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7:50 am Chair’s Opening Remarks

Reality Check: Mapping the Gene Therapy Landscape & Understanding How to Approach Emerging Challenges

8:00 am Presentation Details to be Confirmed


Speaking Position Reserved for BioMarin

8:30 am What About the Long-Run? Questions we Should be Asking About Cell & Gene Therapy Reimbursement

  • Anna Kaltenboeck Senior Health Economist and Program Director, Drug Pricing Lab, MSKCC


  • The first cell and gene therapies to launch in the US have set a high bar for pricing, creating challenges for existing reimbursement practices
  • Novel financing approaches and outcomes-based contracts have been proposed as solutions to prevent the high price points of these therapies from acting as barriers to access
  • However, these approaches do not address important uncertainties about the performance of new treatments, the potential long-term consequences of their wide-scale adoption, and incentives for future innovation

9:00 am Setting Out the Gene Therapy Landscape: Where Are We Now?


  • Establishing the context and understanding how far the field has come in just a few years
  • Understanding how the investment and clinical landscapes are evolving
  • How has the field progressed in the last year and what kinds of challenges have come to the fore?

9:30 am Speaking position reserved for Covance

10:00 am Morning Refreshments & Networking

Aligning Regulatory Expectations with Current CMC Challenges

Establishing Robust Internal & External Manufacturing Approaches

Understanding Product Differentiation in an Increasingly Crowded Therapeutic Landscape

Articulating the Value of Gene Therapies to Overcome Market Access Challenges

10:45am Gene Therapy CMC Strategies, Approaches & Lessons Learned
• What does the final CMC package need to look like?
• Beyond individual assays - how does the entire CMC strategy take shape and what are the unique challenges involved in gene therapy CMC approaches?
• Discussing the ever-evolving regulatory requirement in the field
Chris Morrison, Senior Director, CMC Operations, Gemini Therapeutics

10:45am Effective Approaches to Industrialize the Manufacturing
of AAV Vectors for Gene
Therapy Applications
• CMC competencies required for effective AAV product development
• Building internal vs. leveraging
external capabilities
• Production platform choices and consequences
• Outlook for AAV manufacturing
Luis Maranga, Chief Technical
Operations Officer, Voyager

10:45am Designing Clinical Trials to Achieve the Target Product Profile (TPP)
• To differentiate or not to
differentiate – the importance of a
robust Target Product Profile (TPP)
• Designing a clinical program of
studies to achieve the TPP
• Choice of comparators (activecontrol,
Natural history studies,
• Superiority vs Noninferiority trial
designs in the context of rare
disease studies
• Utility of data extrapolation
Jason Mallory, Clinical Development Leader, Spark Therapeutics

10:45am Uncovering Creative
Approaches to Generate
Relevant Data to Support
Access to these Therapies
• Understanding approaches to articulate the value of gene therapies to put you in a position to provide access to these therapies
• What are the obstacles to patient access to gene therapy products?
• Highlighting the importance of early evidentiary needs assessment and high-quality data collection to illustrate the benefits of one-time treatments to support reimbursement decisions
Doug Danison, VP, Head of Access, Value & Evidence Strategy (AVES), bluebird bio

11:15am Assessing the Current Regulatory CMC Environment for Gene Therapies
• Understanding the CMC requirement to obtain regulatory approval for new gene therapy products
• Identifying regulatory gaps in gene therapy manufacturing approaches
• Highlighting the crucial differences between gene therapy CMC challenges other biologics
Quin Christensen, Senior Manager, Regulatory CMC, Orchard Therapeutics

11:15am State of Gene therapy Manufacturing at Sarepta
• Insights into the quality and purity of material generated by various manufacturing methods
• Investigating new developments to optimize the productivity of the platforms currently available
• Highlighting the approaches most amendable to scale-up and late-stage development
Palani Palaniappan, SVP & HEad of Global Technical Operations, Andover Site, Sarepta

11:15am Recognizing the Best Clinical Candidates for a Gene Therapy
• Identifying the best clinical candidate for specific indications
• Establishing approaches that coud robustly select vectors on a case-by-case basis
• Investing in vectors with the best long-term outcomes
Vivian Choi, Had of Global Gene Therapy Research, Takeda

11:15am Speaking Position Reserved for BioMarin

11:45am Can a Platform Approach Address CMC Challenges Specific to CGT for Rare Disorders?
• Discussing technical challenges faced due to novelty of product modality, manufacturing process and limited prior experience
• Understanding the limited time for CMC development due to accelerated pathways
• Addressing the manufacturing experience due to small number of clinical batches
Yogesh Waghmare, Associate Direction & Head of Downstream Vector Process Devlopment & CMC Lead, bluebird bio

11:45am Investigating Make Vs Buy Decisions and Establishing Mutually Beneficial Relationships with Contract Companies
• Designing and optimizing manufacturing processes to scale-up to commercial scale
• Facilitating complex relationships with multiple contract manufacturers to deliver
• Understanding the challenges involved in bringing manufacturing in house
Scott Cross, VP, Vector Operations, Orchard Therapeutics

11:45am What Must be Improved in AAV-Based Gene Transfer Technologies?
• Analyzing current limitations to AAV therapies
• Investigating novel technologies that optimise delivery
• Novel therapeutic applications in sight
Olivier Danos, CSO, REGENXBIO

11:45am Speaking Position Reserved for Trinity

12:15 pm Networking Lunch

Evaluating and Guaranteeing the Quality of Raw Materials

Investigating the Key Factors Involved in Implementing Cost-Effective and Efficient Manufacturing Processes at Every Scale

Channelling Patient Input for a More Successful Gene Therapy Program

Evaluating Rare Disease Business Models & Integrating the Patient Perspective in Affordability & Access Discussions

1:45pm Tackling Key Challenges Involving Raw & Starting Materials for Gene Therapy Manufacturing
• Understanding the quality of raw materials required for gene therapy products
• Establishing effective standards and parameters to maintain the quality of raw materials
• How can you re-evaluate raw material strategies during scale-up to ensure they are appropriate?
Richard McFarland, President, Standards Coordinating Body

1:45pm Leveraging High-Throughput
Strategies to Accelerate AAV Purification Process Development

• Building high throughput toolkits for Gene therapy process development
• Case study 1: developing a serotype agnostic capture step using the HTP approach
• Case study 2: mimicking a gradient operation through the pseudogradient approach on a liquid handing system
Xiaotong Fu, Senior Engineer, Biogen

1:45pm Overcoming Barriers to Patient Recruitment for Clinical Trials
• Realizing the need for earlier and better diagnosis
• Capturing patient insights to inform
clinical trial endpoints and study logistics
• Minimizing practical hurdles to reaching the clinic
• Clarifying eligibility of patients for trials – matching the right trial to the patient
Emily McGinnis, VP, Patient Advocacy & Professional Relations, AveXis

1:45pm Rare Disease Portfolio: Financial Engineering Can Help Bring More Funding to Develop Orphan Drugs
• The portfolio approach reduces volatility of investing in rare disease therapeutics
• Leveraged financing using structured debt can reduce risk and increase returns
• Investors can do well by doing good by investing in rare disease portfolios
• Increased funding can help more rare disease patients have access to much needed therapies
Nora Yang, Managing Director & CSO, Cura Ventures

2:15pm Roundtable Session: Transitioning Efffectively to GMP Raw Materials
• What does phase-appropriate development really mean in the context of raw materials?
• How can you re-evaluate raw material strategies during clinical process to ensure they are appropriate?
• Investigating the technical requirements at each stage of development

2:15pm Implementing Effective Technologies for AAV Development and Manufacture
• Engineering the baculovirus
• Investigating approaches for product quality control and improved potency
• Process intensification through new media and feeds
Peter Slade, Director, Cell Culture Development, Voyager Therapeutics

2:15pm Partnering with Patient Advocacy Groups: An Integrated Approach to Gene Therapy
• Discussing when and how best to establish connections with patient advocacy groups
• Liaising with advocates in a way that maximises outcomes for all stakeholders
• Understanding the role patient advocacy groups play at multiple stages of the program
Emily Milligan, Executive Director, Barth Syndrome Foundation

2:15pm Integrating Patient Preferences into Payment & Access Mechanisms for Curative Gene Therapy Treatments

• Bridging the gap between patients and payers
• Case study: How the Cystic Fibrosis Foundation is addressing cost, affordability and access
• Investigating the realities of patient access to high-priced treatments

Lisa Feng, Senior Director, Policy &
Advocacy, Cystic Fibrosis Foundation


2:45 pm Afternoon Refreshments & Networking

Involving the Patient Perspective in Gene Therapy Development & Commercialization

3:45 pm Incorporating the Patient Voice at Every Stage of the Program


  • Understanding patient views to explain their unmet needs to regulators for trial approval
  • Strategies to allow optimal patient input for clinical programs
  • Different approaches for different circumstances

4:15 pm Panel Discussion: How Can Patients, Advocacy Groups and Companies Work More Effectively with Payers?


  • Including the patient voice to educate and inform payers with real-life experiences from patient advocates
  • Understanding the implications of reimbursement strategies on patients and the wider family
  •  Contrasting the discussions in indications with existing therapeutic options and those with a lack of treatment options

4:45 pm Chair’s Closing Remarks