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
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
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
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
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