Gene Track

Gene Track, Wednesday 25 April 2018





last published: 19/Mar/18 14:25

Gene Track, Thursday 26 April 2018



Katherine High

Keynote address: A new era in medicine- introducing gene therapy

  • What we’ve learned – lessons science has taught us as we create the path for the first gene therapy for an inherited disease in the U.S.
  • Staying true to our roots while preparing the patient, medical and policy maker communities for a new chapter in the retinal disorders textbook
  • Assessing the impact of potential one-time treatments like gene therapies for patients with rare diseases
Hans Bishop

Keynote address: Cell and gene therapy innovation and their role in the future of rare disease research

  • Current state of cell and gene therapies and why this field is becoming more and more significant 
  • Looking into scalability and centralized approaches for cell and gene therapy development and commercialization
  • Novel approaches and how these therapies benefit the continuum of research for rare diseases
Andre Choulika

Keynote address: Next- generation CAR T-cells to cure cancer - paving a new way for R&D, value-assessment and pricing and reimbursement of treatments for genetic diseases

  • Learnings from CAR-T development that can be applied to rare disease drug development 
  • Will CAR therapy pricing impact pricing schemes of other orphan drugs and gene therapies? 
  • Exploring aspects of these different therapeutic areas that share common goals, personalization and pricing challenges 
Panel discussion

Keynote CEO Panel: Gene therapy - a new paradigm for patients, new challenges for industry

  • Looking back at the commercial performance of the first generation of gene therapies- from proof of concept to the road to approval 
  • How will gene therapy innovation look in the next 10 years?
  • Considering potential new value propositions for patients, payers and the health care system
  • Strengthening clinical development, exploring novel technologies while maintaining safety- what needs to be improved? 


Gene Track

Genetic Testing, Genome Sequencing

Daniel Anderson
Gene Track

Genetic testing for patient engagement – shortening the diagnostic odyssey and connecting patients to research and treatments

  • No-cost genetic testing programs support patients and clinicians to gain an accurate diagnosis quicker by removing barriers to genetic testing
  • Drive patient identification, expedite trial enrollment and accelerate time to market for new therapeutics
  • Create engaged patient communities that partner biopharma and patient advocacy groups through Patient Insights Networks (PIN)
Christina Waters
Gene Track

The value of genomic data to uncover pathogenic mutations linked to rare diseases

  • Leveraging next generation sequencing and genome interpretation for rare disease research 
  • Combining data science and systems biology to accelerate rare disease diagnosis and therapeutic discovery
  • Searching for and decoding rare disease “genomic shields”
  • Fostering partnerships to advance rare disease therapies  
Christina Waters, Senior Vice President and General MAnager, Rare Disease Programs, WuXi NextCODE
Tero-Pekka Alastalo
Gene Track

Validating and optimizing NGS-based diagnostics to improve diagnostic yield and clinical utility

  • Challenges in the field of genetic diagnostics and current technologies used to address them
  • Introducing the concept and importance of transparent, traceable, and comprehensive analytic validation of an NGS platform for clinical diagnostics
  • Patient examples which demonstrate the importance of high quality and comprehensive genetic diagnostics for improved outcomes for rare disease patients
  • Controlling costs by utilizing high quality WES derived sequencing data for rare disease patients


Yann Le Cam

Lunch keynote address: Breakthrough therapies call for breakthrough processes to ensure sustainable patient access


2:30 ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min – attendees can select two roundtables to attend)

If you are interested in sponsoring a roundtable contact André Singer at or +1 646 619 1797

ROUNDTABLE 1: Pediatric trials - reducing the financial, logistical and emotional burden for patients and families participating in pediatric trials

Andrew McFadyen

ROUNDTABLE 2: Compassionate Use Programs - helping patients get access to medication, educating the community, physicians and investors to ensure safe access to experimental treatments

Scott Schliebner

ROUNDTABLE 3: Virtual Trials - enabling clinical trials to be more virtual, reducing distance times, overcoming geographic barriers and increasing data collection through the use of wearables

Jayne Gershkowitz

ROUNDTABLE 4: Expanded access - incorporating the patient advocacy perspective to develop a review process and a formalized procedure on expanded access

Joseph Musumeci

ROUNDTABLE 5: BD & Forecasting - pricing and access considerations and understanding how to build the patient forecast based on available epidemiological information

Yvette Venable

ROUNDTABLE 6: Strategy - patient engagement and pipeline building for the commercialization and access of rare disease products

Francesca Cook

ROUNDTABLE 7: Gene therapy – new payment and financing models are needed to facilitate access

Michael Murphy

ROUNDTABLE 8: Clinical development – When the sum is greater than the parts through academic, pharmaceutical and CRO collaboration in orphan disease drug development

Robert Donnell

ROUNDTABLE 9: Managed Access Programs - How MAPs (pre-commercial supply to patients with unmet medical needs) can be a valuable component of launch strategy for orphan drugs

Samantha Parker

ROUNDTABLE 10: Gene therapy - Optimizing gene therapy clinical development through education, collaboration, patient input and involvement in advisory boards

Philip J. Brooks

ROUNDTABLE 11: Scientific innovation - platform approaches to evaluate multiple genetic diseases at the same time

David Lapidus

ROUNDTABLE 12: Epidemiology and forecasting - using epidemiological data to determine market viability, niche populations and commercial differentiation

Betsy Ricketts

ROUNDTABLE 13: Government affairs - developing a company’s footprint and contributions to impact policy, reimbursement and patient engagement

Anne Cropp

Roundtable 15: Expanded Access - drug development strategy considerations for open label extension or expanded access protocols

Ari Brettman

ROUNDTABLE 16: VC funding – successfully addressing investors interests and challenges when assessing companies of all sizes to guarantee funding of orphan drug biotechs

Trista Morrison

ROUNDTABLE 17: Disease awareness - best practices and tips for smaller companies to tell a corporate or rare disease story and raise awareness


Emerging Markets

Clinical Development

Olivier Danos
Gene Track

Creating value in gene therapy clinical development programs, achieving trial optimization and supporting the path to commercialization

  • Investigating novel vector technologies that can improve efficacy while reducing immunogenicity and potential re-dosing 
  • Thinking about end to end clinical development including trial design, enrollment, execution, supply chain and manufacturing 
  • How can clinical development be optimized to support scalability? What are the current limitations faced by this field despite a decade of improvements?
Dana Levasseur
Gene Track

Gene editing approaches in rare blood disorders

  • Unmet need for transformative therapies in hemoglobinopathies
  • Disrupting an erythroid specific BCL11A enhancer with zinc finger nucleases for the treatment of sickle cell disease and beta-thalassemia 
  • The potential to reactivate fetal hemoglobin to treat patients with SCD and beta-thalassemia
Mariah Baltezegar
Gene Track

Achieving patient retention in gene therapy follow up

  • Strategies for optimizing patient retention throughout gene therapy programs (Phase 1-3, follow-up studies, registries)
  • New ways to better understand and navigate patient challenges first hand (logistical hurdles presented by moving from the initial “treatment” site/country (HCP) to potentially new follow-up sites/HCPs) 
  • Gene therapy trials vs. other trials and what needs to be considered 



Douglas Ingram

Keynote panel: How rare disease companies are ramping up innovation to boost orphan drug development and patient centricity

  • Incorporating patients in the drug development process and working towards commercial viability- how to fully engage? 
  • How do you view disruptive therapies such as (gene therapy/gene editing) impacting the broader orphan disease development paradigm? How are you innovating within your company?
  • Responding to the changes in the political, regulatory and reimbursement landscape while guiding the direction of rare disease companies 
  • How to achieve industry collaboration and idea exchanges that can better serve the patient community? 

6:20 NETWORKING COCKTAIL PARTY – Reserved for Gold Sponsor

last published: 19/Mar/18 14:25