Cell and Gene Therapy for Neurological Indications

20 Jan 2023
Modalities and Indications Track

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


Andreas Kouri
Senior Vice President, Supply Chain Strategic Operations
Kim Raineri
Chief Technology Officer
Aspen Neuroscience
Lisa Deschamps
Chief Executive Officer
Sarah Busch
Vice President of Regenerative Medicine, Head of Nonclinical Development
Suyash Prasad
Chief Medical Officer and Head of Research & Development
Taysha Gene Therapies
Michael Lawlor
Medical College of Wisconsin