8:00 am Check- In & Morning Coffee
8:50 am Chair’s Opening Remarks
REVIEWING CNS TARGETING GENE THERAPY PROGRAMS IN THE CLINIC TO LEARN FROM SUCCESSES & FAILURES TO TURBOCHARGE DEVELOPMENT
9:00 am Navigating New Clinical Data to Hone the Current State of Play in the Industry & Implement Learnings onto Your Gene Therapy
Synopsis
- Evaluating clinical data to take back the learnings from it
- Translating clinical outcomes into your gene therapy development
- Learning from clinical trials how to best address challenges related to safety and immunogenicity
9:30 am Identifying the Reasons Behind Failures in Clinical Trials to Learn from Them & Update Preclinical Testing & Clinical Trial Design
Synopsis
- Analyzing recent clinical trial failures to determine the cause
- Understanding how preclinical testing can be updated to prevent clinical failures
- Turning failures into successes by learning from errors and strengthening clinical trial design
10:00 am Roundtable Discussion: Analyzing a Commercialized CNS Targeting Gene Therapy Program to Understand How to Successfully Take Your Product from Bench to Bedside
Synopsis
- Deconstructing a full development life cycle to commercialization to understand the importance of each step
- Examining the key challenges in each step from discovery to clinical development and manufacturing
- Identifying key commercial insights and data to support your trials
10:30 am Morning Break & Speed Networking
Synopsis
This session is an opportunity to connect with peers and hold in-depth conversations with some of the brightest minds in the gene therapy industry to establish meaningful and lasting relationships
INNOVATING NOVEL VECTOR DEVELOPMENT & OPTIMIZING EXISTING VECTORS TO SUCCESSFULLY CROSS THE BLOOD-BRAIN BARRIER & IMPROVE CNS TARGETING
11:30 am Finding the Needle in the Haystack: Finding the Right Capsid to Successfully Deliver Your Gene Therapy to the Brain
Synopsis
- What are the approaches to achieve a suitable biodistribution in the primate brain
- How can engineered capsids help to achieve suitable biodistribution in the primate brain? What is possible today, and what can we expect in the near future?
- What are some of the opportunities and challenges for moving engineered capsids towards the clinic?
12:00 pm Reprogramming AAV Capsids to Target Human Receptors for Enhanced CNS Delivery
Synopsis
- Optimizing for translation in the clinic should start with capsids interacting with human receptors
- Regulatory guidance in gene therapy and direct FDA feedback across multiple programs outlines preclinical path for human-specific capsid and use of humanized animal models
- Surrogate mouse BBB-crossing capsids can be used in efficacy studies in parallel with distribution studies enabling dose translation between surrogate and clinical capsids targeting human receptors
12:30 pm Improving Likelihood of Clinical Translation by Engineering Novel AAV Capsids that Bind BBB Receptors
Synopsis
- Identified capsids that bind both human and NHP orthologs of receptors expressed in the BBB
- AI-powered approach to further optimize CNS tropism
- Validation of CNS biodistribution in mouse, NHP, and human models
1:00 pm Lunch & Networking
IMPROVING THERAPEUTIC EFFICACY TO CNS DISEASES BY REDUCING OFF-TARGET TOXICITIES & PREVENTING IMMUNOGENICITY
2:00 pm Machine-Guided Design of AAV Capsids for Highly Specific CNS-IV Delivery
Synopsis
- Multi-property optimization of capsid sequences using machine learning
- Demonstrating efficient pan-brain CNS-IV gene delivery and exceptional liver detargeting in adult Cyno monkeys
- Confirming conservation of BBB-crossing mechanisms between NHP and humans
2:30 pm Balancing Durability & Safety to Ensure Efficacy of Therapy Without Inducing Systemic Toxicity
Synopsis
- Exploring the balance between durability and toxicity to correctly dose the patient
- Identifying the correct balance to ensure high efficacy
- Examining the drawback of high toxicity and low efficacy on clinical trial success
3:00 pm Afternoon Networking Break
4:00 pm Roundtable Discussion: Exploring the Effects of Toxicity on the Liver & Other Organs to Understand the True Effect of the Gene Therapy on the Entire Body
Synopsis
- Discuss and learn how you can manage and benchmark toxicity
- Expanding beyond the liver to understand the effects of toxicity on other vital organs
- Reviewing how to control systemic toxicity and not just toxicity to the liver