Conference Day Two

Thursday, December 12

9:00 Chair's Opening Remarks

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

Optimizing Transduction Efficiency & Measuring Target Engagement

9.15 Novel AAV-delivered Gene Silencing Technologies Targeting the CNS: Imaging of miRNAs & Therapeutic Efficacy in Huntington’s Disease & SCA3

Sander van Deventer
Executive Vice President, Research & Product Development
UniQure

9.45 Developing a CRISPR Tool to Facilitate Quantification of AAV Transduction in the Mouse CNS

Joyce Lo
Scientist II
Biogen

10.15 Morning Refreshments & Networking

Improving Preclinical Models for Calculating Dosage & Testing Toxicity

11.15 Evaluating In Vivo Pharmacology Studies for Neurological Gene Therapies

Martin Citron
Vice President, Neuroscience Research
UCB Pharma

11.45 Panel Discussion: Discussing Current Preclinical Animal Models for Neurological Disorders

  • What value do rodent studies provide?
  • Should large mammal disease models be more readily available?
  • Do animal models make any sense at all in this field except for safety studies?
  • Are organoid models better? Are patient-derived neurons better?

Moderator:

Panelists:

omar khwaja
Emily Hickey
Martin Citron - UCB
dylan yao head shot

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

Emily Hickey
Chief Operations Officer
Biomere

Martin Citron
Vice President & Head, Neuroscience
UCB Pharma

Dylan Yao
Chief Scientific Officer & Senior Vice President
JOINN Laboratories

12.15 Lunch & Networking

Mitigating Immunogenicity & Dosage Challenges for Optimal Efficacy

1.15 Immunogenicity of AAV - Implications for CNS Gene Transfer

Klaudia Kuranda
Immunology Leader
Spark Therapeutics

1.45 Finding the Right Patient: The Balance Between Immunogenicity, Safety & Efficacy

Mark Milton
Global Head, Pharmacokinetic Sciences Ophthalmology Therapeutic Area & Department Lead for Gene Therapies
Novartis

2.15 The Immunosuppression Debate

• How do we need to address immunogenicity for neurological disorders?

• Is immunosuppression a safe and viable option?

• Considering pre-existing immunity and re-dosing, how can we navigate the risks and rewards?

Mark Milton
Global Head, Pharmacokinetic Sciences Ophthalmology Therapeutic Area & Department Lead for Gene Therapies
Novartis

Boris Gorovits
Senior Director
Pfizer

2.45 Afternoon Networking & Refreshments

Safeguarding the Future of Gene Therapy Patients

3.15 Trial Design Considerations for Rare Diseases

Petra Kauffman
Vice President, R&D Translational Medicine
AveXis

3.45 Panel Discussion: Discussing the Unknown Long Term Safety Issues of Gene Therapies

  • Considering the success seen with neurological gene therapies and the potential for the future, are  the current long-term safety regulations correct?
  • Where can improvement be made on gene therapy safety precautions?
  • What are the major risk factors to be considering for the gene therapy patients?

Gabriele Proetzel
Director, Neuroscience External Research
Takeda

Greg LaRosa
Senior Vice President & Head of Scientific Research, Rare Disease Research Unit
Pfizer

Timothy MacLachlan
Executive Director, Global Head of Biologics Safety Assessment
Novartis

4.30 Chair’s Closing Remarks

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