*All times listed are in EST

WELCOME TO GENE THERAPY FOR NEUROLOGICAL DISORDERS 2020

8:00 am Chairs Opening Remarks

8:15 am Exploring Genomic Medicine Approaches for Neurological Disorders

  • Amy Pooler Vice President, Neuroscience, Sangamo Therapeutics

Synopsis

• Highlighting zinc finger protein-mediated gene editing and gene regulation

8:35 am Market Trends in the Use of Nucleic Acid Technologies to Treat Neurological Disorders

  • Vanessa Almendro Head of Strategy & External Innovation, Eisai Center for Genetics Guided Dementia Discovery (G2D2)

Synopsis

• Focusing on how novel technologies in the nucleic acid space (gene therapies, ASOs, gene editing) will shape the market in neurological indications
• Exploring the use of these technologies in large indications (e.g, Alzheimers) compare to rare diseases
• How this supposes a paradigm shift at so many levels (for industry, CMOs, practitioners, payers and regulatory agencies)

8:55 am Future Development: What Can be Learnt from Approved Products?

  • Petra Kaufmann Senior Vice President, Clinical Development & Translational Medicine, AveXis

Synopsis

• Exploring the successes seen in SMA and ALS
• Discussing both monogenic and multigenic disorders

9:15 am Q&A Panel Discussion

  • Amy Pooler Vice President, Neuroscience, Sangamo Therapeutics
  • Petra Kaufmann Senior Vice President, Clinical Development & Translational Medicine, AveXis
  • Vanessa Almendro Head of Strategy & External Innovation, Eisai Center for Genetics Guided Dementia Discovery (G2D2)

9:45 am Speed Networking

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own kitchen and jump straight into your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!

10:30 am Morning Break

KEY INDUSTRY UPDATES FROM THE CLINIC

11:00 am Comparison of AAV Distribution & Expression Across Preclinical Species

Synopsis

• Mouse CRISPR approach to study expression of AAV serotypes across CNS cell types
• Comparison of AAV serotypes in NHP following CSF injections
• Comparison of ASO and AAV activity in mouse

11:20 am Clinical Progress in Rare Monogenic Neurological Disorders

Synopsis

• Recent clinical update for PBGM01 trials
• Learning lessons from smaller patient populations to apply to future research

11:40 am Moving into Common Neurological Disorders: Parkinson’s Disease

Synopsis

• Reviewing recent clinical program in Parkinson’s Disease
• Discussing challenges associated with more common disease populations

12:00 pm Q&A Panel Discussion

12:30 pm Lunch & Networking

VECTOR DESIGN & DISCOVERY FOR THE CNS

1:00 pm Evaluation of AAV Capsids & Routes of Administration for CNS Gene Therapy Applications

Synopsis

• AAV-mediated gene transfer to various regions of the brain and spinal cord remains a significant challenge in the development of successful gene therapies for neurological disorders
• Biodistribution of known serotypes and novel capsids using various routes of administration including intravenous, lumbar intrathecal, intracisternal magna, intracerebroventricular and direct parenchymal injections will be presented
• Strategies to further understand cell-type specific transduction, immunological issues, and other barriers to CNS gene therapy will also be discussed

1:20 pm Gene Therapy for Glut1 Deficiency Syndrome: Upregulation of Cerebral Glucose at the Blood-Brain Barrier

Synopsis

• Glucose transporter 1 Deficiency Syndrome (Glut1 DS) is a neurodevelopmental genetic disorder caused by haploinsufficiency of SLC2A1 gene encoding Glut1
• There is currently a high unmet need for a long-term therapy that does not bypass the utilization of glucose as an energy source in the brain
• Demonstrating that rAAV9 mediated delivery of Glut1 in neonatal mice restores the function of the deficient Glut1 in the Glut1 DS mouse model

1:40 pm Q&A Panel Discussion: Vector Engineering & Selection

Synopsis

• How do you choose the vector? Optimal
• Engineer the vector?
• Reviewing vectors suitable for CNS targets
• Discussing vector library data
• Evaluating promoter choices

2:00 pm Afternoon Break

MANUFACTURING & REGULATORY CHALLENGES

2:30 pm Gene Therapy Manufacturing: A Translational Challenge Panel Discussion

  • John Pieracci Director, Gene Therapy Process Development, Biogen

3:00 pm Biotech Quick Fire Round: Introducing the New Players in Gene Therapy for Neurological Disorders

3:30 pm Chairs Closing Remarks

3:45 pm Scientific Poster Session & Virtual Networking

5:00 pm End of Day One