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

8:15 am Virtual Networking

8:15 am Virtual Networking

8:15 am Virtual Networking

8:15 am Virtual Networking

8:15 am Virtual Networking

8:15 am Virtual Networking

8:15 am Virtual Networking

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

8:50 am Chair’s Opening Remarks

Optimizing Your Clinical Trial Design

Optimizing Your Clinical Trial Design

Optimizing Your Clinical Trial Design

Optimizing Your Clinical Trial Design

Optimizing Your Clinical Trial Design

Optimizing Your Clinical Trial Design

Optimizing Your Clinical Trial Design

Optimizing Your Clinical Trial Design

9:00 am Understanding the “Ins & Outs” of Patient Recruitment for Rare Metabolic Diseases

9:00 am Understanding the “Ins & Outs” of Patient Recruitment for Rare Metabolic Diseases

9:00 am Understanding the “Ins & Outs” of Patient Recruitment for Rare Metabolic Diseases

9:00 am Understanding the “Ins & Outs” of Patient Recruitment for Rare Metabolic Diseases

9:00 am Understanding the “Ins & Outs” of Patient Recruitment for Rare Metabolic Diseases

9:00 am Understanding the “Ins & Outs” of Patient Recruitment for Rare Metabolic Diseases

9:00 am Understanding the “Ins & Outs” of Patient Recruitment for Rare Metabolic Diseases

9:00 am Understanding the “Ins & Outs” of Patient Recruitment for Rare Metabolic Diseases

Synopsis

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

9:20 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

9:20 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

9:20 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

9:20 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

9:20 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

9:20 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

9:20 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

9:20 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

Synopsis

  • 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

Overcoming Barriers to Patient Access for Rare Populations

Overcoming Barriers to Patient Access for Rare Populations

Overcoming Barriers to Patient Access for Rare Populations

Overcoming Barriers to Patient Access for Rare Populations

Overcoming Barriers to Patient Access for Rare Populations

Overcoming Barriers to Patient Access for Rare Populations

Overcoming Barriers to Patient Access for Rare Populations

Overcoming Barriers to Patient Access for Rare Populations

9:50 am Live Q&A Session

  • Reena Kartha Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota
  • Kevin Fowler President, The Voice of the Patient Inc

9:50 am Live Q&A Session

  • Reena Kartha Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota
  • Kevin Fowler President, The Voice of the Patient Inc

9:50 am Live Q&A Session

  • Reena Kartha Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota
  • Kevin Fowler President, The Voice of the Patient Inc

9:50 am Live Q&A Session

  • Reena Kartha Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota
  • Kevin Fowler President, The Voice of the Patient Inc

9:50 am Live Q&A Session

  • Reena Kartha Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota
  • Kevin Fowler President, The Voice of the Patient Inc

9:50 am Live Q&A Session

  • Reena Kartha Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota
  • Kevin Fowler President, The Voice of the Patient Inc

Synopsis

• Have the speakers answer your questions

9:50 am Live Q&A Session

  • Reena Kartha Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota
  • Kevin Fowler President, The Voice of the Patient Inc

9:50 am Live Q&A Session

  • Reena Kartha Associate Director Translational Pharmacology, Center for Orphan Drug Research, University of Minnesota
  • Kevin Fowler President, The Voice of the Patient Inc

10:20 am Morning Break & Virtual Networking

10:20 am Morning Break & Virtual Networking

10:20 am Morning Break & Virtual Networking

10:20 am Morning Break & Virtual Networking

10:20 am Morning Break & Virtual Networking

10:20 am Morning Break & Virtual Networking

10:20 am Morning Break & Virtual Networking

Synopsis

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!

10:20 am Morning Break & Virtual Networking

11:30 am Panel Discussion: Tackling Ultra-Rare Inborn Errors of Metabolism: Addressing a Huge Unmet Medical Need

11:30 am Panel Discussion: Tackling Ultra-Rare Inborn Errors of Metabolism: Addressing a Huge Unmet Medical Need

11:30 am Panel Discussion: Tackling Ultra-Rare Inborn Errors of Metabolism: Addressing a Huge Unmet Medical Need

11:30 am Panel Discussion: Tackling Ultra-Rare Inborn Errors of Metabolism: Addressing a Huge Unmet Medical Need

11:30 am Panel Discussion: Tackling Ultra-Rare Inborn Errors of Metabolism: Addressing a Huge Unmet Medical Need

11:30 am Panel Discussion: Tackling Ultra-Rare Inborn Errors of Metabolism: Addressing a Huge Unmet Medical Need

11:30 am Panel Discussion: Tackling Ultra-Rare Inborn Errors of Metabolism: Addressing a Huge Unmet Medical Need

Synopsis

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

12:00 pm Patient Engagement Throughout the Drug Development Process in Genomic Medicines

12:00 pm Patient Engagement Throughout the Drug Development Process in Genomic Medicines

12:00 pm Patient Engagement Throughout the Drug Development Process in Genomic Medicines

12:00 pm Patient Engagement Throughout the Drug Development Process in Genomic Medicines

12:00 pm Patient Engagement Throughout the Drug Development Process in Genomic Medicines

12:00 pm Patient Engagement Throughout the Drug Development Process in Genomic Medicines

12:00 pm Patient Engagement Throughout the Drug Development Process in Genomic Medicines

Synopsis

• 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 Patient Engagement Throughout the Drug Development Process in Genomic Medicines

12:30 pm Lunch

12:30 pm Lunch

12:30 pm Lunch

12:30 pm Lunch

12:30 pm Lunch

12:30 pm Lunch

12:30 pm Lunch

12:30 pm Lunch

1:30 pm

Optimizing Patient Centric Drug Development Approaches

1:30 pm Effective Collaborations Between Industry and Patient Organizations

  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation

1:30 pm

Optimizing Patient Centric Drug Development Approaches

1:30 pm Effective Collaborations Between Industry and Patient Organizations

  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation

1:30 pm

Optimizing Patient Centric Drug Development Approaches

1:30 pm Effective Collaborations Between Industry and Patient Organizations

  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation

1:30 pm

Optimizing Patient Centric Drug Development Approaches

1:30 pm Effective Collaborations Between Industry and Patient Organizations

  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation

1:30 pm

Optimizing Patient Centric Drug Development Approaches

1:30 pm Effective Collaborations Between Industry and Patient Organizations

  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation

1:30 pm

Optimizing Patient Centric Drug Development Approaches

1:30 pm Effective Collaborations Between Industry and Patient Organizations

  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation

1:30 pm Effective Collaborations Between Industry and Patient Organizations

  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation

Synopsis

• 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

Optimizing Patient Centric Drug Development Approaches

1:30 pm Effective Collaborations Between Industry and Patient Organizations

  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation

1:30 pm

Optimizing Patient Centric Drug Development Approaches

2:00 pm Transplantation for MPS I & ALD: Implications of Newborn Screening

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

2:00 pm Transplantation for MPS I & ALD: Implications of Newborn Screening

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

2:00 pm Transplantation for MPS I & ALD: Implications of Newborn Screening

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

2:00 pm Transplantation for MPS I & ALD: Implications of Newborn Screening

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

2:00 pm Transplantation for MPS I & ALD: Implications of Newborn Screening

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

2:00 pm Transplantation for MPS I & ALD: Implications of Newborn Screening

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

Synopsis

• 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?

2:00 pm Transplantation for MPS I & ALD: Implications of Newborn Screening

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

2:00 pm Transplantation for MPS I & ALD: Implications of Newborn Screening

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

2:30 pm Live Q&A

  • Christeen Moburg Director Patient Advocacy, Sangamo Therapeutics
  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation
  • Paul Orchard Professor Pediatric Blood & Marrow Transplantation, University of Minnesota

2:30 pm Live Q&A

  • Christeen Moburg Director Patient Advocacy, Sangamo Therapeutics
  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation
  • Paul Orchard Professor Pediatric Blood & Marrow Transplantation, University of Minnesota

2:30 pm Live Q&A

  • Christeen Moburg Director Patient Advocacy, Sangamo Therapeutics
  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation
  • Paul Orchard Professor Pediatric Blood & Marrow Transplantation, University of Minnesota

2:30 pm Live Q&A

  • Christeen Moburg Director Patient Advocacy, Sangamo Therapeutics
  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation
  • Paul Orchard Professor Pediatric Blood & Marrow Transplantation, University of Minnesota

2:30 pm Live Q&A

  • Christeen Moburg Director Patient Advocacy, Sangamo Therapeutics
  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation
  • Paul Orchard Professor Pediatric Blood & Marrow Transplantation, University of Minnesota

2:30 pm Live Q&A

  • Christeen Moburg Director Patient Advocacy, Sangamo Therapeutics
  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation
  • Paul Orchard Professor Pediatric Blood & Marrow Transplantation, University of Minnesota

2:30 pm Live Q&A

  • Christeen Moburg Director Patient Advocacy, Sangamo Therapeutics
  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation
  • Paul Orchard Professor Pediatric Blood & Marrow Transplantation, University of Minnesota

2:30 pm Live Q&A

  • Christeen Moburg Director Patient Advocacy, Sangamo Therapeutics
  • Joslyn Crowe Executive Director, National Niemann Pick Disease Foundation
  • Paul Orchard Professor Pediatric Blood & Marrow Transplantation, University of Minnesota

Synopsis

• Have the speakers answer your questions

3:00 pm Chair’s Closing Remarks

3:00 pm Chair’s Closing Remarks

3:00 pm Chair’s Closing Remarks

3:00 pm Chair’s Closing Remarks

3:00 pm Chair’s Closing Remarks

3:00 pm Chair’s Closing Remarks

3:00 pm Chair’s Closing Remarks

3:00 pm Chair’s Closing Remarks