8:45 am Check- In & Morning Coffee
9:20 am Chair’s Opening Remarks
OPTIMIZING TRANSLATIONAL DEVELOPMENT TO GAIN REGULATORY APPROVAL & SUCCESSFULLY MOVE INTO CLINICAL TRIALS
9:30 am Selecting Human-Translatable BBB-Crossing Capsids Using Humanized Mice
Synopsis
- Providing an overview of current strategies for engineering BBB-crossing capsids that target human receptors
- Highlighting recent advancements in differential directed evolution for identifying and optimizing capsids
- Exploring ongoing challenges in the field, including issues of cross-species reactivity and translational barriers
10:00 am Roundtable Discussion: Identifying Biomarkers & Endpoints in Slowly Progressing Neurodegenerative Diseases to Predict Disease Progression & Improve Patient Targeting
Synopsis
- Exploring emerging fluid and imaging biomarkers to get a better understanding of progressing neurodegenerative diseases
- Discussing what endpoints are important to better understand disease progression
- Analyzing the difficulty in understanding disease progression and its effects on clinical and preclinical trials
10:30 am Preclinical Studies for Gene Therapies in Common NDDs: From Human-Based Cellular Through Mouse Models
Synopsis
- Neurodegenerative diseases (NDDs) in aging, on the spectrum of Alzheimer’s and Parkinson’s diseases, have unmet clinical need with no effective and safe disease modifying therapy (DMT)
- Gene-targeted therapies will move the field foward towards precision medicine
- Epigenome editing platform delivered by AAV offers the opportunity for the development of innovative therapeutics for NDDs caused by dysregulation of targeted gene expressionÂ
11:00 am Morning Break & Networking
TRANSLATING LEARNINGS TO ALZHEIMER’S, PARKINSON’S, & OTHER DISEASES TO WIDEN THE TARGET PATIENT POPULATION
12:00 pm Roundtable Discussion: Diving into the Intricacies of Alzheimer’s & Parkinsons to Open Up New Disease Options for Gene Therapy Modalities
Synopsis
- Comparing the differences between diseases affecting paediatric populations and older patients to understand how it affects gene therapy development
- Unpacking the unique pathophysiological mechanisms of Alzheimer’s and Parkinson’s to identify gene therapy targets
- Assessing the challenge with delivery, targeting, and timings of administering the gene therapy
1:00 pm Strategies for Developing Therapies for Rare CNS Disorders
Synopsis
- Building on previous preclinical and clinical data
- Establishing a collaborative network to gain market intelligence
- Overcoming regulatory and financial hurdles to streamline development
1:30 pm Lunch & Networking
2:30 pm Bridging the Gap: Applying Learnings from Gene Therapies Targeting Other CNS Diseases to the Giants of Neurodegenerative Disorders to Widen the Target Patient Pool
Synopsis
- Utilizing learnings from SMA, Huntington’s, and other diseases to understand how to target other neurodegenerative diseases
- Identifying if there are shared biological pathways that can be easily targeted Learn from clinical and preclinical data to inform discovery development for these diseases
UNDERSTANDING THE IMPACT OF EARLY-STAGE DEVELOPMENT ON DOWNSTREAM MANUFACTURING PROCESS TO STREAMLINE THEM & REDUCE OVERALL COSTS
3:00 pm Understanding the Hurdles with Scaling Up & How Vector Technologies Affect it to Prevent Manufacturing Setbacks
Synopsis
- Identifying common scale up challenges associated with vector manufacturing
- Analyzing the influence of vector technologies on manufacturing processes
- Developing strategies in vector development to mitigate manufacturing errors