8:00 am Online Registration & Virtual Coffee Networking


9:00 am 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

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!


11:30 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

11:50 am Utilising Rational Vector Design for Neurological Targets

  • Omar Khwaja Chief Medical Officer & Head, R&D, Voyager Therapeutics


  • Utilising pre-existing knowledge of disease biology to engineer optimised vectors
  • Highlighting specific considerations for cell-specific targeting in the brain
  • Walking through a case study of vector design for a neurological disorder

12:10 pm 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

12:30 pm Live Discussion & Question Time

1:00 pm Lunch & 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?


2:00 pm Convection Enhanced Delivery – Exploring Novel Devices for Gene Therapy Delivery

  • Miroslaw Zabek Chair of Neurosurgery Department Neurotherapy Center, Brodno Hospital


  • 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

2:20 pm 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

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 Reserved for Atuka

3:15 pm Live Discussion & Question Time

  • Miroslaw Zabek Chair of Neurosurgery Department Neurotherapy Center, Brodno Hospital
  • Sander van Deventer Executive Vice President, Research & Product Development, uniQure
  • Toby Ferguson Vice President, Head of Neuromuscular Development Unit, Biogen

3:45 pm Afternoon Break & PANEL DISCUSSION: Rare vs Common Neurological Diseases


  • 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?


4:45 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

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


  • 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 End of Summit & Virtual Happy Hour Begins


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.

7:15 pm End of Virtual Happy Hour