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8:20 am Chair’s Opening Remarks

ARTICULATING THE VALUE OF GENE THERAPIES TO OVERCOME MARKET ACCESS CHALLENGES

8:30 am The Evaluation of Cell and Gene Therapies

  • Nick Crabb Programme Director, Scientific Affairs, NICE

Synopsis

  • To outline the challenges of applying health technology assessment to cell and gene therapies
  • To consider the need for managed access arrangements
  • To provide case histories from NICE’s experience

9:00 am The Evidence Generation Continuum: Articulating the Value of Gene Therapies to Diverse Stakeholders

  • Marcus Droege Global Head, RWE & Evidence, AveXis, a Novartis Company

Synopsis

  • Map (non-clinical) data generation activities in support of emerging gene therapies
  • Illustrate how evidentiary needs change over time
  • Anticipate the evolving needs of global payors to enable reimbursement

9:30 am Demonstrating the Value of Gene Therapy Products Across Indications

  • Jim Thomson Senior Manager, Market Access & Gene Therapy, Pfizer

Synopsis

  • Negotiating novel models of reimbursement for gene therapy products
  • Assessing gene therapies as valuable treatments: what does value mean to the patient?
  • Cross-platform insights: what can be applied across a range of early stage gene therapy programs in different indications?

10:00 am Morning Refreshments & Networking

DEVELOPING EFFECTIVE CHARACTERIZATION & ANALYTICAL TOOLS

10:45 am Towards a 3rd Generation AAV Manufacturing Platform & In-Process Control

Synopsis

  • Losses due to shear, processing methods such as precipitation, freeze/thaw and tangential flow filtration (TFF) promote formation of stable associations between virus, DNA and proteins
  • A non-affinity process is outlined to manage formation of aggregates. Future development in progress towards extra low DNA
  • Rapid and sensitive analytics for in process control of AAV manufacturing are presented, with focus on HPLC and different in-line detectors. Qualitative and quantitative information can be obtained in near real-time to assist with process decision-making

NAVIGATING THE COMPLEX EUROPEAN REGULATORY ENVIRONMENT

11:15 am Incorporating the Patient Perspective in Clinical Development

Synopsis

  • Involving patients in every stage of gene therapy clinical development
  • Working with advocacy networks, patients and new technologies to shape trials and ensure they’re appropriate and relevant to the unique, indication-specific context
  • Investigating the utility of patient reported outcomes in gene therapy trials

11:45 am GS010 Gene Therapy in LHON Subjects: Results of Two Phase III Studies

12:15 pm European Regulatory Perspective on the Development of Gene Therapies for Rare Disorders

  • Rune Kjeken Scientific Director, Advanced Therapies, CAT, EMA

Synopsis

  • Gaining insights into the regulatory landscape in Europe
  • Understanding how regulatory pathways are becoming more clearly defined, and the key learning points on this steep learning curve
  • Learning how regulators can react effectively to new developments and technologies in the field

12:45 pm Lunch & Networking

1:45 pm Applicants & Regulators: Engaging in Effective Collaboration & Clear Dialogue

  • Pedro Campino Global Regulatory Lead, Gene Therapy, CSL Behring

Synopsis

  • Contrasting the regulatory environments in Europe and the US – what information is it important to communicate to regulators when working with them on gene therapy programs
  • What are the key considerations to take into account when working with Regulators?
  • Unique regulatory challenges encountered in gene therapy development, including CMC, safety assessments and long-term follow up

2:15 pm Panel Discussion: Defining a Harmonised, Globally-Applicable Regulatory Framework for Gene Therapies

Synopsis

  • How are organisations adapting to the evolving gene therapy regulatory landscape?
  • Defining the risk, benefit and value of gene therapies
  • Gaining clarity on acceptable, clinically-relevant endpoints
  • Working towards a single, global, unified position

2:45 pm Afternoon Refreshments & Networking

BEYOND AAV: EXPLORING THE REALITIES OF PIONEERING LENTIVIRAL GENE THERAPIES

3:15 pm Demonstrating the Potential of Haematopoietic Stem Cell Gene Therapies

  • Mahesh Kudari Program Leader, Clinical Development, Orchard Therapeutics

Synopsis

  • Insights into Orchard’s gene therapy work and the breadth of the portfolio
  • Understanding the potential of using viral vectors and haematopoietic stem cells
  • Case study: first-hand insights into the development of a gene therapy

3:45 pm A Path Towards Freedom From a Lifetime of Disease: Lentiviral Investigational Gene Therapies for Common & Rare Lysosomal Storage Disorders

Synopsis

  • Lentiviral investigational gene therapies for common and rare lysosomal storage disorders
  • Considerations for global lentiviral gene therapy program strategy and execution
  • Recap of Fabry FAB-201 clinical study

4:15 pm Chair’s Closing Remarks

4:30 pm Close of Conference