Interactive Workshops - Monday February 22, 2021

Morning Workshops

Workshop A:  9:00am - 12:00pm EST

Discussing the Value of Standards in the Gene Therapy Field & Sharing the World Health Organisation (WHO) International Gene Therapy Standards in Development


Establishing relevant and applicable standards to apply to gene therapy development is essential in maintaining safe and effective therapeutics. This also provides clarity and consistency for companies developing these therapeutics, especially as gene therapies progress into the late stages of development. Setting standards is especially challenging in the gene therapy space, where changes to manufacturing processes are frequent and the development is in orphan indications, limiting the possibilities for comparison with other approaches. Attend this workshop to learn about:
  • The development of international World Health Organization standards for lentiviral gene therapies
  • The current standards that exist for AAV therapeutics and the kinds of additional standards that are required
  • The importance of safety and potency standards
  • Data generated from the development of gene therapy standards, as well as research into the development of new assays and control testing of gene therapies
Workshop Leader: Jim Richardson, Senior Scientific Liaison, US Pharmacopeia

Workshop B: 9:00am - 12:00pm EST

Building Internal Gene Therapy Manufacturing Capacity


Robust, scalable and safe gene therapy manufacturing relies both on appropriate processes and facilities. With an increasing number of gene therapies progressing into late-stage development, the requirement for manufacturing capacity has brought facility design to the top of the agenda.

Attend this workshop to learn about:

  • Optimizing scalable manufacturing practices to scale up to commercial manufacturing
  • Understanding the regulatory requirements for gene therapy facilities and how internal builds and external contract facilities can ensure they meet these standards
  • Designing flexible manufacturing facilities to adapt to future demands
  • Discussing case studies from previous vector manufacturing facilities: what can be learnt to positively impact the future of the field?

Workshop Leader: Lance Weed, Consulting, Lance Weed Consulting

Workshop C: 9:00am - 12:00pm EST

Meaningful Clinical Endpoints in Rare Disorder Trials


Establishing clear, meaningful and rigorous clinical endpoints is essential in any clinical trial. The variability across patient populations, small trial numbers, and limited data on natural histories
of rare diseases makes identifying and validating endpoints in rare disorder trials difficult. With more gene therapies in the clinic than ever before, it is important to identify clinical endpoints in a robust and
worthwhile way.

Attend this workshop to learn about:

  • Tailoring clinical endpoints where classical endpoints may not be suitable
  • Acknowledging and assigning surrogate endpoints
  • Identifying and validating meaningful endpoints for the patient
  • Maximising outcome endpoints with the fewest number of patients
  • Setting a precedent for a primary endpoint

Workshop Leaders: Larissa Lapteva, Associate Director, OTAT, CBER FDA
                             Dawn Phillips, Director, Outcomes Research, REGENXBIO

Workshop D: 9:00am - 12:00pm EST

Evaluating the Realities of 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 workshop will focus on the real-world applications of value-based payment models and how these approaches can best be implemented in the near future.

Attend this workshop to learn about:

  • The implementation of a framework to better define when outcomes-based approaches are most appropriate
  • Understanding how barriers to the adoption of innovative new therapeutics can be reduced
  • Investigating the legal and regulatory challenges specific to the gene therapy space
  • Gaining insights into the practical realities of different payment models: what do they mean to the patients?
  • What would an outcomes-based model look like within Medicaid at the state level?
  • How can mechanisms for outcomes-based models at Medicare be developed within current regulatory capabilities?

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

Afternoon Workshops

Workshop E: 1:00pm - 4:00pm EST

Unifying Process & Analytical Development to Establish Robust, Scalable Gene Therapy Manufacturing Processes


Establishing scalable manufacturing processes from early in development has a lot of advantages, but is complex, requiring the integration of analytical, process development and regulatory expertise. It is also a balancing act, requiring thorough analysis of development and clinical timelines, as well as the constantly evolving regulatory landscape, to ensure that processes are established in a timely and effective manner. This workshop will examine this topic, sharing insights into the establishment of scalable gene therapy processes that will pre-emptively overcome challenges in future scale-up to commercialization.

Attend this workshop to learn about:

  • How analytical development can function in parallel with process development to shorten timelines and decrease costs
  • Approaches to stay ahead of regulatory guidances in an ever-evolving regulatory environment
  • Examining case studies detailing process development and analytical challenges encountered in the development of AAV-based gene therapy products

Workshop Leader: Dave Knop, Executive Director, Process Development, AGTC

Workshop F: 1:00pm - 4:00pm EST

Enhancing the Outsourcing Process: How to Select & Manage Contract Manufacturing Services & the Broader Contract Services Ecosystem Effectively


The days of small-scale academic work in the gene therapy field are morphing into the early stages of the commercial era. For this reason, facilitating successful outsourcing relationships with contract manufacturers, testing labs and logistics companies becomes more essential than ever before, in order to support large-scale commercial manufacturing on a global scale.

Attend this workshop to learn about:

  • What constitutes a good CMO? Investigating the criteria you should be using to adequately assess potential partners to outsource projects to in the gene therapy space
  • The broader contract infrastructure – understand how to improve interactions with CMOs, CROs, contract testing firms and contract shipping and transportation companies
  • Key factors impacting the decision to develop in-house capabilities or outsource services to third parties
  • Highlighting the importance of quality agreements from both parties in establishing successful collaborations

Workshop Leader: Scott Burger, Principal, Advanced Cell & Gene Therapy

Workshop G: 1:00pm - 4:00pm EST

Utilizing Pre-Clinical Data to Overcome the Translational Gap to the Clinic


Pre-clinical work remains the foundation of creating a successful gene therapy. Without robust, accurate, high quality data from pre-clinical studies, it is difficult to progress to the clinic with a likely candidate for a successful therapy. A lot can be learned from pre-clinical data, and maximising the output from those studies is key to paving the way to the clinic.

Attend this workshop to learn about:

  • Establishing efficacy in humans based on animal models by choosing the right
    preclinical readouts
  • Getting the dose right. How best to scale dose from animal models to human patients
  • Modeling the route of administration and biodistribution challenges in preclinical models
  • Facing immunogenicity concerns, use of immunosuppression, and the possibility of redosing in animal models
  • Safety and toxicity testing in animal models. What’s the right pre-clinical safety species?
  • Instituting good laboratory practices: The importance of quality control in reporting animal data

Workshop Leader: Allison Bradbury, Principal Investigator, Nationwide Children’s Hospital

Workshop H: 1:00pm - 4:00pm EST

Post-Approval Commercialization: Understanding the Realities of the Post-Approval Landscape


2018 was the first year that there were more orphan drugs approved than non-orphan drugs by the FDA. A revolution is happening in the biopharmaceutical business model: it’s shifted from blockbuster drugs to drugs for smaller and smaller patient populations. This causes many constantly evolving challenges to the policy and payer communities, many of which are very specific to certain indications, locations and patient circumstances. With the situation only set to increase in complexity given the ever-expanding pipeline of gene therapies, discussions around the emerging issues facing the space are essential.

Attend this workshop to learn about:

  • The changes that need to be made to adjust to the new realities of biopharmaceutical pipelines
  • Establishing benchmarks for value and pricing decisions in a variety of therapeutic areas
  • Discussing how appropriate the QALY approach is to assess the costeffectiveness of orphan drugs
  • What are the key aspects that need to be considered when pricing gene therapies?
  • Investigating approaches to include a wide variety of societal factors from patients, physicians and caregivers in determining the value of gene therapy products.

Workshop Leader: William Smith, Visiting Fellow On Life Sciences, Pioneer Institute