8:00 am Sign In & Familiarise Yourself with the Digital Platform

8:30 am European Experience with Gene Therapy in Neurological Conditions


  • Key learnings from ATMPs assessed in the EU
  • Challenges of extrapolations: from study population to final indication
  • Challenges of externally-controlled confirmatory studies


9:00 am Case Study: Direct Injection into the Brain via Intraparenchymal Delivery


  • Walking through the development of a gene therapy administrated with intraparenchymal delivery
  • Deciding injection site and addressing vector spread challenges
  • Identifying other promising candidates using this route
  • Addressing expertise and instrumentation capacity and cost challenges

9:20 am Translatable Biomarkers in Gene Therapy for Huntington Disease: Learnings from Pre-Clinical Studies


  • Finding adequate efficacy/response biomarkers in neurodegenerative disorders and discussing the challenges associated with this
  • Testing novel approaches in adequate models of disease and tailoring biomarkers for gene therapies
  • Addressing candidate biomarkers to follow-up efficacy of a gene therapy for Huntington Disease
  • Highlighting the experience learnt from preclinical models, ranging from biofluid biomarkers to imaging modalities

9:40 am Case Study: Linking Biological Understanding to Determine Optimal Dose


  • Linking dosage to what is known about the disease and the level of gene expression required
  • Addressing translational challenges of extrapolating animal dosage predictions and demonstrating optimal dose
  • Demonstrating a good mechanistic route between dose, mechanisms of action and endpoints. How precise can we be with our dosing?

10:00 am Live Discussion & Question Time

10:30 am Morning Refreshments & Virtual Speed Networking


Grab a quick cup of tea or coffee from the comfort of your own kitchen and jump straight into your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!

10:50 am Explore the Exhibition Booths & Time for Private Calls


11:10 am Assessing Different Vector Types for CNS Disorders & Promoter Choice


  • Reviewing different types of gene therapy and their suitability for neurological disorders
  • Discussing the properties of vector design and novel gene control mechanisms
  • Exploring appropriate promoter choice for CNS indications

11:30 am Direct to CNS Gene Therapy for Lysosomal Storage Diseases: Translational Aspects


  • Routes of delivery of AAV vectors to the CNS
  • Considerations about target engagement in lysosomal storage diseases
  • Examples from Lysogene’s MPS IIIA and GM1 gangliosidosis gene therapy programs

11:50 am Overcoming AAV Immunogenicity & Redosing Challenges

  • Nicholas Buss Senior Director Preclinical Development, REGENXBIO


  • Addressing immunogenicity with direct injection into the CNS
  • Exploring redosing challenges for neurological patients
  • Measuring immune responses

12:10 pm Live Discussion & Question Time

12:30 pm 15 Minute Break

12:45 pm PANEL DISCUSSION: Navigating the Regulatory Challenges for Neurological Gene Therapy Development


  • Exploring standardisation of approaches in gene therapy studies
  • Assessing geo differences for regulatory challenges
  • Reviewing new FDA guidelines and understanding Europe regulators point of view
  • How do regulators view the development of gene therapies for ultra-orphan indications?

1:10 pm Lunch, Private Chat Time & Explore Virtual Exhibition Booths


1:40 pm Convection Enhanced Delivery – Exploring Novel Devices for Gene Therapy Delivery


  • Exploring the state-of-the-art technical improvements for gene therapy delivery
  • Introducing the latest MRI-friendly cannulas and needles
  • Discussing the common challenges with age of patient, tech transfer and scaling

2:00 pm Targeted Gene Therapy for Rare CNS Diseases

  • Matthew Klein Global Head of Gene & Mitochondrial Therapies, PTC Therapeutics


  • Overview of targeted gene therapy strategy
  • Lead programme update: AADC deficiency
  • Pipeline of gene therapy products

2:20 pm Data-Driven Evolution of MRI-Guided Delivery for Gene Therapy

  • Paul Larson Professor & Vice Chair of Neurological Surgery; Chief of Neurosurgery, University of California San Francisco; San Francisco VA Medical Center


  • Shortcomings of traditional stereotactic techniques for gene therapy
  • Common sources of off-target delivery
  • Application and benefits of real-time MRI guidance for delivering gene therapy

2:40 pm Addressing the Challenges of Indirect Gene Therapy Administration

  • Toby Ferguson Vice President, Head of Neuromuscular Development Unit, Biogen


  • Evaluating cisterna magna and intrathecal administration and the clinical challenges
  • Reviewing the IV administration data for gene therapy for neurological disorders
  • Exploring the current clinical data for most appropriate administration routes depending on indication

3:00 pm Live Discussion & Question Time

  • Miroslaw Zabek Chair of Neurosurgery Department Neurotherapy Center, Brodno Hospital
  • Toby Ferguson Vice President, Head of Neuromuscular Development Unit, Biogen
  • Matthew Klein Global Head of Gene & Mitochondrial Therapies, PTC Therapeutics
  • Paul Larson Professor & Vice Chair of Neurological Surgery; Chief of Neurosurgery, University of California San Francisco; San Francisco VA Medical Center

3:30 pm 10 Minute Break

3:40 pm PANEL DISCUSSION: Rare vs Common Neurological Diseases

  • Krystof Bankiewicz President & Chief Executive Officer, Brain Neurotherapy Bio, Inc.
  • Petra Kaufmann Senior Vice President Translational Medicine & Clinical Development, AveXis
  • Claire Gelfman Vice President, Pharmaceutical Development, Adverum Biotechnologies


  • Discussing the current clinical programmes and review of approved candidates – challenges and successes in rare neurological disorders
  • Evaluating the opportunity for gene therapy for neurodegenerative disorders such as Parkinson’s Disease and Alzheimer’s Disease
  • Considering route of administration and patient population size, is gene therapy a viable option for Alzheimer’s Disease and Parkinson’s Disease?

4:10 pm Afternoon Break & Time to Explore


4:30 pm Overcoming the Translational Challenges with Animal Models


  • Exploring biodistribution studies, large animal selection criteria and accounting for species limitations
  • Discussing models available for CNS diseases and the value they provide – small to large
  • Modelling endpoints, dose prediction and PKPD in the brain

4:50 pm Atuka – a Neuroscience-Focussed CRO Providing Gene Therapy Solutions in Rodents & Non-Human Primates


  • Biodistribution studies following administration of gene vectors to the periphery,
    CSF or directly to brain
  • Non-GLP toxicology / efficacy assessments – in-life and post mortem
  • Collection of peripheral and central regions of interest
  • Transgene and DNA expression analyses to evaluate distribution
  • Biomarker assessments to monitor target engagement

5:05 pm Exploring Rare Disease Endpoints to Detect Clinical Efficacy

  • Petra Kaufmann Senior Vice President Translational Medicine & Clinical Development, AveXis


  • Navigating clinical endpoints with little regulatory precedent for rare disease clinical trials
  • Building on natural history datasets for trial planning
  • Highlighting SMA case study examples

5:25 pm The Evolution of Gene Therapy for Neurological Disorders


  • Can gene therapy influence underlying neuropathological changes associated with CNS disorders?
  • Reviewing the importance of gene delivery and how it has evolved over the decades
  • Gene therapy, over the fence and into the commercialisation forest – how to scale it up?

5:45 pm Live Discussion & Question Time

6:15 pm Virtual Speed Networking Session


The time of day we’ve all been waiting for! Grab a beverage of your choice from your home bar and use this final networking opportunity to follow up on connections made earlier in the day, ask that burning question to a speaker whose talk got you thinking and forge relations that could lead to your next collaboration.

6:45 pm End of Summit