March 26-28, 2019 | Boston, MA
Register by Friday, March 22 to secure the best rate!

Morning Workshops

09.00 - 12.00

Overcoming Analytical Challenges Encountered Throughout the Development of Gene Therapies

As an increasing number of gene therapy products progress into the advanced stages of development, the role of analytical testing is becoming increasingly crucial. These tests are invaluable in clarifying the quality of products and mitigating against unexpected delays at every stage of this development journey.

Attendees will discuss:

  • The unique analytical challenges posed by the complexity of gene therapy products
  • Quality attributes that need to be closely monitored throughout gene therapy drug development, including potency, purity, safety, stability and efficacy
  • Next Generation Sequencing and the useful analytical information it the insights it can provide, including linking this to regulatory evaluations

Workshop Leader: Wei-Chiang Chen, Senior Scientist, Solid Biosciences

With Additional Insights From:

Sarah Sullivan, Scientist I, Biogen

Xiaohui Lu, Director, Ultragenyx

Yu Wang, Scientist II, Biogen

William Zolla, Research Associate, Analytical Development, Ultragenyx

09.00 - 12.00
WORKSHOP B | Bridging the Gap to the Clinic by Investigating Novel Preclinical Modelling Approaches

Developing robust and translatable preclinical models to recapitulate human disease states is a major challenge, especially for rare disorders with no existing models available. This workshop will discuss the creating of novel translatable animal models, as well as the insights that can be gleaned from these models that will support the translation of early promise into clinical success.

Attendees will discuss:

  • How to select appropriate preclinical models to support development in rare genetic disorders
  • The unique challenges encountered in investigating gene therapies preclinically and approaches to overcome these challenges
  • First-hand insights from

Workshop Leader: Christian Mueller, Associate Professor, UMass Medical School

With Additional Insights From:

Florie Borel, Associate Director, Preclinical Research, Apic Bio

09.00 - 12.00
WORKSHOP C | Ethical Issues in the Development, Testing and Translation of Innovative Gene Therapies

The successful development and subsequent FDA approval of commercial gene therapies has been met with exciting opportunities for treatment and prevention for previously untreatable genetic diseases and conditions. However, the advent and increasing availability of these therapies has also highlighted important ethical challenges that must be addressed. Attendees in this workshop will discuss several ethical challenges in the development, testing and dissemination of gene therapies, and how these challenges can be proactively identified and addressed early in the development pipeline, as well as throughout the translation process.

Attendees will discuss:

  • Examples of successful gene therapies, and classic cases in research ethics involving gene therapy
  • Ethical challenges in testing and development including clinical trial recruitment, ethical challenges in comparative effectiveness studies, informed consent for minors
  • Ethical challenges in translation, including cost, access, side effects and informed consent
  • Ethical challenges in social implications including issue related to access to other supports and treatments and impacts on disability identity
  • Discussion of potential tools and models for proactive consideration of ethical challenges related to gene therapies

Workshop Leader: Holly Tabor, Associate Professor, Stanford University Medical Center

Afternoon Workshops

13.00 - 16.00
WORKSHOP D | A Call to Arms for Standardization: Establishing Regenerative Medicine Standards

Regenerative medicines such as gene therapy products pose a host of unique challenges involving testing, protocol, product quality and performance criteria. The development of standards to which the entire community can adhere will improve gene therapy development by improving the quality and safety of therapeutics, as well as assisting the regulatory authorities in developing regulations. This workshop will gather a variety of perspectives to discuss the importance and application of standards to enhance gene therapy development.

Attendees will discuss:

  • Outlining the current landscape of standards Identifying gaps in the landscape and discussing approaches that can be taken to address this
  • Engaging the entire community about the importance of standardization
  • The value of standards in enhancing product development and regulatory predictability
  • Learning about the role of the Standards Coordinating
  •  Body (SCB) in facilitating the development of standards

Workshop Leaders:

Allison Getz, Senior Operations Program Manager, Standards Coordinating Body (SCB)

Dawn Kenke, Senior Technical Program Manager, Standards Coordinating Body (SCB)

Kelly Sauerwein, Technical Program Manager, Standards Coordinating Body (SCB)

13.00 - 16.00

How Industry Can Reshape the Development Paradigm of AAV Gene Therapies for Rare Diseases

The realization of commercial gene therapy promises to bring in a new era of medicine for the treatment of genetic disease. However, while the pathophysiology of many rare monogenic disorders are well understood and amenable to AAV gene therapy, the low prevalence of many disorders presents a nearly intractable economic obstacle in the development of therapies for such diseases.

There is a cogent need for novel pre-clinical and clinical development strategies fostered by cooperativity within the gene therapy space. Platform vector gene therapy, standardization, and novel clinical trial design represent just some of the areas industry can work together to build a new, lean model of AAV development.

Attendees will discuss:

  • How industry can develop standard platform vector gene therapies
  • Standardization in manufacturing and release criteria to enable a more streamlined approach to writing CMC sections of INDs
  • Novel clinical trial designs which address ultra-small patient populations which may not be amenable to traditional trial designs
  • Advantages of a pre-competitive consortium for gene therapy for ultra-rare diseases
  • Novel concepts for cost reduction to help simulate commercialization of gene therapies for ultra-rare disease

Workshop Leader: Harrison Brown, CSO, Odylia Therapeutics

13.00 - 16.00
WORKSHOP F | Overcoming the Challenges Involved in Implementing New Value-Based Payment Models

The ever-expanding pipeline of innovative yet costly therapeutics, like gene therapies, is causing existing payment approaches to be reconsidered so they can support better outcomes for patients as well as better value across the system. Value-based payments can link products to outcomes such as clinical measurements and patient-reported outcomes. This means that various stakeholders can share risk by having a direct stake in the real-world performance of the product. This workshop will focus on identifying barriers to new value-based payment models, as well as discussing the factors key in facilitating their implementation.

Attendees will discuss:

  • Identifying opportunities to improve patient care
    Understanding how barriers to the adoption of innovative new therapeutics can be reduced
  • Gaining insights into the practical realities of different payment models: what do they actually mean to the patients?
  • Learning about payment models that encourage rather than stifle innovation
  • Overcoming the operational challenges encountered in the implementation of these approaches

Workshop Leader: Marianne Hamilton Lopez, Research Director, Value-Based Payment Reform, Duke-Margolis Center for Health Policy