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March 26-28, 2019 | Boston, MA

Day One
Wednesday March 27, 2019

Day Two
Thursday March 28, 2019

Chair’s Opening Remarks


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?

FDA’s Efforts to 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

Morning Refreshments

Overcoming Key Strategic Manufacturing Challenges

11.00 The Pharmaceutical Development, Pilot Plant, CMO Continuum for AAV Vector Manufacturing

  • Understanding key manufacturing challenges encountered at various stages of development
  • Establishing effective outsourcing relationships with CMOs to handle large-scale vector manufacturing
  • Insights into the unique challenges encountered in AAV vector development and manufacturing

Reed Clark, SVP, Ultragenyx

11.30 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

12.00 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

Building a Robust Path to the Clinic

11.00 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

11.30 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

12.00 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

11.00 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

11.30 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

Scott Dorfman, CEO, Odylia Therapeutics

12.00 Panel Discussion: Understanding the Large Pharma Perspective on the Gene Therapy Space • How can

  • How can large companies without a history in the gene therapy space invest resources most effectively to rapidly establish market share?
  • Investigating the differences in developing gene therapies at large pharma vs small biotech organizations
  • Evaluating the stories behind recent collaborations and acquisitions in the field

Troy Dale, Worldwide Head Commercial Functions Strategic Partnership Team, Novartis
Brian Bronk, Head of External Innovation & Rare Disease, Sanofi

Lunch & Networking

Defining Processes to Enhance Long-Term Commercial Manufacturing

13.30 Maximizing the Value of Platform Technology

  • More details to follow

Jude Samulski, Professor, Pharmacology, UNC Gene Therapy Center

14.00 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

Jude Samulski, Professor, Pharmacology, UNC Gene Therapy Center
Tim Miller, President & CSO, Abeona Therapeutics

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 Gene Therapy Clinical Development Program for X-Linked Myotubular Myopathy (XLMTM), a Rare, Severe Neuromuscular Disease

  • Describe the unique challenges in designing a gene therapy development program for a rare neuromuscular disease
  • Rationale of the clinical development program for AT132 (AAV8-Des-MTM1) in XLMTM
  • Present interim efficacy and safety data of ASPIRO, a phase 1/2 clinical trial to assess AT132 in XLMTM

Salvador Rico, VP, Clinical Development, Audentes Therapeutics

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, Adrenas Therapeutics

Afternoon Refreshments


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

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

Chairs’ Closing Remarks

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