LinkedIn Icon
March 26-28, 2019 | Boston, MA

Day One
Wednesday March 27, 2019

Day Two
Thursday March 28, 2019

Chair’s Opening Remarks

Keynote: Turning Genes into Medicines

  • Kathy High President and Head of R&D, Spark Therapeutics


  • Pioneering gene therapies in a range of tissue types and indications
  • Successfully moving from one target tissue to another: what are the cross-tissue insights that can be applied?
  • Navigating key decision points in transitioning academic progress into a reality for patients

Industry Leaders Panel: Pioneering the Next Generation of Gene Therapies


Gene therapies continue to redefine the treatment of rare diseases, but these conditions present many distinct development challenges. This panel discussion will bring together experts from the companies pioneering this field to analyze how we can overcome these hurdles to realize the commercial potential of gene therapies.

  •  Navigating uncharted territory: sharing the decisions industry leaders have made to pioneer progress without a defined pathway
  • Changing the mind-set: understanding how the unique characteristics of gene therapies are in the process of changing the healthcare paradigm and how companies have to adapt to this new landscape
  • Gearing up for commercialization: sharing how the leading companies are preparing for a new wave of gene therapies
  • Ensuring that consistent standards are implemented throughout every stage of the development of gene therapy products

Flexible, Modular Manufacturing to Address Viral Vector Capacity Bottlenecks

  • Xin Swanson Commerical Development Lead, Viral Vector Gene Therapy, Lonza


  • The status and ambitions of cell & gene therapies
  • The critical challenges across the manufacturing pathway for cell and gene therapies
  • Platforms & technologies to address these challenges

Morning Refreshments & Speed Networking

Scaling Up Manufacturing to Meet Future Demand for Gene Therapy Products

11.00 Talk Details to be Confirmed

  • More details coming soon

Derek Adams, Chief Technology & Manufacturing Officer, bluebird bio

11.30 Manufacturing Capacity & Quality Considerations Across a Diversified Gene Therapy Pipeline

  • Capacity considerations for various indications
  • Quality control and characterization
  • Vector potency and concentration

Fraser Wright, Chief Technology Officer, Axovant Sciences

12.00 Successful Tech Transfer from Academia to In-House Manufacturing: It Is Not Just the Process!

  • Understanding the importance of setting strategic goals for process development during tech transfer
  • Design and built a manufacturing facility for long term success
  • Establishing key functions for successful in-house manufacturing

Knut Niss, Chief Technology Officer, Mustang Bio

Navigating the Increasingly Complex Regulatory Landscape

11.00 Understanding How to Harmonize the Global Regulatory Landscape

  • Investigating standards that need to be harmonized that are relevant to advanced therapeutics
  • Introducing the global gene therapy regulatory landscape
  • Learning how the regulatory environment continues to evolve to adapt to novel technologies, applications and regulatory requirements

John Spaltro, Director, Global Regulatory Affairs, Amicus Therapeutics

11.30 Accelerating Concept to Commercial Development of Gene Therapies

  • How to leverage data from similar gene therapy products into clinical trials
  • What to tackle first using accelerated pathways
  • What’s next in gene therapy approaches?

Tim Miller, President & CSO, Abeona Therapeutics

12.00 European Regulatory Perspective on the Development of Gene Therapies for Rare Disorders

  • Gaining insights into the regulatory landscape in Europe
  • Understanding how regulatory pathways are becoming more clearly defined, and the key learning points on this steep learning curve
  • Learning how regulators can react effectively to new developments and technologies in the field

Rune Kjeken, Scientific Director, Advanced Therapies, CAT, EMA

Establishing & Demonstrating the Value of Disruptive Gene Therapy Products

11.00 Insights from the Intersection - Stakeholders Grappling With the Affordability Challenge

  • Outlining the landscape of challenges facing patients, physicians, payers and manufacturers
  • Gaining insights from the pharmacy viewpoint: the touch-point for all relevant stakeholders
  • Understanding how the field can work together to improve patient access to innovative therapeutics

Bill Martin, CCO, Accredo – an Express Scripts Company

11.30 Precision Financing Framework and Pilots: MIT FoCUS Consortium Findings

  • Understanding how high upfront reimbursement for durable precision medicine products will require precision financing approaches
  • Investigating how gene therapies can be analyzed into distinct archetypes and the features of each
  • Understanding the unique financial challenges these different archetypes pose to payers, patients and developers

Mark Trusheim, Strategic Director, NEWDIGS, MIT Center for Biomedical Innovation

12.00 Balancing Access & Affordability in the Context of Innovative but Expensive Therapeutics

  • Understanding how innovative therapies that have the potential to improve the human condition are
    likely to be quite expensive, meaning traditional approaches to obtaining payor coverage may not be sufficient
  • Discussing how payors are thinking about the challenges that present themselves with regard to balancing access and affordability
  • Investigating strategies that are being discussed to assure access to those in a position to benefit
    from these advances

Michael Sherman, CMO & SVP, Harvard Pilgrim Health

Lunch & Networking

14.00 Industrial Purification Platform of AAV

  • Understanding how concentration methods can depress virus recovery and binding capacity, and inflate contamination of subsequent purification steps
  • Investigating how monoliths allow product capture to begin immediately after removal of cell debris and flow rates of several column volumes per minute permit column loading to be completed in less time than the combination of TFF and capture on conventional chromatography media
  • Highlighting an exclusively three step monolithic purification platform: Hydrophobic interactions (HIC) at the beginning are used as a capture step. After this step the AAV fraction is diluted and loaded onto a cation exchanger to disrupt residual virus-protein/DNA complexes, and the cation exchange fraction onto an anion exchanger to separate empty and full capsids
  • The process offers very high yields, is fully scalable and has proven effective for all AAV serotypes
    evaluated to date

Ales Strancar, Managing Director, BIA Separations

14.30 Searching for Functionally Relevant Attributes of Plasmids used in Triple Transfection Viral Vector Production

  • Exploration of triple transfection plasmid quality attributes, control strategies and associated functionality
  • Appreciation for complexity of the study design, level of effort and cross-line collaboration necessary to identify functionally relevant attributes
  • Review of Pfizer’s current investigation/data to determine if plasmid supercoiling percentage is pertinent to efficacy

Lawrence Thompson, Principal Scientist, Pfizer

15.00 Panel Discussion: Comparing Manufacturing Methods for their Suitability for Long-Term Commercial Manufacturing

  • 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 amenable to scale-up and late-stage development
  • Investigating strategies to overcome analytical limitations to make comparability easier
  • Understanding how to convert existing technologies from biologics manufacturing to gene therapies

Palani Palaniappan, Sarepta Therapeutics
Kathleen Hehir, Sanofi Genzyme

14.00 Engaging Regulators Around Key Issues Throughout the Gene Therapy Development Process

  • Understanding the importance of partnering with regulators throughout the development pathway for gene therapy products
  • Learning how and when to engage the authorities most effectively, with objective of agreeing a harmonized global approach
  • Contrasting US and European consultations with regulators and understanding best practices

Ilan McNamara, Senior Director, Global Regulatory Affairs, Prevail Therapeutics

14.30 Regulatory Approaches to Clinical Development & Incorporating Patient Input & Patient Reported Outcome Measures

  • Considerations for use of PRO Measures in clinical trials
  • A Fabry clinical trial example: utilizing a PRO Measure designed for one disease in another disease
  • Understanding the regulatory factors unique to this development process

Nerissa Kreher, CMO, AVROBIO

15.00 Panel Discussion: Defining a Harmonized, Globally-Applicable Regulatory Framework for Gene Therapies

  • How are organizations adapting to the evolving gene therapy regulatory landscape?
  • Defining the risk, benefit and value of gene therapies
  • Gaining clarity on acceptable, clinically-relevant endpoints
  • Investigating collaborative initiatives to share relevant information among organizations active in this field
  • Working towards a single, global, unified position among organizations pioneering this field

Rune Kjeken, EMA
John Spaltro, Amicus Therapeutics
Ilan McNamara, Prevail Therapeutics
Peter Marks, FDA

14.00 Innovative Approaches for Access to Gene Therapies in Rare Diseases

  • Preparing for global commercialization of gene therapies for ultra-rare diseases
  • Emerging access schemes for gene therapies for ultra-rare diseases
  • Facilitating access for patients globally

Markus Peters, Most Recently: CCO, Agilis Biotherapeutics

14.30 Establishing Innovative Payment Models to Improve Patient Access to Gene Therapies While Balancing Requirements of Various Stakeholders Involved

  • Contrasting potential payment options for gene therapy products
  • Understanding the unique challenges involved in pricing potentially one-time, curative therapeutics
  • Assessing pricing and reimbursement frameworks on a global scale

Omar Dabbous, VP, Global HEOR & RWE, AveXis

15.00 Panel Discussion: Learning About Real-World Reimbursement, Pricing & Market Access Mechanisms

  • Investigating real-world learnings from pricing models to incorporate into program designs
  • Beyond the overview – examining at what threshold the budget impact is it even useful or meaningful examine payment over time and performance based models rather than a one-off payment
  • Investigating the challenges in reducing payment models to practice, in terms of data collection, patient tracking, US versus EU experiences, single payer versus multi payer systems

Bill Martin, Accredo – an Express Scripts Company
Mark Trusheim, MIT Center for Biomedical Innovation
Kevin Mayo, PTC Therapeutics

Markus Peters, Agilis Biotherapeutics

Afternoon Refreshments & Scientific Poster Session


Rare Disease Gene Therapeutic Products – The Future is Here!


  • Patients, scientists, clinicians, companies have all been on a rollercoaster ride, now real products are emerging
  • Endpoints that are relevant for approval, relevant for patients and families and for payers must be developed
  • Success in ultra-rare and rare disorders will pave the way for larger footprint diseases

Keynote: Making Medical History – First-Hand Insights into the First FDA-Approved Gene Therapy Procedure for Inherited Disease


  • Translating promise into reality – understanding the true scope of the journey from promising therapeutic to reality for patients
  • Working effectively with patients and defining the patient journey
  • Discussing the future of gene therapy treatments for rare inherited disorders

Panel Discussion: Patient Advocacy in the Gene Therapy Space: A Case Study in Hemophilia


  • How early in the gene therapy development should companies begin their patient advocacy efforts?
  • What are some key considerations for patient advocacy that are unique to gene therapy?
  • Gain transferrable insights from the hemophilia space that can enhance patient advocacy efforts in other disease areas
  • Understanding patient expectations of gene therapies – are these realistic?
  • Discussing examples of patient misconceptions and questions about the field
  • Educating patients and families of the realities and risks associated with gene therapy clinical trials

Chairs’ Closing Remarks & Scientific Poster Session Resumes