*All times listed are in EST
Gene Therapy for Neurological Disorders Landscape
7:50 am Chair’s Opening Remarks
8:00 am Gene Therapy for the Treatment of Autosomal Dominant Alzheimer’s Disease
Synopsis
- The role of presenilin in ADAD and rationale for a gene replacement approach
- Preclinical proof-of-principle data in mouse models and human-derived ADAD mutation-bearing cell lines
- Evaluating expression and biodistribution in nonhuman primates
- Phase 1/2 clinical trial plans
8:30 am VIRTUAL: Developing Gene Therapy Approaches for Severe & Rare Neurological Disease: the Relevance of Payload Design
Synopsis
Talk details to follow
9:00 am Keeping Up to Date with Ongoing Trials in Gene Therapy for Neurological Disorders
Synopsis
- Overview of the current active clinical and preclinical landscape within gene therapies for neurological disorders along with key regulatory milestones
- Approval and discontinuation lifecycles
- Analysis into the disease landscape within neurological disorders, comparing rare vs non-rare disease indications
9:20 am Target Specific, Tailored Catheter Geometry: Facilitating Optimum Catheter Placement & Associated Infusion Performance
Synopsis
- How catheter design can positively affect versatility for both catheter placement and infusion distribution characteristics.
- Utilising the presence of the infusion reflux phenomena to influence functional performance of the intraparenchymal infusion mechanism. A design attribute that enables the manipulation of a desired distribution pattern to best-fit the chosen target structure.
- Using a common platform for multiple indications: One-off, acute infusions through standard connectors or long-term chronic, intermittent reaccess infusions via a novel transcutaneous port
9:50 am Panel Discussion: Translating from Rare to Common Neurological Disorders
10:30 am Morning Break
Effectively Demonstrating Disease Improvement
11:20 am Challenges to Implementing & Translating Biomarker Strategies in Gene Therapy for Neurodegenerative Diseases
Synopsis
- Translating biomarker strategies for gene therapy for neurodegenerative diseases programs presents unique challenges
- Efficacy studies in mice usually are not performed in translatable matrices
- Safety studies in mice or NHP usually do not reflect response in humans
- Snapshot of some possible ways to navigate issue
11:50 am An Exploratory Approach to Survey the Mouse Genome for New Candidates in Neurodegeneration
Synopsis
- Are wild mice smarter or fitter?
- What can consomic strains help us learn?
12:00 pm VIRTUAL: Simoa Technologies for Ultrasensitive Biomarker Detection
Synopsis
- Understanding on the 1000x fold sensitivity improvement over traditional
immunoassay technologies - Simoa quantification of low abundance proteins, minimally invasive low volume
samples, and better stratification between comparator groups - A deep-dive on some of Quanterix’ top technology including the SP-X, SR-X and HD-X
- How critical biomarker detection is applicable to a wide range of therapeutic areas
- Assay development and custom services
12:10 pm A Pre-Clinical Perspective on Genetic Therapeutic Approaches for CNS Targets
Synopsis
- The challenges, and opportunities, of modulating CNS targets
- Expanding beyond rare monogenic CNS disorders
- Keeping clinical translatability at the core of programs
12:40 pm Lunch Break
Clinical Gene Therapy Delivery to the CNS
1:40 pm Optimizing Intraparenchymal Delivery of Therapeutics
Synopsis
- Patient-specific measures of infusion coverage of regions of interest.
- Individualizing parameters based upon anatomy and target.
- Future tools to standardize dosage and coverage
2:10 pm VIRTUAL: Translation from Animals to Humans: Surgical Delivery
Synopsis
- Exploring surgical device considerations for gene therapy
- Discussing current surgical protocol considerations
- Walking through regulatory agency approval strategies
2:40 pm RoundTable Discussion: Advancing Gene Therapy for CNS Disorders
Synopsis
1. Discovering Novel Vectors for CNS Targets
2. Utlizing Preclinical Models & Biomarker Strategies
3. Overcoming Clinical & Regulatory Challenges
4. Translating Devices for Targeted Delivery to the CNS
3:15 pm Afternoon Break
Collaborating to Advance Neurology Pipelines & Deliver Value to Patients
3:40 pm Finding the Value for a Parkinson’s Patient
Synopsis
Talk details to follow
4:00 pm Optimizing Gene Therapy Development for Rare Disease Through Collaborations Between Industry & Patient Organizations
Synopsis
- Reviewing the landscape for collaboration strategies
- Providing examples of successful collaborations within the neurological space
- Highlighting current roadblocks for rare and ultra-rare disease gene therapy development and discuss how collaboration can help to overcome
4:20 pm Panel Discussion: Encouraging Collaborations to Deliver Value for Neurological Disorders Patients
5:10 pm Chair’s Closing Remarks
(V) = Speaker Presenting Virtually
