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
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