9:00am Chair Introduction

Kim Raineri, Chief Technology Officer, Aspen Neuroscience

9:05am Presentation: The Potential of Gene Therapy Delivery to Transform the Lives of People Suffering from Neurological Disorders

• The impact of gene therapies – a personal motivation to help transform lives
• The urgent, unmet need in neurological disorders – a patient’s story
• AviadoBio’s unique heritage, delivery approach, platform and pipeline

Lisa Deschamps, Chief Executive Officer, AviadoBio

9:20am Presentation: Autologous iPSC-Derived Neuron Replacement for Parkinson’s Disease

• Pre-clinical characterization of patient-specific neurons
• PluriTest and NeuriTest: A Genomic strategy for characterizing cell products

Andrés Bratt-Leal, Co-Founder, Senior Vice President, Research & Development, Aspen Neuroscience

9:35am Presentation: Developing Gene Therapy Approaches for Rett Syndrome, a Severe & Rare Neurological Disease: the Relevance of Payload Design

• Rett Syndrome is an X-linked, severe neurodevelopmental disorder with only supportive treatments
• Given that females affected by Rett are mosaics, and the protein if overexpressed is toxic, a novel approach to regulating the output of protein from the AAV construct has been developed
• Preclinical data from this construct is supportive of moving into clinic

 Suyash Prasad, Chief Medical Officer and Head of Research & Development, Taysha Gene Therapies

9:50am Presentation: MultiStem® Cell Therapy for Neurological Indications

• Athersys is developing MultiStem®, an allogeneic adult bone marrow-derived cell therapy, for the treatment of critical care conditions
• Extensive clinical safety and preclinical efficacy data across multiple disease and injury models supports the ongoing MASTERS-2 Phase 3 pivotal clinical trial in acute ischemic stroke

Sarah Busch, Vice President, Regenerative Medicine, Athersys

10:05am Closing Panel with Q&A

With all session participants

Joined by: 

Michael Lawlor, Professor, Medical College of Wisconsin