CONFERENCE DAY TWO

8:20 am Registration & Morning Coffee

9:20 am Chairs Opening Remarks

OVERCOMING DISTRIBUTION CHALLENGES ASSOCIATED WITH GENE THERAPIES & THE CNS

9:30 am Discussing AAV-ligand Conjugates for Improved Efficacy & Distribution in CNS

Synopsis

  • Discussing the challenges of deep brain delivery and distribution of gene therapies
  • Overcoming these challenges with the use of AAV-ligand conjugates
  • Improving efficacy of gene therapies in the CNS

10:00 am Roundtable Discussion: From Bench to Clinic: Discussing Best Pre-Clinical Strategies to Successfully Gain IND Approval

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

Synopsis

  • Comparing the use of mouse and non-human primate models for improved translatability to the clinic
  • Looking into capsid design for optimal delivery
  • How do mouse and non-human primate models correlate to clinical trial design

10:30 am Morning Refreshments & Networking

11:00 am An Overview of the Neurochase Delivery Technology: Targeted Delivery of CNS Therapeutics

  • Steven Gill Founder & Chief Medical Officer, Neurochase

Synopsis

  • An opportunity for those keen to learn more about a scalable, targeted delivery approach for CNS gene therapy delivery

SPECIFYING DISEASE SPECIFIC CLINICAL TRIAL CHALLENGES FOR IMPROVED CLINICAL OUTCOMES

11:30 am Determining Appropriate Clinical Endpoints for Parkinson’s Disease Clinical Trials

Synopsis

  • Outlining currently used clinical endpoints and where limitations lie
  • Discussing ideas for novel endpoints
  • Disease specific endpoint selection for Parkinson’s disease

12:00 pm Gene Therapy Strategies for CNS Diseases

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

EXPANDING OPPORTUNITIES FOR SUCCESSFUL GENE THERAPY DELIVERY ACROSS THE BLOOD BRAIN BARRIER

12:30 pm Lunch Break

1:30 pm Selecting the Most Appropriate Disease Specific Capsids for your Gene Therapy Product

  • Anindya Sen Executive Director - Research & Development & Prevail Therapeutics, Eli Lilly & Co.

Synopsis

  • Outlining the landscape of research vectors
  • Utilising clinically approved vectors for delivery to the CNS
  • Looking at insights into the future landscape of gene therapy for neurological disorders

2:00 pm Highlighting the Benefits of Using Hematopoietic Stem Cell Gene Therapy for Delivery to the CNS

  • Laura Campbell Vice President, Clinical Development, Orchard Therapeutics

Synopsis

  • Outlining options for delivery to the CNS for treatment of neurological disorders
  • Challenges in CNS delivery and discussing delivery to patients
  • The future of HSC gene therapy

ADVANCING STAKEHOLDER CONNECTIVITY TO GET GENE THERAPIES FOR NEUROLOGICAL DISORDERS TO PATIENTS FASTER

2:30 pm Navigating Regulatory Challenges in Patient Selection for Clinical Trials in Europe

Synopsis

  • Discussing cross-border patient selection and the challenges in finding appropriate patients when working with rare diseases
  • Cross-functional relationships between European countries will aid in the development of gene therapies for rare diseases
  • Outlining best strategies for patient selection when working with rare diseases

3:00 pm End of Conference Day Two – Refreshments & Networking