizations
• What makes collaboration effective?
• How can patient organizations be of use to the industry?

1:30 pm Understanding the “Ins & Outs” of Patient Recruitment for Rare Metabolic Diseases


• The importance of engaging of patients
• How to engage with patients across the development timeline

2:00 pm Live Q&A


• Have the speakers answer your questions

2:30 pm Round Table Discussion: Addressing the Halt in Drug Development Process Within the Inherited Metabolic Disease Space Due to COVID-19


This round table discussion will discuss how COVID-19 has affected the progress of drug development within the inherited metabolic disease space. A practical and interactive session where attendees can crowd source solutions and share opinions this roundtable will aim to kickstart the conversation around trying to pick up momentum again for drug development in the inherited metabolic disease space.

3:00 pm Chair’s Closing Remarks