Day One

Tuesday, December 7

Day Two

Wednesday, December 8

*All times listed are in EST

Gene Therapy for Neurological Disorders Landscape

8:00 am Start of Day

8:30 am 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

Synopsis

Talk details to follow

8:50 am Keeping Up to Date with Ongoing Trials in Gene Therapy for Neurological Disorders

Synopsis

  • 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

9:10 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

9:40 am Morning Break

Next Generation Gene Therapy

10:40 am Challenges to Implementing & Translating Biomarker Strategies in Gene Therapy for Neurodegenerative Diseases

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

Synopsis

  • 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

11:00 am A Pre-Clinical Perspective on Genetic Therapeutic Approaches for CNS Targets

  • Sue Browne Vice President, Research, Passage Bio

Synopsis

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

11:20 am Panel Discussion: Advancing Gene Therapy & Biomarker Technology

  • Li Chin Wong Director, Bioanalytical & Biomarker Development, Prevail Therapeutics
  • Sue Browne Vice President, Research, Passage Bio

Proving Efficacy to Regulators

10:40 am Outcome Measures for Rare Neurological Disorders

Synopsis

Talk details to follow

11:00 am A Regulatory Perspective: Gene Therapy for Common & Rare Neurological Disorders

Synopsis

Roundtable Discussion

11:20 am Panel Discussion: Overcoming Regulatory Challenges

12:45 pm Lunch Break

Clinical Gene Therapy to the CNS

1:30 pm Evaluation Current CNS Delivery Strategies Used in the Clinic

Synopsis

  • Exploring Intraparenchymal Drug Administration (CED) devices
  • Discussing ICV / ICM routes for drug administration
  • Highlighting drug delivery to the CNS unmet needs

1:50 pm Speaker TBC

2:20 pm Translation from Animals to Humans: Surgical Delivery

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

Synopsis

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

2:40 pm Panel Discussion: Gene Therapy Administration Devices

  • Chris Tarapata Head, Implantable & Specialized Devices, Takeda
  • Paul Larson Professor & Vice Chair of Neurological Surgery , The University of California San Francisco

3:10 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

Synopsis

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

Synopsis

  • 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

4:50 pm Take Home Messages: Clinical, Preclinical & Regulatory Learnings

  • John Shoffner Vice President, Head of CNS Therapeutics, Clinical Sciences, Sangamo Therapeutics

Synopsis

Summarizing conference lessons by highlighting areas of agreement and areas of controversy to provide an overview for the audience of major trends identified during the conference that impact gene therapy clinical development.

5:10 pm Chair’s Closing Remarks

GO TO DAY TWO