Conference Day Two: Thursday, March 28

7:30 am Registration, Coffee & Breakfast Briefings

8:20 am Chair’s Opening Remarks

PROGRESSING THE PRICING, REIMBURSEMENT & MARKET ACCESS LANDSCAPE

8:30 am Federal-Led Models for Lowering Costs & Improving Access to Gene Therapies (Virtual)

  • Liz Fowler Director, CMS Innovation Center, Centers for Medicare & Medicaid Services

Synopsis

  • Exploring key goals for the Center for Medicare and Medicaid Innovation’s (CMMI) ‘Cell & Gene Therapy Access’ model
  • How are the CMMI approaching quality measurement for gene therapies, including patient-reported outcomes?
  • Addressing the short- and long- term implications of these two models for gene therapy drug developers
  • Considerations for what reimbursement pathway for gene therapies actually looks like

9:00 am Highlighting the Many Innovative Payment Tools to Enable Market Access

Synopsis

  • Exploring the wide of variety of new payment models available 
  • Considerations for selecting a model for your unique condition and therapeutic
  • Planning to overcome the legal and implementation barriers

9:30 am Outlining Success Stories in Value Demonstration to Private Insurers

  • Pat Furlong Founding President & Chief Executive Officer, Parent Project Muscular Dystrophy

Synopsis

  • The Duchenne Muscular Dystrophy Story: Driving forward access of an approved gene therapy by working with patients and insurers
  • Leveraging publications from healthcare professionals around value-based assessments and the therapeutic need
  • Addressing the need for consistent messaging between stakeholders in one disease community

10:00 am Panel Discussion: Gene Therapy Health Plan Coverage & Reimbursement Challenges for Private Payers

Synopsis

  • Understanding payer concerns around gene therapy coverage: why are some therapies deemed too high-risk to cover?
  • Comparing current methods payers are introducing to help make value-based contracts work
  • Exploring novel financing options to ensure these high-cost speciality drugs can be made accessible to those in need

10:30 am Morning Refreshments & Networking

MANUFACTURING

Benchmarking Innovation in Manufacturing

11:15 am Navigating Complexities of LV Vector from Clinical to Commercial Production: Lesson Learnings

  • Monika Pema Snr Manager Process Validation, AGC Biologics

Synopsis

  • Learn about the journey from clinical manufacturing to process validation
  • Importance of characterization and supportive studies through a case study

11:45 am HEK293 Suspension Based Production as a Scalable Platform for AAV Manufacturing

Synopsis

  • Improvements in HEK293 productivity over the years
  • Major cost drivers when scaling up to larger scales
  • Opportunities for process intensification and reduced costs

12:15 pm Efficient Viral Vector Manufacturing with Microcarriers

  • Trent Lyman Director Process Development, Matica Biotechnology

Synopsis

  • Understanding the viral vector manufacturing challenges often faced by drug developers
  • Advantages of using microcarriers
  • Upstream and downstream considerations for microcarriers cell culture system

CLINICAL

Ensuring Safety in Clinical Trial Design & Mitigating the Immune Response

  • Andy Kinley Vice President - Innovation & Clinical Science, Precision for Medicine

11:15 am Monitoring Safety & Efficacy of Rare Gene Therapies During Clinical Development & in the Commercial Setting

Synopsis

  • Reviewing the potential direct and indirect risks of genetic therapies
  • Implementing safety monitoring before and after approval of bluebird’s gene therapies
  • Long-term follow-up of patients receiving genetic therapies now and in the future

11:45 am IgG Cleaving Enzymes as a Potential Solution to the NAb Challenge

Synopsis

  • An overview of IgG cleaving enzymes: their MOA and translatability for use in conjunction with gene therapies
  • Discussing clinical data from non-gene therapy indications
  • Considerations for treatment timing and dosing of gene therapy post-administration of enzyme

12:15 pm Leveraging the Latest Understanding of Viral Vector Immunogenicity to Better Inform Clinical Strategy

  • Soumi Gupta Executive Director, Head of Clinical Immunology, BioMarin Pharmaceutical

Synopsis

  • Highlighting the latest understanding of viral vector immunogenicity seen in the clinical setting
  • Outlining new trends and emerging standards in immune mitigation strategies
  • Guidance for clinical assay selection to monitor the humoral and cellular immune response to a gene therapy

REGULATORY

Mapping Out Key Regulatory Touchpoints

11:15 am Maximizing the Benefits of a Type D Meeting

  • Darshna Patel Vice President - Regulatory Affairs, Neurocrine Biosciences

Synopsis

  • Understanding the scope, associated timelines, and practicalities of FDA Type D meetings and any follow-up opportunities
  • Reflections from engaging in a Type D meeting and advice for others seeking to engage
  • How do these meetings fit into an overall development plan?

11:45 am Designing & Implementing a Robust Late-Clinical Stage Regulatory Action Plan

  • Jim Wang Vice President - Regulatory Affairs & Genetic Medicines, Regeneron

Synopsis

  • Reflecting on lessons learned preparing for and undertaking a BLA/MAA filing
  • Top tips for data demonstration earlier in clinical development to support a marketing application
  • Facilitating smooth translation of your therapy into a commercial-ready product

12:15 pm Assessing In-Market Performance: Lessons Learned from an Approved Gene Therapy

  • Michael Ruggiero Senior Vice President, Global Policy, Advocacy And Government Affairs, CSL Behring

Synopsis

  • Presenting a case study on an in-market gene therapy: challenges and strategies in reaching the target patient population
  • Lessons learned in improving gene therapy access to patients in a timely manner
  • In the context of more gene therapies due to hit the market, how can industry ensure these are covered by insurers?

12:45 pm Lunch & Networking

Negotiating Manufacturing Challenges Under Compressed Timelines

1:45 pm The Evolution of Vector Production – Is Suspension Really the Solution?

  • Lucia Fernandez Director - Process Development & Manufacturing, Vinta Bio

Synopsis

  • Assessing adherent and suspension process impact on critical quality attributes
  • Leveraging innovation to drive increased performance of adherent process
  • Economic evaluation of adherent and suspension processes

2:15 pm Panel Discussion: Establishing a Commercial-Ready CMC Checklist for Expedited Pathways

  • Mi Jin Senior Director, Head of Downstream & Drug Product Development, Spark Therapeutics
  • Khandan Baradaran VP Regulatory CMC, Ultragenyx Pharmaceutical Inc
  • Tayler shebly senior director cmc regulatory affairs, Akouos Inc.

Synopsis

  • Defining key sticking points to ensuring CMC and clinical development are aligned when a programme is under an expedited pathway
  • Considerations for CMC-focused meetings with the regulators when moving through the clinic
  • Laying out a commercial manufacturing plan upon receiving an expedited pathway designation

Clinical Endpoints for Rare Disease Studies

  • Andy Kinley Vice President - Innovation & Clinical Science, Precision for Medicine

1:45 pm Outlining How to Establish & Run an Effective Natural History Study

Synopsis

  • How to effectively incorporate multiple stakeholder perspectives when planning and designing natural history data collection efforts
  • Challenges faced and solutions implemented in running a natural history study
  • Top tips for maximizing the use of natural history data in clinical trial design and outcome assessments

2:15 pm Development of Primary Endpoints for Rare Gene Therapy Clinical Studies: The Lovo-Cel Case Study

  • Anjulika Chawla Senior Director - Clinical Research Medical, bluebird bio

Synopsis

  • Reviewing the selection of endpoints for lovo-cel clinical studies
  • Exploring the background and impact of changes made to endpoints during clinical development
  • Future direction of clinical endpoints as the use of genetic therapies expands

Defining the Role of Companion Diagnostics

1:45 pm Strategies for Effective Companion Diagnostic Co-Development with Gene Therapies (Virtual)

  • Brian Baker Senior Director, Companion Diagnostics Regulatory Affairs, Regeneron Pharmaceuticals Inc

Synopsis

  • Key considerations for CDx assays used in Gene Therapy development
  • Coordination of regulatory strategies and submissions for the drug and CDx

2:15 pm Regulatory Considerations for Gene Therapy-Specific Companion Diagnostic Devices

Synopsis

  • FDA oversight of in vitro diagnostic devices, including companion diagnostics (CDx)
  • Importance of Co-development of a CDx and planning for contemporaneous approval
  • Overcoming challenges associated with CDx development

2:45 pm Afternoon Refreshments & Networking

BRIDGING THE GAP BETWEEN DRUG SPONSORS & PATIENT COMMUNITIES

3:30 pm First-Hand Experiences of the Clinical Trial Process

Synopsis

  • Outlining the decision-making process: assessing risks, benefits, and drivers for commencing a clinical trial
  • Reflecting on the burden of participating: which elements of the experience were de-stressed and where could improvements be made in the future?
  • Assessing levels of communication from healthcare providers throughout
  • How can drug developers help to empower patients to partner with them on drug development?

4:00 pm Panel Discussion: Evolving the Patient Engagement Paradigm in Rare Disease

  • Pat Furlong Founding President & Chief Executive Officer, Parent Project Muscular Dystrophy
  • Julia Taravella Executive Director, Rare Trait Hope Fund
  • Jacose Bell Director, Patient Advocacy, Spark Therapeutics

Synopsis

  • Outlining how all stakeholders stand to benefit from patient groups being involved throughout the drug development path
  • How can drug developers ensure that the patient voice is never lost throughout the development process?
  • Where in the drug development pathway are their further opportunities for drug developers to engage rare disease patient groups?
  • Stories of successful patient engagement and potential areas for industry to improve on

4:30 pm Chair’s Closing Remarks

4:40 pm End of Conference Day Two