Day 1 Congress

  • What are the challenges and how they can be addressed?
  • Overcoming the issues of misdiagnosis
  • Challenges facing clinicians who care for affected individuals
  • Challenges to investigators regard the difficulty and expense of assembling large cohorts of affected individuals for study, and garnering funding for research

Moderator: Alison Schecter, Global Rare Diseases, External Opportunities, Sanofi Genzyme
Panelist: Radhika Tripuraneni, Vice President, Medical Affairs, Prothena Corporation Plc
Barry Ticho, Chief Medical Officer, Stoke Therapeutics, Inc.
Ray Takigiku, Co-Founder, President and CEO, Bexion Pharmaceuticals, Inc.
Sabrina Mogle, Co-Founder and CEO, RareMoon

  • In this talk we shall explore:
  • The key attributes of a successful orphan drug launch
  • Examples of a successful orphan drug launch

Anthony J Arleth, Managing Director, Pennside Partners

In this talk we shall explore:

  • Demonstration of clinical benefit and contribution to patient care
  • Regional differences and requirements to achieve Orphan Drug Designation.

Lori Shafner, VP, Global Development Team Leader
Mike Page, Executive Director, Global Regulatory Affairs , Alexion Pharmaceuticals

    • An Overview of FDA’s Expedited Programs for Orphan Drugs.
    • How have Orphan Drug Designations and Expedited Programs stimulated the Industry.
    • A look at the numbers.
    • Influence of designations on approval timelines.
    • What pace should be expected?
    • The Breakthrough Therapy Designation.
    • Preparing for the speed.
    • The benefits and the burdens.
    • Closing remarks

    Sabrina Mogle, Co-Founder and CEO, RareMoon

  • Improved clinical outcomes require more than just clinical development
  • Understanding the patient journey in rare diseases is critical to successful clinical development
  • Innovative communication strategies, patient journey insights and behavioural science techniques can all contribute to improved patient outcomes

Cara Blair, Client Services Director,
Louise Picken, Senior Services Director, Complete HealthVizion

  • Trends in Orphan Diseases partnering deals
  • Orphan diseases partnering agreement structure
  • At which stage of the drug development does in-licensing occur and what is the reason for this trend?
  • What criteria do partners look for when partnering for in and out-licensing?

Moderator: Brian Bronk, Head of External Innovation, Rare Diseases, Sanofi
Panelist:
Toby Freyman, Senior Director, Business Development, Shire
Michael A. Swit, Esq., FDA Legal Counsel, Law Offices of Michael A. Swit
Mike Kelliher, Vice President, Business Development, Horizon Pharma
Mahesh Kolar, Vice President, Strategy and Business Development , Rarity Health

  • Case study: RVT-802 for the treatment of Complete DiGeorge Anomaly (cDGA)
  • Overview of expedited regulatory pathways available for innovative rare disease therapeutics
  • Managing multi-stakeholder collaboration including academia, patient advocacy, government, and industry
  • Novel strategies to promote patient/market access

Alvin Shih, CEO, Enzyvant Therapeutics

  • Gene therapy is often most beneficial in pediatric patients; however, AAV vectors are non-replicating and hence transgene expression is expected to wane over time as patients grow
  • It would be desirable to be able to re-treat patients in life, but the formation of neutralizing antibodies against AAV gene therapy vectors prevents the ability to re-dose AAV
  • We have developed synthetic vaccine particles encapsulating rapamycin (SVP-Rapamycin) that have been shown to mitigate immunogenicity against a variety of biologic drugs by inducing immune tolerance to the biologic
  • We will present pre-clinical data demonstrating the ability of SVP-Rapamycin to inhibit the formation of anti-AAV antibodies and successfully enable repeat administration of gene therapy in animals and share data from an ongoing Phase 2 clinical trial of SVP- Rapamycin combined with a therapeutic enzyme

Kei Kishimoto, CSO, Selecta Biosciences

  • Screening strategies and logistics to reach patients
  • Biomarker development and implementation as a diagnostic
  • Understanding the epidemiology of the disease to streamline a diagnostic strategy

Justin Bingham, Vice President of Business Development, Centogene

  • Efficiencies in developing well-characterized molecules in novel targets in rare diseases
  • Speeding up the drug development process to rapidly deliver treatments to patients
  • Diversifying a pipeline of potential treatments

David Cory, President and CEO, Eiger Pharmaceuticals

  • High – throughput screening for drug repurposing
  • “Basket” trials of drugs targeting shared molecular etiologies
  • Gene therapy and gene editing
  • Platform approaches to clinical trials

Philip J. Brooks, Program Director, Division of Clinical Innovation , NCATS, NIH

  • Importance of disease insights to guide development
  • Role of increasing access to genetic testing to aid early and accurate diagnosis
  • How sponsored genetic testing programs can help patients and clinicians to position for long-term success

Katherine Stueland, Chief Commercial Officer , Invitae
Nan Doyle, Senior Manager – Diagnostics , Alnylam Pharmaceuticals

  • What innovative technologies in clinical trials on rare diseases are available to speed-up development?
  • Limitations and challenges of these technologies
  • Considerations for developing orphan drugs required to advance biomarker and endpoint use
    in clinical development

Tim Miller, Co-Founder, President and Chief Scientific Officer, Abeona Therapeutics Inc.

  • Patient recruitment and retention for a successful clinical trial in rare diseases
  • How can you prepare healthcare providers to know about the therapy and identify the right patients for your therapy?
  • How can developers make research goals and use of data to be transparent and to be informed about progress or roadblocks in order to build a relationship based on trust with patients?
  • What enhanced ongoing patient support beyond the clinic can be provided to encourage positive relationships between patients and healthcare providers?

Moderator: Christian Girard, Co-Founder & CEO, Overlord Pharmaceuticals
Panelist: Leslie Leahy, VP, Medical Sciences, Ovid Therapeutics
Luke Rosen, Patient Engagement, Ovid Therapeutics
Khrystal K. Davis, CEO and Patient Advocate, Zebra Leaf Publishing
Mahesh Kolar, Vice President, Strategy and Business Development , Rarity Health

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