Post-Conference Delivery Focus Day

November 9, 2023

8:30 am Morning Coffee & Registration

9:20 am Chair’s Opening Remarks

Breaking Boundaries of Gene Therapy with Non-Viral Vector Development for Improved Blood Brain Barrier Penetration

9:30 am Understanding the Opportunities & Limitations of Nano Particles in Gene Therapy Delivery to the CNS

Synopsis

  • Rational design in nanoparticle delivery across the blood-brain barrier for gene therapy
  • Considerations relevant to screening of nanoparticles for improved brain transport
  • Pros and cons of different types of nanoparticles with respect to gene therapy applications

10:00 am Outlining the Current Landscape of Capsid Design for Neurological Gene Therapies

Synopsis

  • Reviewing recent developments in viral vector capsid design, summarizing the recent developments in identifying rodent and primate brain tropic capsids.
  • Assessing gaps development and challenges posed highlighting the lack of in vitro models to accelerate screening of capsids.
  • Debating the challenges posed in choosing capsids with such a broad viral vector revealing data with benchmark capsids, such as AAV9 and AAV PHP.B.

10:30 am Morning Refreshments & Networking

Advancing Viral Vectors & Identifying Successes in Novel Delivery

11:00 am Roundtable Discussion: Expanding Opportunities with Convection Enhanced Delivery Through the CSF

Synopsis

  • Delivering small volumes of gene therapies to targeted regions of the brain quickly with CED
  • Risks and benefits of utilizing CED in direct brain delivery
  • Treating Huntington’s disease with CED

11:30 am Outlining Advancements in AAVs: the Gold Standard of CNS Gene Therapies

12:00 pm Lunch & Networking

Navigating the Blood Brain Barrier with Different Delivery Modalities

1:00 pm Panel Discussion: Identifying Mechanisms of Successful Penetration of the Blood Brain Barrier

Synopsis

  • Outlining advancements in blood brain barrier opening technologies and weighting up Pros and Cons
  • Discovering differences between specific and non-specific facilitation of blood brain barrier crossing
  • Avoiding non-specific transport of potentially toxic compounds across the blood brain barrier and other preclinical safety considerations

1:45 pm Leveraging Focussed Ultrasound to Open the Blood Brain Barrier & Highlighting Limitations

  • Nick Todd Assistant Professor, Brigham and Women's Hospital

Synopsis

  • Discussing mechanisms of action for focused ultrasound-mediated disruption of the blood-brain barrier
  • Opportunities for utilizing this technology to improve delivery of therapeutics to the brain
  • Understanding physiological and technological limitations of focused ultrasound

2:15 pm Underpinning the Potential of Herpes Simplex Virus (HSV) in CNS Gene Therapies

Synopsis

  • Methods for complete HSV vector gene silencing to achieve vector safety and prevent the induction of inflammatory processes
  • Methods to create 40kb of space for very large or multiple transgenes and the development of engineering methods for transgene cassette design and genomic positioning that overcome transgene silencing and provide for regulated, long-term transgene expression in neurons
  • Vector production for application to animal model testing. This gene delivery technology will be broadly useful for the treatment of neurological diseases that require large or multiple genetic interventions

2:45 pm Afternoon Refreshments & Networking

Transforming Standard Routes of Administration for CNS Delivery

3:15 pm Roundtable & Audience Discussion: Weighing up Options of Device Enabled Delivery to the CNS

  • Fergus Quigley Director, Device Development Lead (Early Stage Development), Takeda Pharmaceutical Co. Ltd.

Synopsis

  • Outlining available delivery options (intraparenchymal, ICV, ICM, IT etc.)
  • Understanding Safety and Efficacy of merging route of administrations
  • Opportunities for innovation beyond existing RoA’s

3:45 pm Reversible, Covalent DNA Condensation Approach for Enhanced Non-Viral Gene Delivery to the CNS

Synopsis

  • Outlining work on utilizing covalent DNA condensation as an approach for neurological gene therapy delivery
  • Highlighting the benefits of a non-viral gene therapy approach
  • Discovering opportunities in non-viral delivery to the CNS

4:15 pm End of Post-Conference Delivery Focus Day