March 26-28, 2019 | Boston, MA
Register by Friday, March 22 to secure the best rate!

Day One
Wednesday March 27, 2019

Day Two
Thursday March 28, 2019

Registration & Light Breakfast

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 Scaling Gene Therapy Manufacturing: What is Our Goal?

  • Highlighting the challenge of dramatic patient benefit with under-developed manufacturing processes
  • Outlining the inextricable link between manufacturing and the patient experience for autologous cell therapies
  • Considering the different scaling goals: commercial viability, reliability, patient access, etc.

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 Accelerating to Commercial Viral Vector Manufacturing

  • Context around the current regulatory environment and demand
  • Qualifying and operationalizing facilities and systems
  • Building the workforce and competencies for production ramp-up

Christopher Murphy, Chief Operations Officer, Brammer Bio

Establishing Global Clinical & Regulatory Standards

11.00 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

11.30 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

12.00 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
Peter Marks, FDA

Tim MillerAbeona Therapeutics

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

13.30 Towards a 3rd Generation AAV Manufacturing Platform & In-Process Controls

  • One of the key challenges in manufacturing viral vectors is managing the interface between upstream and downstream processing.
  • In addition to losses due to shear, processing methods such as precipitation, freeze/thaw and tangential flow filtration (TFF) promote formation of stable associations between virus, DNA and proteins. These complexes lower virus recovery and process capacity, depress robustness, and inflate contamination at each processing step. They accordingly have strong potential to influence long term clinical safety, especially with respect to residual RNA/DNA.
  • 2nd generation AAV platform engineered specifically to address these issues was developed.
  • This orthogonal process achieves very high recoveries (>70%) with AAVs that are secreted from the host cells and very efficient and improves overall reduction of all contaminating viruses.
  • Rapid sensitive analytics using multiple in-line HPLC detectors to provide insightful process development and manufacturing documentation will also be presented.

Ales Strancar, Managing Director, BIA Separations

14.00 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

14.30 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

13.30 Development of a Complement Targeting Gene Therapy for Dry & Wet Age-Related Macular Degeneration (AMD), Interim Results of Two Clinical Trials

  • AMD is the most common cause of blindness in the elderly
  • There is currently no treatment for the dry form of AMD and treatment for wet AMD requires monthly intraocular injections
  • Complement is activated in AMD
  • We have developed a gene therapy approach (HMR59) targeting the membrane attack complex (MAC), the final step of activated complement
  • Safety data from a 6 month, 17 patient Phase I dry AMD study as well as well as interim efficacy data from the first 13 patients of a wet AMD study will be presented

Rajendra Kumar-Singh, Founder, Hemera Biosciences

14.00 Ex-Vivo Lentiviral Vector Mediated Gene Therapy for Fabry Disease; An Update.

  • Considerations for a global clinical program
  • Preliminary clinical data
  • Future perspectives

Birgitte Volck, President, Research & Development, AVROBIO

14.30 Panel Discussion: Enhancing Gene Therapy Clinical Development

  • Learning from innovative gene therapy trial designs in the rare disease space – structure, patient selection/recruitment/retention
  • Identifying appropriate biomarkers for gene therapy clinical trials
  • Discussing how endpoint selection can have far-reaching consequences in the drug development and commercialization timeline

Rajendra Kumar-Singh, Founder, Hemera Biosciences

Birgitte Volck, President, Research & Development, AVROBIO

Harrison Brown, CSO, Odylia Therapeutics

13.30 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

14.00 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

Ramesh Arjunji, Senior Director, HEOR & RWE, AveXis

14.30 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

Marcus Peters, Agilis Biotherapeutics
Mark Trusheim, MIT Center for Biomedical Innovation

Kevin Mayo, PTC Therapeutics


Afternoon Refreshments & Scientific Poster Session


Creating a Novel Business Model for Ultra-Rare Disease


  • Developing a commercial business model for ultrarare gene therapy
  • Advantages of non-profit over for-profit models
  • Investigating the needed paradigm change for ultra-rare disease translation

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

Chair’s Closing Remarks & Scientific Poster Session Resumes