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

  • Ben Deverman Director - Vector Engineering at the Stanley Center for Psychiatric Research, The Broad Institute


  • 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

  • Eric Kelsic Co Founder & Chief Executive Officer, Dyno Therapeutics


  • 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

  • John Reece-Hoyes Senior Director - Head of Vector Biology, Affinia Therapeutics


  • 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


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

  • Eric Kelsic Co Founder & Chief Executive Officer, Dyno Therapeutics


  • 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

  • David Ojala Director, AAV Engineering, Sangamo Therapeutics


  • 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


  • 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


  • 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


  • 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


  • Cell type specific promoter identification
  • Introducing regulatory elements to prevent off-target effects
  • Ensuring highly selective rescue of gene expression

4:00 pm Chair’s Closing Remarks

4:10 pm End of Conference Day One