Pre-Conference Workshops

Workshop A: An Introduction to Gene Therapy for Neurological Disorders

Tuesday, December 8 | 8.30am - 11.30am EST | 5.30am-8.30am PST

As gene therapy becomes a more prevalent therapeutic approach for neurological disorders, it is important to be familiar with the common challenges which still exist for gene therapy experts

Attend this workshop to:
• Discuss the basic principles of gene therapy vectors and how these can be applied to neurological indications
• Highlight common challenges for gene therapy vectors
• Introduce different administration routes and their respective advantages and disadvantages

Workshop Leader:

Ronald Crystal - Cornell B&W

Ronald Crystal
Chairman, Department of Genetic Medicine
Weill Cornell Medicine

Workshop B: Reducing the Translational Gap in Neurology

Tuesday, December 8 | 8.30am - 11.30am EST | 5.30am-8.30am PST

The most pressing translation challenge when developing therapeutics for neurological disorders remains the lack of predictable preclinical models available

Attend this workshop to:

• Explore novel in vitro and in vivo models for neurological disorders
• Discover translational strategies for improving clinical predictions
• Look specifically at biodistribution and dosage extrapolation studies

Workshop Leaders:

Jeffrey Kordower - Rush B&W

Jeffrey Kordower
Rush University Medical Center

MugShot(D171213R): Dr. Steven Gray (PhD, Neurology).

Steven Gray
Associate Professor
University of Texas Southwestern Medical Center

Workshop C: Optimizing Vector Engineering for Improved Gene Therapy Delivery to Target

Tuesday, December 8 | 12pm - 3pm EST | 9am-12pm PST

Delivery is the greatest challenge for gene therapies targeting the CNS. Innovative vector technology is being developed to help improve the target engagement and cell specificity of gene therapy delivery

Attend this workshop to:

• Explore the use of vector engineering to improve gene therapy delivery
• Discuss the promotors and regulators required for cell-specific expression
• Debate the use of non-viral vs viral vectors

Workshop Leader:

Melissa Kotterman

Melissa Kotterman
Vice President, Vector Design
4D Molecular Therapeutics

Workshop D: Linking Clinical Endpoints to Gene Therapy Effectiveness

Tuesday, December 8 | 12pm - 3pm EST | 9am-12pm PST

Demonstrating neurological disease improvement in the clinic is challenging. Gene therapy for neurological disorders is particularly hard as the patient populations can be old and slowly progressing for Alzheimer’s and Parkinson’s Disease, or extremely young with rare genetic neurologic disorders. Measuring an effect as a result of altered gene expression and demonstrating this efficacy to regulators

Attend this workshop to:

• Learn how approved gene therapy demonstrated clinical effectiveness to regulators
• Discuss the clinical challenges for gene therapy trials
• Explore novel strategies to detect neuronal damage and clinical improvement

Workshop Leader:

Krystof Bankiewicz - California B&W

Krystof Bankiewicz
President & Chief Executive Officer
Brain Neurotherapy Bio, Inc.

Mark T

Mark Tuszynski
Director, UCSD Translational Neurosciences Institute; Distinguished Professor of Neurosciences
University of California - San Diego


J Fraser Wright
Professor of Pediatrics
Center for Definitive & Curative Medicine, Stanford University School of Medicine