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Conference Day Two

Thursday, December 12

8:30 Chair's Opening Remarks

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

Optimizing Transduction Efficiency & Measuring Target Engagement

8.45 Translating Preclinical Responses: Addressing the Challenges of Gene Therapy

Carl Morris
Chief Scientific Officer
Solid Biosciences

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

Joyce Lo
Scientist II
Biogen

9.45 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

10.15 Morning Refreshments & Networking

Improving Preclinical Models for Calculating Dosage & Testing Toxicity

10.45 Evaluating In Vivo Pharmacology Studies for Neurological Gene Therapies

Martin Citron
Vice President, Neuroscience Research
UCB Pharma

11.15 Mastermind Discussion: Discussing Current Preclinical Animal Models for Neurological Disorders

This mastermind session will host interactive discussions amongst your tables and allow you to share your opinions with your peers on the value of using in vivo models for preclinical gene therapy research.

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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?

12.00 Lunch & Networking

Mitigating Immunogenicity & Dosage Challenges for Optimal Efficacy

1.00 Immunogenicity of AAV - Implications for CNS Gene Transfer

Klaudia Kuranda
Immunology Leader
Spark Therapeutics

1.30 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

12.00 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

Carl Morris
Chief Scientific Officer
Solid Biosciences

Boris Gorovits
Senior Director
Pfizer

2.45 Afternoon Networking & Refreshments

Safeguarding the Future of Gene Therapy Patients

3.15 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

Gavin Corcoran
Chief Research & Development Officer
Axovant

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

4.00 Chair’s Closing Remarks

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