Digital Conference Agenda
Tuesday August 18

8:30 am - 7:00 pm EDT      |      5:30am - 4:00 pm PDT

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

For Pacific Times, please download the full event guide here

8:30 am Online Networking

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own home office 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!

8:45 am Chair’s Opening Remarks

The Approach to Patients with Bleeding Disorders

8:50 am A Patient-Centric Approach to Drug Development Across the Lifecycle

  • Eileen Sawyer Vice President, Global Medical Affairs, uniQure, Inc

Synopsis

• Incorporating patient perspectives and how to involve patients in gene therapy drug development
• At what stage of the program lifecycle should patients be involved in order to meet their needs and to optimize trial conduct
• How to provide a compelling value proposition for multiple stakeholders

9:20 am Discussing the Key Challenges Associated with Treating Pediatric Patients with Hemophilia

Synopsis

• How can administrative procedures be improved to reduce the trauma children have to go through when receiving regular treatments?
• What is the potential of gene therapy treating children when there is a high turnover of cells in the liver?
• Discussing the ethical issues parents face when choosing a treatment plan for their child

9:40 am A Hitchhiker’s Guide to Innovative Lab Solutions from a Coagulation- Focused Lab/CRO

  • Mike Ero Founder & CEO, Machaon Diagnostics Inc.

Synopsis

  • Hemophilia lab testing solutions: a 360 degree approach
    • Real time and database driven subject recruitment using existing Machaon patient
    databases
    • Eliminating barriers to testing and access in the time of COVID-19

10:10 am Live Presenter Q&A

  • Eileen Sawyer Vice President, Global Medical Affairs, uniQure, Inc
  • Angela Weyand Assistant Professor, University of Michigan
  • Mike Ero Founder & CEO, Machaon Diagnostics Inc.

10:40 am Online Networking Break

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own home office 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!

11:15 am Current Treatment Options Available for the Inhibitor Population

  • David Wraith Institute Director and Professor of Immunology / Chief Scientific Officer, University of Birmingham / Apitope

Synopsis

• Details to be confirmed

11:35 am Challenges Facing Providers and Women with Hemophilia

Synopsis

• To describe the evolving nomenclature
• To characterize the challenges in current diagnostic testing and discrepancy in levels and clinical severity
• To provide an overview of evolving management strategies

11:55 am ATHN Rising: The Evolution of Our Clinical Research Effort

  • Michael Recht Chief Science Officer, American Thrombosis and Hemostasis Network (ATHN)

Synopsis

• Details to be confirmed

12:25 pm Live Presenter Q&A

  • David Wraith Institute Director and Professor of Immunology / Chief Scientific Officer, University of Birmingham / Apitope
  • Robert Sidonio Assistant Professor, Emory University
  • Michael Recht Chief Science Officer, American Thrombosis and Hemostasis Network (ATHN)

1:00 pm Online Networking Break

Synopsis

Grab a quick bite to eat from the comfort of your own home office 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!

Addressing the Challenges of Extended Half-life Therapies to Advance Drug Development

1:45 pm A Novel Long-Term Treatment for Hemophilia A Using Shielded Living Therapeutics

  • Devyn Smith Chief Operating Officer, Sigilon Therapeutics

Synopsis

• Shielded living therapeutics utilize a novel cell platform combined with a novel biomaterial platform to potentially provide long lasting therapy for patients
• Pre-clinical data demonstrates both safety as well as efficacy in animal models
• Therapeutic is poised to enter the clinic this year

2:05 pm An Approach Towards Safe and Effective Extended 14 and 21-Day Dosing with rFIX-FP in Hemophilia B

  • Brahm Goldstein Executive Director & Global Therapeutic Area Head, Hematology, Clinical Research & Development, CSL Behring

Synopsis

• Extended dosing with rFIX-FP should be based upon an individual’s pharmacokinetic and clinical response- Allowing an adequate observation period during an extended dosing trial is key to establish safety and effectiveness
• Following a clear and strategic dosing strategy, safe and effective extended dosing in hemophilia B patients is possible

2:25 pm Live Presenter Q&A

  • Devyn Smith Chief Operating Officer, Sigilon Therapeutics
  • Brahm Goldstein Executive Director & Global Therapeutic Area Head, Hematology, Clinical Research & Development, CSL Behring

2:45 pm Determining Effective Diagnostics and Treatment for Patients with Von Willebrand Disease

  • Jonathan Roberts Associate Director - Medical & Research & Assistant Professor of Pediatrics, Bleeding & Clotting Disorders Institute

Synopsis

• Improving testing methods to identify phenotypic variants of VBW
• Could gene therapy provide a long-lasting alternative to patients?
• What novel drugs are being developed to improve patient care?

3:15 pm Online Networking Break

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own home office 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!

Understanding the Potential of Gene Therapy in the Treatment of Hemophilia

3:30 pm Gene Therapy for Hemophilia: Learnings from Phase 1/2 studies

  • John Murphy Vice President, Discovery Biology, Rare Disease Research Unit, Pfizer

Synopsis

• AAV mediated gene delivery of FIX and FVIII to treat HemB and HemB results in sustained (up to 4 year) therapeutic correction of bleeding with a good safety profile
• Assays to monitor factor activity following treatment give variable results and the choice of relevant assay is a topic of interest
• While all patients show a significant cessation of bleeding at doses chosen for phase 3 development, there is patient to patient variability in level of expression. Determinants of this variability are currently unknown

3:50 pm Clinical Development of Advanced Gene Therapies for Hemophilia

Synopsis

• Over the next 5-10 years the management and treatment of hemophilia will experience a revolution. A single intervention of gene therapy will become the treatment of choice for many patients
• Standard gene therapy can achieve a permanent and successful amelioration of the bleeding episodes in adults with hemophilia, eliminating the need for prophylactic replacement therapy
• However, the pediatric population requires gene editing to fully integrate the replacement gene into the cell. This approach maintains the production of the missing coagulation factor despite rapid turnover of liver cells

4:10 pm Adeno-Associated Virus Vectors for Hemophilia Gene Therapy

  • Robert Kotin Adjunct Professor, University of Massachusetts

Synopsis

• rAAV manufacturing capacity for clinical and nonclinical research
• Vector quality and research outcomes
• Clinical benefit and persistence and safety

4:30 pm Live Presenter Q&A

  • John Murphy Vice President, Discovery Biology, Rare Disease Research Unit, Pfizer
  • Oscar Segurado Chief Medical Officer, ASC Therapeutics
  • Robert Kotin Adjunct Professor, University of Massachusetts

5:00 pm Online Networking Break

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own home office 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!

Addressing the Patient/Payer Paradigm

5:15 pm Discussing the Relationship Between Patients & Payers to Treat the Unmet Clinical Need

Synopsis

• Clinical recommendations for therapy
• Site preparation for administration of therapy
• Follow up care and treatment
• Payer interaction: reimbursement and effective management of cost and outcomes

5:45 pm Chair’s Closing Remarks & End of Conference