CONFERENCE DAY ONE

8:00 am Registration & Morning Coffee

8:50 am Chairs Opening Remarks

ADVANCING PRECLINICAL MODELS FOR IMPROVED TRANSLATABILITY TO THE CLINIC FOR NEUROLOGICAL GENE THERAPIES

9:00 am Discussing Advancements in Preclinical Models for CNS Gene Therapies

Synopsis

  • Delving into the most recent advancements and developments in preclinical modelling for gene therapies in the CNS to improve understanding of the current landscape
  • Discussing how to adapt and overcome the limitations in using mice and rat models in preclinical studies for neurological disorders
  • Outlining the current limitations and areas for future development to progress preclinical assessments

9:30 am AAVs for Modelling Parkinson’s Disease in Nonhuman Primates

Synopsis

  • Discussing the use of AAVs in non-human primates for preclinical modelling in Parkinson’s disease
  • Outlining the challenges in modelling AAV in Parkinson’s Disease
  • Dealing with specific challenges related to preclinical modelling for Parkinson’s disease

10:00 am Leveraging Successful Platforms to Accelerate Preclinical Development: DTDS case study

  • Leigh Shaw Senior Vice President, Regulatory Affairs & Non- Clinical GLP Safety/Tox, Bloomsbury Therapeutics

Synopsis

  • Developing approaches to progress CNS programs to clinic faster
  • Discussing strategies to gain regulatory approval
  • Delving into the BGT-DTDS case study

10:30 am Morning Refreshments & Speed Networking

Synopsis

This session is an opportunity to connect with peers working gene therapy for neurological disorders and hold in-depth conversations with some of the brightest minds to establish meaningful and lasting relationships.

UTILIZING IN VITRO MODELING TO SUPPLEMENT YOUR NONCLINICAL DATA PACKAGES

11:30 am Enhancer-AAVs to Express Transgenes in Defined Brain Circuits for Therapeutic Intervention

  • Boaz Levi Associate Investigator, Allen Institute

Synopsis

  • Analysis of detailed genomic datasets to reveal enhancers throughout the brain
  • A discovery platform for enhancer-AAV creation and testing
  • New collections of enhancer-AAVs that express transgenes in defined brain circuits
  • Validation of enhancer-AAVs in the multiple species

OPTIMIZING TARGETING AND DELIVERY TO THE CNS USING NOVEL VECTORS

12:00 pm Outlining the Developmental Landscape of Next Generation Viral Vectors

Synopsis

  • Benchmarking the state of development of novel viral vectors for use in neurological gene therapies
  • Reimagining novel engineered viral vectors for improved targeting and delivery across the blood brain barrier
  • Exploring the future of viral vector delivery of gene therapies to the CNS

12:30 pm Developing Organoids for Nonclinical Development for Gene Therapies of Neurological Indications

  • Fabio Cavaliere Director, Biomodel Platform for Human Research, Achucarro Basque Center for Neuroscience

Synopsis

  • Outlining the advantages of organoids to supplement preclinical data packages
  • Developing organoids for neurological indications, and how these can apply to your nonclinical development
  • Current limitations, and future applications of in vitro modelling of gene therapies for neurological disorders

1:00 pm Lunch

EXPLORING NOVEL GENE THERAPY DELIVERY TECHNIQUES FOR NEUROLOGICAL DISORDERS

2:00 pm Using Interventional Catheter Placement for Improved Delivery of Gene Therapies to the CNS

  • Stéphane Palfi Professor neurosurgery, Portail Assistance Publique-Hopitaux de Paris

Synopsis

  • Discussing how using an MRI-guided surgical catheter placement can improve delivery to the CNS
  • Visualising administration and infusion in real time
  • Improving accuracy, efficiency and safety across a range of neurological Disorders

2:30 pm Optimising Delivery of CNS Gene Therapies using Case Study Led Examples

  • William Gray Professor of Neurosurgery, Cardiff University

Synopsis

  • Hearing about first hand case-study led examples of successful and improved delivery to the CNS and deep brain
  • Overcoming the blood brain barrier in gene therapy delivery
  • Outlining the future of delivery to the CNS for improved safety and efficacy

3:00 pm Improving Delivery Across the Blood Brain Barrier Using Stem Cell Gene Therapy

  • Brian Bigger Chair of Cell and Gene Therapy, University of Manchester

Synopsis

• Delving into advancements in enhancing delivery to the CNS using lentiviral vectors and stem cell gene therapy

• Discussing opportunities in stem cell gene therapy delivery to the CNS and areas of excitement for future development

• Navigating the challenge of the blood brain barrier and how to best help overcome this setback

3:30 pm Poster Session

Synopsis

While the formal presentations of Day One come to an end, the learning doesn’t stop as we enter the Scientific Poster Session. This will serve as the perfect opportunity to showcase your recent work to your peers and allow you to learn and share insights in a relaxed atmosphere.

4:30 pm End of Conference Day One