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


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

  • Morrie Ruffin Co-Founder & Senior Advisor, Alliance for Regenerative Medicine
  • Lyndsey Scull Vice President, Communications, Alliance for Regenerative Medicine


  • Exploring the evolving clinical and investment landscapes
  • Examining the recent progress and near-term potential of the gene therapy space
  • Estimating and measuring the impact this sector is poised to make on the healthcare system overall
  • Understanding current public perception of gene-based medicines, and further education needs

FDA’s Efforts to Facilitate the Development and Availability of Gene Therapy Products


  • Provide a brief overview of recent advances in the area of gene therapy
  • Review the regulatory framework relevant to the approval of gene therapy products
  • Discuss expedited programs applicable to gene therapy products
  • Provide an overview of applicable guidance documents and other resources for product developers
  • Detail challenges currently limiting the potential application of gene therapy to broader populations, and discuss some potential solutions

Challenges and Considerations for Large Scale Manufacture of an AAV-Based Gene Therapy Product


  • Regulatory expectations: understanding stringent and rigorous requirements and how best to leverage knowledge gained over 35 years of producing recombinant DNA derived products
  • Strategic process development: holistic approach focused on producing high quality products and scalable processes leading to rapid approval of safe and efficacious therapeutic products for patients
  • The potential: clinical outcomes from phase 1/2 studies conducted over a 2-year period

Optimizing Clinical Development in Rare Disease: A CRO Perspective


  • Relevance of broad therapeutic and scientific expertise for novel therapeutic approaches such as gene therapy
  • Development and applications of data and technologies in rare disease
  • Role of biomarker data to provide “Virtual Natural History” studies in rare diseases

Morning Refreshments & Networking

Overcoming Key Strategic Manufacturing Challenges

10.45 Scaling Manufacturing to Deal with the Requirements of Systemically Administered Gene Therapies

  • 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

Palani Palaniappan, Head, Technical Operations, Sarepta Therapeutics

11.15 Establishing Long-Term, Scalable Manufacturing Processes Early in Development

  • Speed to clinical data vs. speed to market
  • The cost of changing – time vs. money
  • Patients-first approach

Dave Knop, Executive Director, Process Development, AGTC

11.45 Considerations for a Successful Gene Therapy Facility Build

  • Understanding the site selection process including a “make vs. buy” business case analysis
  • Utilitzing modular and single use technologies
  • Setting up the build for success including creating proper project work streams
  • Other considerations such as phased implementation, commissioning and qualification strategies, and change management

Tony Khoury, Principal Engineer, Project Farma

Greg Gara, VP, Technical Operations & Engineering, AveXis

Building a Robust Path to the Clinic

10.45 Overcoming Translational & Clinical Barriers to Enhance the Development of Novel Vectors

  • Identifying clinically relevant routes of administration
  • Understanding approaches to address the issue of neutralising antibodies
  • Improving the tissue targeting of gene therapy products

Melissa Kotterman, Senior Director, Head of Discovery & Engineering, 4D Molecular Therapeutics

11.15 Overcoming the Unique Translational Challenges Posed by Single Dose Gene Therapies

  • Investigating surrogate and non-invasive biomarkers to support the development of gene therapy products
  • Understanding dose selection challenges
  • Precision medicine in the gene therapy space – addressing the unique challenges

Carl Morris, CSO, Solid Biosciences

11.45 Navigating the Gene Therapy Path for CNS Diseases to the Clinic – Rationale, Pharmacology, and Delivery

  • Understanding the unique challenges posed by delivering gene therapies to the CNS
  • Learning how to improve the translatability of preclinical gene therapy programs
  • Investigating transferrable lessons learnt that can lead to success in gene therapy programs for a
    range of indications

Ralph Laufer, CSO, Lysogene

Evaluating Potential Gene Therapy Commercialization Strategies

10.45 Assessing the Value of Gene/Cell Therapies

  • Understanding how to effectively demonstrate value of your gene therapy in an ever crowded and complicated space
  • Creating demand across stakeholders
  • Effectively & accurately forecasting demand and estimating share for increasingly complex markets/products
  • Impact / limitations of gene therapy assessments, such as more complex pricing/contracting models

Krista Perry, Principal, Trinity Partners

11.15 The Emerging Business Models for Commercializing Gene Therapy Treatment for Hemophilia

  • The emerging novel market for gene therapy, along with the specific dynamics of hemophilia, together create an intriguing business challenge treatments with curative potential
  • There is a complex array of stakeholders in the market including government and private payors, hemophilia treatment centers, specialty pharmacies, and patients and patient advocacy groups
  • Gene therapy holds great promise and offers significant value to patients and payors

Jonathan Garen, CBO, uniQure

11.45 Innovative Partnerships That Accelerate Research & Treatment in Rare Disease by Increasing Access to Genetic Testing

  • Removing entrenched roadblocks to genetic testing that perpetuate the longstanding challenges of time to diagnosis by connecting patients to clinical trials and on-market therapies in rare diseases
  • Discovering how innovative partnerships are breaking down these barriers, taking 1-2 years off time to diagnosis and significantly accelerating clinical trial enrolment
  • Outlining how these programs create value for all involved — physicians, advocacy, biopharma and, most importantly, patients and their families — as they support faster drug development and the transition to market for new therapies
  • Discussing future partnership trends that will further open up access to genetic testing

Katherine Stueland, CCO, Invitae

Lunch & Networking

Defining Processes to Enhance Long-Term Commercial Manufacturing

13.30 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

14.00 Discovering, De-coding and Programming AAV

  • What are the structural challenges of AAV discovery and design?
  • Description of a novel methodology for AAV diversification through evolutionary modelling
  • Presentation of novel approaches to interrogate AAV function
  • Illustration of how structure-function studies can guide rational AAV design

Luk Vandenberghe, Associate Professor, Harvard Medical School

14.30 Maximizing the Value of Platform Technology

  • More does not mean better!
  • One hit wonders are in vogue!
  • 7,000 genetic disorders and climbing

Jude Samulski, Professor, Pharmacology, UNC Gene Therapy Center

Investigating Innovative, Patient-Centric Clinical Strategies

13.30 Revealing How Patients Can Help Create and Tailor Gene Therapy Development by Providing Real-World Input

  • Understanding how patient and physician engagement can lead to accelerated study enrolment
  • Investigating the factors that are actually important to the patient and how clinical development plans can reflect that
  • How can the patient’s voice be represented clearly and comprehensively?

Jill Dolgin, Head of Patient Advocacy, AGTC

14.00 Centering the Rare Disease Community as Research Partners: An Overview of Collaborative Research & Registry Models

  • Describe how collaborative research models empower rare disease communities
  • Demonstrate the value of longitudinal patient registries and natural history studies
  • Identify challenges and considerations for conducting rare disease research

Vanessa Boulanger, Director of Research, National Organization for Rare Disorders

14.30 Panel Discussion: Evaluating How Potentially Curative Gene Therapies Can Change the Patient Care Paradigm

  • Understanding the experiences of current clinical trial patients to inform the structures and support that needs to be put in place in the future
  • Representing the patient perspective at every stage of gene therapy development
  • Discussing strategies to alleviate the patient burden

Jill Dolgin, Head of Patient Advocacy, AGTC

Vanessa Boulanger, Director of Research, National Organization for Rare Disorders

Ralph Laufer, CSO, Lysogene

Establishing Mutually Beneficial Collaborations & Partnerships

13.30 Leveraging Academic Research & Innovations for Business Success

  • Sustained long term interest and organic growth of gene therapy capabilities at academic centers has thrust academia front and center into gene therapy drug development
  • Centers have invested significantly in creating lead drug candidates, preclinical research, incubator spaces, cGMP manufacturing capabilities and clinical study sites
  • This has created unique opportunities for academic center and businesses alike to work together and bring novel and transformative therapies to patients with rare diseases

Satinder Rawat, Licensing Officer, UMass Medical School

14.00 Navigating the Gene Therapy Rare Disease Landscape from the Perspective of a Start-Up

  • Key first hires
  • What to build and what to outsource
  • Negotiating and working effectively with academia
  • Working with regulators

Eric David, CEO, BridgeBio

14.30 Panel Discussion: How can Organisations Forge Effective Collaborations in the Gene Therapy Field?

  • Evaluating the nature of collaborations between biopharma
    companies, academic institutions and solution provider companies– how have they
    evolved and how can they be improved?
  • How can organisations in the field collaborate more effectively to bring therapies to
    patients more rapidly?

Satinder Rawat, Licensing Officer, UMass Medical School

Eric David, CEO, BridgeBio

Brian Bronk, Head of Business Development, Rare Diseases & Rare Blood Disorders, Sanofi


Afternoon Refreshments


Panel Discussion: Evaluating the Advantages of Novel Platform Technology Commercialization Templates


  • Contrasting potential approaches for collaborations between pharma and biotech
    companies in achieving commercialization
  • Investigating approaches to manage risk when simultaneously developing a range of platform programs
  • Understanding the future of novel capsid design

Using the NAV Platform of AAV Vectors for Gene Therapy in the Eye: The Case of Wet AMD


  • Why AAV vectors?
  • Capsids and routes of Administration
  • Investigating RGX-314, an AAV8-based gene therapy treatment for wet AMD

Mechanistic Insights & Enhancements Toward Effective CNS Transduction After Vascular Delivery


  • Transient BBB disruption potentiates AAV CNS gene therapy interventions
  • Species specificity of mouse-evolved AAV capsids
  • Mechanistic insights from mouse strain specificity of AAV-PHP.B

Chair’s Closing Remarks

Close of Day Two & End of Gene Therapy for Rare Disorders 2019