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?