Breakfast Briefing Hosted by Pall Biotech

The View from the Cutting Edge: Gaining the Perspective of Industry Leaders

8:20 am Chair’s Opening Remarks

8:30 am Lumevoq Gene Therapy in Leber Hereditary Optic Neuropathy (LHON)


  • LHON is a maternally inherited mitochondrial disease. It is a rare disease, with an incidence of 1500 new patients per year in Europe and in the US.
  • The 11778 mutation on the ND4 mitochondrial gene is the mutation causing the most severe clinical form of LHON. Lumevoq specifically targets this mutation
  • Lumevoq is a recombinant adenovirus vector type 2, containing the wild type ND4 DNA. Lumevoq is injected via Intravitreal Injection
  • GenSight Biologics have filed for EMA registration in September 2020, and then be moving to BLA for US registration in 2021

9:00 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

9:30 am Tactical Implementation of Patient- Centricity in Gene Therapy Development Programs

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

10:00 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?

10:30 am Morning Refreshments & Networking

Aligning Regulatory Expectations with Current CMC Challenges


Establishing Scalable Manufacturing Processes

Understanding Product Differentiation in an Increasingly Crowded Therapeutic Landscape

Articulating the Value of Gene Therapies to Overcome Market Access Challenges

11:00am 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
Mauricio Umana, Associate Director, Global Regulatory CMC Lead, Biogen

11:00am Effective Approaches toIndustrialize 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
Speaker to be confirmed, Voyager Therapeutics

11:00am Landscape Review of Gene Therapies for Rare Disorders
• A landscape review of the therapy and trials within gene therapy and rare disorder space
• With focus on some of the specific disease areas where gene therapies are seeing success and setbacks
(blood disorders, neurology and ophthalmology disorders, etc.)
• The take at the later phase assets and how they will contribute to the future of gene therapy
Paulina Szymanska, Senior Account Manager, Beacon Targeted Therapies

11:00am 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

11:30am 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
Speaker to be confirmed

11:30am 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

11:30am 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

11:30am 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

12:00pm 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
Speaker to be confirmed,
Halo Labs

12:00pm Roadmap to Successful Commercialization
With the current increase in development and investment, gene therapy companies will be looking at the 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, Manufacturing & Operations, MilliporeSigma

12:00pm Speaking Position Reserved for the Jackson Laboratory

12:00pm Speaking Position Reserved for Invitae

12:30 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

1:30pm 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?
Jim Richardson, Senior Scientific Liaison,US Pharmacopeia

1:30pm Challenges of Manufacturing Gene Therapies into Commercial Realities
• Overview of bluebird bio’s efforts to advance gene therapies for severe genetic disorders and cancer immunotherapy
• Challenges faced in manufacturing cell therapies as they move toward commercial phase – with a particular focus on managing a network of CDMOs
• Approaches bluebird bio is implementing to overcome some of these challenges
Steven Goodman, Head, Drug Product Manufacturing, bluebird bio

1:30pm 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, Chief Patient Officer & Head of Government Affairs, Taysha Gene Therapies

1:30pm 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

2:00pm 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

2:00pm 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 management
• Potential benefits to productivity, product quality, yield and workflow efficiency will be discussed
Shelly Parra, Director, Global Field Applications, Repligen

2:00pm 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

2:00pm  Value-Based Agreements: In Theory vs In Practice

• Articulating value of gene therapy to each stakeholder will be important regardless of reimbursement model
• Value-based models sound good in principle – how will this play out in practice?
• Will every gene therapy need a bespoke reimbursement model?

Mindy Cameron, Consultant, LittleHercules Foundation


2:30 pm Afternoon Refreshments & Networking

Involving the Patient Perspective in Gene Therapy Development & Commercialization

3:30 pm Integrating Patient Preferences into Payment & Access Mechanisms for Transformative Gene Therapy Treatments

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


  • 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

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 Emphasizing the Role of the Patient in Trial Design


  • Understanding what a meaningful outcome is for the patient
  • Designing trials to minimise impact on patient everyday life
  • Establishing meaningful endpoints using patient input

4:30 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)

5:00 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

5:30 pm Chair’s Closing Remarks