Conference Day One

Wednesday, December 11

8:15 Chair's Opening Remarks

Omar Khwaja
Chief Medical Officer & Head of Research & Development
Voyager Therapeutics

Gene Therapy: The Promising Future for Neurological Disorders

8.30 Beyond Small Molecules & Protein Therapeutics: New Modalities for Treatment of CNS Diseases

Natasha Penner
Director, Clinical Pharmacology & Pharmacometrics

9.00 Targeted Micro-Dosing Strategy in Gene Therapy to Deliver Clinical Results

Jodi Cook
Head of Gene Therapy Strategy
PTC Therapeutics

9.30 Speed Networking

This session is the ideal opportunity to meet face-to-face with the key thought leaders working in the neurological gene therapy field. Specifically designed to connect you with new contacts from the most active companies in the field, the renowned Speed Networking session will be one of the most valuable hours you spend at GTxN.

10.15 Morning Refreshments

Delivery, Delivery, Delivery: Accessing the CNS

10.45 Evaluating the Barriers to AAV Delivery into the Central Nervous System 

Robert Bell
Associate Research Fellow

11.15 Achieving Therapeutic AAV-based Gene Delivery to the Brain & Nervous System

Omar Khwaja
Chief Medical Officer & Head of Research & Development
Voyager Therapeutics

11.45 Introduction to Atuka

Jonathan Brotchie
Co-Founder, Director & President
Atuka Inc

12.00 Lunch & Networking

Improving Specific Cell Targeting for More Efficient Cell Transduction

1.00 Impact of Capsid Engineering & Synthetic Components to Advance CNS Gene Therapy

Martin Childers
Chief Medical Officer

1.30 Optimizing Targeting & Gene Expression: Defining Disease, Region & Cell Type

Lamya Shihabuddin
Head, Genetic Neurologic Diseases

Improving the Efficiency of Gene Therapy Development

2.00 Panel Discussion: The Gene Therapy Journey in Neurologic Disease: Designing Clinical Programs to Meet Patient Needs

  • Understanding and balancing the burden of gene therapy clinical trials from the patient’s perspective
  • What hurdles can we realistically lessen or eliminate for patients pursuing gene therapies – perspectives from the sponsor, CRO and investigator
  • How to plan for a more patient-centric trial in early development



Untitled design (11)

Carol Satler, M.D., Ph.D.
Vice President, Regional Medical Officer
PPD Biotech

Untitled design (10)

Ira Goodman
Principal Investigator
Compass Clinic

Miller, Tim

Timothy Miller
Vice President & Area Head, Pediatrics & Rare Diseases

2.30 Afternoon Refreshments & Networking

3.30 Aligning Preclinical Research with Current Regulation & Clinical Considerations

David Theron
Prospective & Opportunities Director, CNS

4.00 Achieving Clinical Progress while Establishing Mutually Beneficial Links with Academia

Gavin Corcoran
Chief Research & Development Officer

4.30 Chair’s Closing Remarks

Omar Khwaja
Chief Medical Officer & Head of Research & Development
Voyager Therapeutics

4.45 Scientific Poster Session

The learning and networking continues at the Poster Session, an informal part of the conference agenda, allowing you to connect with your peers in a relaxed atmosphere and continue to forge new, beneficial relationships. You will have the opportunity to present and review presentations displaying new data from preclinical gene therapy research and clinical gene therapy progress in neurological disorders.