Gene Therapy for Neurological Disorders Europe

We’re speaking at the Gene Therapy for Neurological Disorders Europe conference, which will be held as a virtual meeting on July 15, 2020. The conference unites industry leaders to help them overcome translational challenges and successfully bring CNS gene therapy candidates into the clinic.


9:40 am Case Study: Linking Biological Understanding to Determine Optimal Dose
Nathalie Cartier-Lacave, Founder & Chief Scientific Officer, BrainVectis (acquired by AskBio in April 2020)

  • 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?

11:10 am Assessing Different Vector Types for CNS Disorders & Promoter Choice
Michael Roberts, Chief Technology Officer, AskBio, Europe

  • 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

12:45 pm PANEL DISCUSSION: Navigating the Regulatory Challenges for Neurological Gene Therapy Development
Nathalie Cartier-Lacave, Founder & Chief Scientific Officer, BrainVectis
Elena Hernandez Martinez de Lapiscina, Scientific Officer & Clinical Neurologist, Office of Therapies for Neurological & Psychiatric Disorders, European Medicines Agency
Hans-Juergen Fuelle, VP, Head Global Regulatory Strategy, AveXis
Victoria Palmi Reig, Product Lead, Advanced Therapy Medicinal Products, European Medicines Agency

  • Exploring standardization 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?

3:40 pm PANEL DISCUSSION: Rare vs Common Neurological Diseases
Krystof Bankiewicz, President & CEO, Brain Neurotherapy Bio, Inc.
Petra Kaufmann, SVP Translational Medicine & Clinical Development, AveXis
Claire Gelfman, VP, Pharmaceutical Development, Adverum Biotechnologies

  • Discussing the current clinical programs 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?

5:25 pm The Evolution of Gene Therapy for Neurological Disorders
Krystof Bankiewicz, President & CEO Brain Neurotherapy Bio, Inc. (Partnered with AskBio)

  • 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 commercialization forest – how to scale it up?