7:30 am Registration, Coffee & Networking

8:20 am Chair’s Opening Remarks

DEFINING THE CURRENT INDUSTRY LANDSCAPE FOR GENE THERAPIES IN THE RARE DISEASE SPACE

8:30 am Keynote: Setting Out the Gene Therapy Landscape: Where Are We Now?

  • Lyndsey Scull VP, Communications, Alliance for Regenerative Medicine (ARM)

Synopsis

  • Establishing the context and understanding how far the field has come in just a few years
  • Exploring the evolving clinical and investment landscapes – globally and in Europe
  • Examining the recent progress and near-term potential of the gene therapy space, with particular focus on rare disease
  • Estimating and measuring the impact this sector is poised to make on the healthcare system overall

9:00 am Achieving Clinical Progress while Establishing Mutually Beneficial Links with Academia

Synopsis

  • Sharing case studies from the clinical development of AAV-based gene therapies for the treatment of GM1 and GM2 gangliosidosis
  • Exploring strategies to interact with academic institutions in a more standardized and effective way
  • Looking ahead: what’s on the horizon for gene therapies and how will this future necessitate change in the field

9:30 am An Innovative Approach to a Gene Therapy Facility Start Up

  • Tony Khoury Vice President of Technical Services, Project Farma

Synopsis

  • Understanding the site selection process including a “make vs. buy” business case analysis
  • Utilising modular and single use technologies
  • Setting up the build for success including creating proper project work streams
  • Other considerations such as phased implementation, commissioning and qualification strategies, and change management

10:00 am Speed Networking & Morning Refreshments

ESTABLISHING ROBUST, SCALABLE AND COST-EFFECTIVE MANUFACTURING PROCESSES

11:15 am Manufacturing of a New Gene Therapy Product: Long Term Impact from Early Decision to Selection for Phase I to BLA / Commercial Strategy

Synopsis

  • Process development strategies from early academic discovery with potential GLP tox capabilities
  • Impact of early decision on process development academic to CDMO with phase 1 to commercial capabilities vs. integrated manufacturing
  • Make or buy or new alternatives for VCs funded biotech? Scenario analysis

11:45 am Key Factors for Successful AAV Vector Manufacturing

Synopsis

  • Critical starting materials that can make a difference
  • Process parameters affecting product quality
  • Improving the yield of downstream processes

12:15 pm Panel Discussion: Addressing Barriers to Adoption of Advanced Therapies

  • Ian McKay Innovation Lead, Advanced Therapies, Innovate UK

Synopsis

  • Developing an ecosystem to support the adoption of advanced therapies: establishing advanced therapy treatment centres
  • Understanding the unique barriers that need to be overcome to deliver advanced therapies in a clinical environment
  • Investigating efforts to expand viral manufacturing facilities and increase the range of products produced

12:45 pm Lunch & Networking

ENHANCING GENE THERAPY CLINICAL DEVELOPMENT IN THE RARE DISEASE SPACE

1:45 pm Achieving Success in Gene Therapy Clinical Development

Synopsis

  • Understanding the unique requirements for gene therapy products
  • Handling the additional local requirements for approvals when working with innovative gene therapy products
  • Ensuring the best alignment between the regulatory agency and payor

2:15 pm Overcoming Logistical Challenges Involved in Running Global Gene Therapy Clinical Trials

Synopsis

  • Investigating approaches to work with a small set of select clinical sites
  • Strategies to recruit patients worldwide
  • Exploring case studies discussing multiple late-stage gene therapy clinical trials

2:45 pm Patient-Focused Approaches to Clinical Development of Cell & Gene Therapies

Synopsis

  • Working with patient organisations in cell and gene therapy clinical development
  • Speeding up development by incorporating the patient perspective in study designs and key documentation
  • Challenges of clinical development in rare diseases

3:15 pm Afternoon Refreshments & Networking

PUTTING THE PATIENTS FIRST

3:45 pm 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

4:15 pm Panel Discussion: Keeping Patients at the Centre of Gene Therapy Development

Synopsis

  • Discussing how patients and patient organisations can work together with drug developers, regulators and clinicians to ensure gene therapy development is patient-centric
  • Addressing frequently asked questions and establishing educational initiative to inform patients about gene therapies
  • Educating start-ups and companies new to the space on effective and appropriate patient engagement
  • Overcoming misconceptions to give a real-world viewpoint on the realities of gene therapy development
  • Gain transferrable patient advocacy insights that can be applied across various rare disease indications

5:00 pm Chair’s Closing Remarks

5:15 pm End of Conference Day One