CONFERENCE DAY ONE - 21st NOVEMBER 2024
8:00 am Check In & Coffee
8:50 am Chair’s Opening Remarks
Analyzing the Performance of Novel Vectors to Optimize Gene Delivery to the CNS
9:00 am Reprogrammed AAV Capsids to Mediate Brain-Wide Gene Delivery
Synopsis
- Developing vehicles that efficiently deliver genes throughout the CNS
- Engineering an AAV capsid that binds to TfR1
- Enhancing CNS-specific tropism in preclinical mouse models
9:30 am Holy Grail of CNS Gene Therapy: Systemic Administration of a BBB Penetrant Capsid
Synopsis
- Discussing the interaction between the vector and the delivery device to successfully deliver a novel vector to a human brain
- Leveraging the delivery of novel, systemically delivered capsids to treat whole-brain diseases
- Tackling species specific differences in capsid interactions with the BBB
10:00 am Engineering novel CNS AAV capsids with improved likelihood of translating to the clinic
Synopsis
- Identifying capsids that bind both human and NHP orthologs of likely BBB receptors
- AI-powered approach to further optimize CNS tropism
- Validation of novel capsid biodistribution in a large brain model
10:30 am Morning Refreshments & Speed Networking
Synopsis
This session is the ideal opportunity to get face-to-face time with many of the brightest
minds working in the CNS directed gene therapy field and establish new relationships
and connect with old friends.
11:30 am Roundtable Discussion: Treading the Regulatory Path for Novel CNS Capsids
Synopsis
- Identifying the binding receptor for the capsid to cross the BBB
- Discussing the relevant clinical data sets necessary to support a regulatory filing for novel capsids
- Outlining the steps required around how this novel technology can be successfully moved into patients
12:00 pm Developing Capsids and Cargo to Achieve Brain-Wide Epigenetic Regulation for the Treatment of Neurological Disorders
Synopsis
- Engineering and characterization of a novel blood-brain barrier penetrant capsid
- Optimizing cargo for potent and specific epigenetic regulation in the CNS
- Evaluating transduction and target engagement with single cell resolution
12:30 pm Lunch & Networking
Optimizing Translational Studies in CNS Gene Therapies
1:30 pm The Potential for Non-Replicative HSV Platforms in Neurology
Synopsis
- Leveraging the natural neurotropism and retrograde transport ability of HSV for efficient and targeted gene delivery to neurons
- Discussing the safety profile of nrHSV vectors
- Exemplifying in different indications in PNS and CNS
2:00 pm Engineering Capsid Substructure and Cell Specificity
Synopsis
- Massively paralleled, high throughput, unbiased screening of AAV capsids
- Designing capsids to accurately target tissues and reduce off-target effects
- Minimizing the risks of immunogenicity and toxicity to optimize payload delivery to the CNS
2:30 pm Capsid Design to Human Receptors
Synopsis
- Optimizing translation in the clinic should start with capsids interacting with human receptor
- Engineering capsids to interact with human receptors of interest is an attainable and reproducible process that is low cost and achieved in less than 12 months
- FDA guidance in gene therapy outlines preclinical path for human specific compounds and animal use
3:00 pm Afternoon Refreshments & Scientific Poster Session
Optimizing Translational Studies in CNS Gene Therapies
3:30 pm Identifying Novel Promoters for Neuronal Cell Targeting
Synopsis
- Cell type specific promoter identification
- Introducing regulatory elements to prevent off-target effects
- Ensuring highly selective rescue of gene expression