*All times listed are in EST

Gene Therapy for Neurological Disorders Landscape

7:50 am Chair’s Opening Remarks

  • Chris Tarapata Vice President, Platform Lead, Device Development, Akouos

8:00 am Gene Therapy for the Treatment of Autosomal Dominant Alzheimer’s Disease


  • The role of presenilin in ADAD and rationale for a gene replacement approach
  • Preclinical proof-of-principle data in mouse models and human-derived ADAD mutation-bearing cell lines
  • Evaluating expression and biodistribution in nonhuman primates
  • Phase 1/2 clinical trial plans

8:30 am VIRTUAL: Developing Gene Therapy Approaches for Severe & Rare Neurological Disease: the Relevance of Payload Design

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


Talk details to follow

9:00 am Keeping Up to Date with Ongoing Trials in Gene Therapy for Neurological Disorders


  • Overview of the current active clinical and preclinical landscape within gene therapies for neurological disorders along with key regulatory milestones
  • Approval and discontinuation lifecycles
  • Analysis into the disease landscape within neurological disorders, comparing rare vs non-rare disease indications

9:20 am Target Specific, Tailored Catheter Geometry: Facilitating Optimum Catheter Placement & Associated Infusion Performance

  • Owen Lewis Technical Manager, Drug Delivery Device Research & Product Development, Renishaw


  • How catheter design can positively affect versatility for both catheter placement and infusion distribution characteristics.
  • Utilising the presence of the infusion reflux phenomena to influence functional performance of the intraparenchymal infusion mechanism. A design attribute that enables the manipulation of a desired distribution pattern to best-fit the chosen target structure.
  • Using a common platform for multiple indications: One-off, acute infusions through standard connectors or long-term chronic, intermittent reaccess infusions via a novel transcutaneous port

9:50 am Panel Discussion: Translating from Rare to Common Neurological Disorders

  • Gregory LaRosa Vice President, Head of Scientific Research & Development, Rare Disease RU, Pfizer
  • Mathew Pletcher Senior Vice President, Gene Therapy , Astellas
  • Eliseo Salinas Chief R&D Officer, Passage Bio
  • Suyash Prasad Chief Medical Officer & Head of Research & Development, Taysha Gene Therapies
  • Eric Schaeffer Chief Scientific Officer, Paros Bio

10:30 am Morning Break

Effectively Demonstrating Disease Improvement

11:20 am Challenges to Implementing & Translating Biomarker Strategies in Gene Therapy for Neurodegenerative Diseases

  • Li Chin Wong Director, Bioanalytical & Biomarker Development, Prevail Therapeutics


  • Translating biomarker strategies for gene therapy for neurodegenerative diseases programs presents unique challenges
  • Efficacy studies in mice usually are not performed in translatable matrices
  • Safety studies in mice or NHP usually do not reflect response in humans
  • Snapshot of some possible ways to navigate issue

11:50 am An Exploratory Approach to Survey the Mouse Genome for New Candidates in Neurodegeneration

  • Zhiying Li Director, Life Science and Technology, GemPharmatech


  • Are wild mice smarter or fitter?
  • What can consomic strains help us learn?

12:00 pm VIRTUAL: Simoa Technologies for Ultrasensitive Biomarker Detection


  • Understanding on the 1000x fold sensitivity improvement over traditional
    immunoassay technologies
  • Simoa quantification of low abundance proteins, minimally invasive low volume
    samples, and better stratification between comparator groups
  • A deep-dive on some of Quanterix’ top technology including the SP-X, SR-X and HD-X
  • How critical biomarker detection is applicable to a wide range of therapeutic areas
  • Assay development and custom services

12:10 pm A Pre-Clinical Perspective on Genetic Therapeutic Approaches for CNS Targets

  • Sue Browne Vice President, Research, Passage Bio


  • The challenges, and opportunities, of modulating CNS targets
  • Expanding beyond rare monogenic CNS disorders
  • Keeping clinical translatability at the core of programs

12:40 pm Lunch Break

Clinical Gene Therapy Delivery to the CNS

1:40 pm Optimizing Intraparenchymal Delivery of Therapeutics


  • Patient-specific measures of infusion coverage of regions of interest.
  • Individualizing parameters based upon anatomy and target.
  • Future tools to standardize dosage and coverage

2:10 pm VIRTUAL: Translation from Animals to Humans: Surgical Delivery

  • Paul Larson Professor & Vice Chair of Neurological Surgery , The University of California San Francisco


  • Exploring surgical device considerations for gene therapy
  • Discussing current surgical protocol considerations
  • Walking through regulatory agency approval strategies

2:40 pm RoundTable Discussion: Advancing Gene Therapy for CNS Disorders

  • Chris Tarapata Vice President, Platform Lead, Device Development, Akouos
  • Monica Bennett Head of Gene Therapy Unit, Neuroscience, Novartis Institutes for BioMedical Research (NIBR)
  • Marie-Laure Nevoret Senior Clinical Development Lead, REGENXBIO


1. Discovering Novel Vectors for CNS Targets

2. Utlizing Preclinical Models & Biomarker Strategies

3. Overcoming Clinical & Regulatory Challenges

4. Translating Devices for Targeted Delivery to the CNS

3:15 pm Afternoon Break

Collaborating to Advance Neurology Pipelines & Deliver Value to Patients

3:40 pm Finding the Value for a Parkinson’s Patient

  • Brian Fiske Co-Chief Scientific Officer, The Michael J Fox Foundation


Talk details to follow

4:00 pm Optimizing Gene Therapy Development for Rare Disease Through Collaborations Between Industry & Patient Organizations

  • Amanda Haidet-Philips Senior Director, Translational Medicine & Ultra-Rare Diseases, Novartis Gene Therapies


  • Reviewing the landscape for collaboration strategies
  • Providing examples of successful collaborations within the neurological space
  • Highlighting current roadblocks for rare and ultra-rare disease gene therapy development and discuss how collaboration can help to overcome

4:20 pm Panel Discussion: Encouraging Collaborations to Deliver Value for Neurological Disorders Patients

  • Brian Fiske Co-Chief Scientific Officer, The Michael J Fox Foundation
  • Amanda Haidet-Philips Senior Director, Translational Medicine & Ultra-Rare Diseases, Novartis Gene Therapies

5:10 pm Chair’s Closing Remarks

(V) = Speaker Presenting Virtually