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Scientific and Ethical Underpinnings of Gene Transfer/Therapy in Vulnerable Populations

March 7, 2019 @ 8:00 am - 4:15 pm

Scientific and Ethical Underpinnings of

Gene Transfer/Therapy in Vulnerable Populations:

Considerations Supporting Novel Treatments

March 7, 2019, 8:00 a.m.-4:15 p.m.
Amicus Therapeutics, 1 Cedarbrook Drive, Cranbury, NJ


Click here to view the agenda.

Join the Discussion:

Development of new advanced therapeutics, including the use of gene transfer for vulnerable rare disease populations and pediatric cohorts presents a host of unique scientific, ethical and clinical development challenges. Hear the latest from disease experts, Patient advocacy representatives and key industry stakeholders who make it their mission to address these challenges on a daily basis to bring new treatment options to Patients, Because Patients Can’t Wait®!

This program is designed especially for industry professionals pursuing the treatment of adult and pediatric rare disease(s), including the following functional areas:

  • Clinical Research Development and Operations
  • Regulatory Strategy and Operations
  • Commercialization, Market Access and Reimbursement

Plenary Sessions Include:

  • Scientific and Medical Challenges: Molecular and Cellular Therapies for Neurodegenerative Diseases
  • Clinical Development: Design Concepts and Challenges for Rare Neurodegenerative Diseases
  • Ethics: Ethical Framework Supporting Gene Transfer in Vulnerable Populations
  • Patient Perspective: Fulfilling Patient Needs and Expectations of Gene Transfer

Panel Discussions Include:
Scientific Challenges

  • Lessons Learned in AAV Gene Therapy Translation
  • Transgene Expression
  • From Bench to Launch
  • Accelerating Clinical Development

Ethical Considerations for Clinical Development

  • Evidence Supporting for FIH Pediatric Clinical Studies
  • Future of Expanded Access Programs for Pediatrics
  • Prioritization of Conditions/Populations for Novel Therapies
  • Identification/Consent of Children with Neurodegenerative Disorders
  • Commercialization Aspects of Gene Therapy

Fulfilling Patient Needs and Expectations

  • Perspectives from Parents Project for Muscular Dystrophy – Pat Furlong
  • Perspectives from Beyond Batten Disease Foundation (BBDF) – Craig Benson


Please join us for an action-packed day! Registration is $110 for BioNJ Members and Affiliate Members and $150 for Future Members.

Thank You to Our Conference Sponsors 


March 7, 2019
8:00 am - 4:15 pm
Event Category:


Amicus Therapeutics
1 Cedar Brook Drive
Cranbury, United States
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