Breakfast Briefing Hosted by Pall Biotech

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 Tactical Implementation of Patient- Centricity in Gene Therapy Development Programs

  • Leone Atkinson Executive Medical Director, Rare Disease & Pediatrics Team, Covance

9:30 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?

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 Textbook vs. Reality: How the “Real World” Evaluates Gene Therapies
From an academic and clinical perspective, the use case for gene therapies seems open and shut.
Yet, when we objectively evaluate current and potential gene therapies in development many questions loom large. Queries touch upon multiple areas - not just clinical, and economic,
but also sensitive ethical and psychological dilemmas stakeholders might face. This presentation will highlight how to best recognize and address these questions through education and evidence generation to ensure optimal launch success.
Nandini Hadker, Trinity
Krista Perry, Trinity

11:15am CMC Developability
• Transition of new gene therapy molecules from research into development space can result in unexpected challenges putting CMC on the critical path
• Develop-ability assessments of new molecules can help predict where the CMC challenges may lie
• Companies can then decide where to invest their resources in CMC, including an outsource v. internalization strategy
Kathleen Hehir, Head of Gene Therapy Manufacturing & Development, Sanofi

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 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

11:45am 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 Establishing Mutually Beneficial
Relationships with Contract
Companies & Managing
Complex Supply Chains
• Facilitating complex relationships
with multiple contract
• Investigating the key constituents
of effective collaborations with
external partners
• Understanding how to manage
gene therapy supply chains to
deliver efficient and cost-effective
James Biggins,
VP, External
Development & Supply, Orchard

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 BioMarin


12:15pm Automated, Low-Volume
Subvisible AAV Aggregate Analysis
with the HORIZON System
• Subvisible particle analysis is a key
predictor of stability and safety
and is an essential parenteral drug quality metric
• Assessing vector particle
aggregates in the subvisible range is especially challenging in AAV formulation development where limited precious sample is available
• The HORIZON® system from Halo Labs employs Backgrounded
Membrane Imaging (BMI)
technology as a solution for
detecting and quantifying
subvisible vector particle
aggregates in low volume, high
throughput format. BMI is fully
automated, fluidics-free, and uses only 25μL of sample
Renee Tobias, Director of Marketing, Halo Labs

12:15pm Roadmap to successful commercialization
With current increase in development and investment, gene therapy companies will be looking at
manufacturing and commercialization of their therapies.
In this presentation, we will present:
• Critical factors to successful commercialization
• Discuss what’s needed for late phase and commercial manufacturing
• Discuss how to pick the right strategic partner CMO
Jessica Hilmoe, Technical Sales Manager, MilliporeSigma

12:15pm Speaking Position Reserved for The Jackson Laboratory

11:45am Presentation Details to be Confirmed
Gautam Mehta, Commercial Partnerships, InVitae



12:45 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

Leslie Powell, Director, Policy &
Advocacy, Cystic Fibrosis Foundation


2:45pm Technology Breakthroughs and
Solutions in Plasmid and Viral
Vector Manufacturing
• Breakthrough clarification and
harvest technology that can signal a
paradigm shift in mAbs bioprocessing
• Advances in multiple disciplines,
combining novel polymer technology,
various filtration modes, hardware,
systems and automated process
• Potential benefits to productivity,
product quality, yield and workflow
efficiency will be discussed
Shelly Parra, Director, Global Field
Applications, Repligen

3:00 pm Afternoon Refreshments & Networking

Involving the Patient Perspective in Gene Therapy Development & Commercialization

3:30 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

4:00 pm Our Life…Our Fight. We need a cure for NF2


• Patient’s perspective on living with a progressing debilitating rare disease
• How patients and families are fighting back
• Novel therapies hope for NF2 (Neurofibromatosis type 2)

4:30 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

5:00 pm Chair’s Closing Remarks