8:30 am
Check In & Coffee


Workshop A

9:00 am CNS Targeted Vectors: Development & Delivery


  • Verifying the use of novel capsids in a large brain model
  • Harnessing the latest technologies to ensure broad distribution of gene therapies in the brain
  • Delivering CNS gene therapies to larger patient populations using optimized delivery techniques and vectors
  • Leveraging Intracisternal injections to achieve higher efficiency and broad distribution
  • Discussing the interplay between delivery devices and up and coming novel capsids


Workshop B

9:00 am Advancing Appropriate Models for Translational Studies in Human Brains

  • Melvin Rincon Principal Scientist - Gene Therapy, UCB
  • Darin Falk Vice President, Discovery and Early Preclinical Development, Neurogene


  • Analyzing the successful validation of a gene therapy in a rodent animal model
  • Deep diving into successfully transitioning from rodent models to NHP studies to prove safety and appropriate biodistribution in large brain organisms
  • Using human cells to scale dosage before conducting NHP studies
  • Discussing humanized mouse models when modelling CNS gene therapies
  • Understanding the differences in AAV tropism between rodent models and large animal models

12:00 pm
Lunch & Networking

Workshop C

1:00 pm Advancing Gene Editing Technologies to Treat CNS Disorders

  • Perry Spratt Program Director - In Vivo Preclinical Programs, Regel Therapeutics


  • Reviewing the current state of play for CNS-directed gene editing therapies
  • Gene editing techniques to improve gene editing efficiency in the brain
  • Debating viral vs non-viral approaches when delivering gene editing technologies to the brain
  • Minimizing potential in vivo toxic effects of gene editing machinery to the brain
  • Articulating the challenges surrounding fitting gene editing machinery into a vehicle that is deliverable to the CNS

Workshop D

1:00 pm Understanding the Regulatory Framework for CNS Gene Therapies

  • Laura Pisani Senior Director - Medical & Central Nervous System Clinical Development Lead, REGENXBIO


  • Understanding the importance of acknowledging the regulatory framework for gene therapies early in development
  • Discussing the advantages and disadvantages of seamless clinical trials and standard clinical trials for rare neurological conditions
  • Diving into the difference in requirements for setting up clinical trials for gene therapies in different countries
  • Rethinking regulatory framework when developing a CNS-directed gene therapy for a larger patient population
  • Tackling the regulatory views of the development of novel BBB-penetrant capsids

4:00 pm
Close of Workshop Day