Agenda

9:20 am Chair’s Opening Remarks

MONITORING AAV SAFETY SIGNALS ACROSS DIFFERENT TARGET INDICATIONS

9:30 am Exploring the Optimal Way to Measure & Monitor AAV Safety Signals in the CNS

  • Darin Falk Chief Scientific Office, Vice President & Head Of Gene Therapy Programs, Lacerta Therapeutics, Inc.

Synopsis

• Assessing the accuracy of imaging technology to inform on the AAV safety profile

• Identifying novel strategies to facilitate monitoring by using biomarkers

• Investigating CNS gene therapy deliver method to minimize safety events

10:00 am Components that Impact AAV Biodistribution in the Nervous System

  • Kathrin Meyer Principle Investigator, The Research Institute at Nationwide Children's Hospital

Synopsis

  • Discuss challenges in AAV biodistribution in the CNS
  • Assessing difficulties in dose-escalation between species
  • Highlight potential solutions to improve AAV biodistribution in the CNS

10:30 am Morning Break & Networking

11:30 am Understanding how to Effectively Monitor AAV Safety in Gene Therapies Employing a Local Route of Administration

Synopsis

• Exploring the differences in how inflammation is tracked locally (eye/ear) compared to systemic ROA

• Delving into the key safety indicators in local delivery of AAV gene therapy

• Reviewing the accuracy of preclinical safety monitoring in locally delivered gene therapies

• Looking to the future of how AAV safety could be tracked for local ROA during the clinical development

12:00 pm Understanding How to Best Conduct Long-Term Safety Monitoring

  • Albena Patroneva Senior Vice President - Clinical Research, Neurogene Inc.
  • Sandra Raff Senior Director - Drug Safety & Pharmacovigilance, Ultragenyx Pharmaceutical Inc

Synopsis

• Assessing the current FDA guidelines

• Reviewing how effective long-term follow up protocol is at truly understanding the long-term safety of AAV gene therapies

• Strategize additional monitoring to better inform the long-term safety profile

12:30 pm Lunch & Networking

1:30 pm Challenges and Advances in AAV Delivery to Muscle and Brain

  • Nicolas Wein Associate professor, Nationwide Children's Hospital

Synopsis

• Discuss challenges in intravenous AAV delivery

• Highlight potential solutions to improve safety

• Discuss alternative delivery routes targeting muscle and CNS

DELVING INTO THE ONCOGENIC RISK OF AAV GENE THERAPY

2:00 pm Understanding the Risks Associated with AAV Vector Genome Integration

  • Weidong Xiao Associate Director of the Gene and Cell Therapy Program, Herman B Wells Venter for Pediatric Research, Indiana University

Synopsis

• Assessing how real the risk of AAV vector genome integration is in humans

• Exploring the current protocols to identify oncogenic risk and identifying elements to improve as the severity of risk is more understood

• Highlighting the best methods to monitor genome integration before, during and after trials

2:30 pm Liquid Biopsy Integration Site sequencing (LiBIS-seq) for tracking AAV genomic integration sites from blood-plasma cell-free DNA

Synopsis

• Genotoxicity associated to gene therapy treatments is an outstanding issue for the whole gene therapy field, including AAV and gene editing technologies

• The identification and tracking of genetically-modified cells is fundamental for the assessment of the efficacy and safety of gene therapy treatments and is required by regulatory authorities

• Current molecular tracking technologies rely on the analysis of DNA from circulating blood cells or solid tissue biopsies, which provides only a partial snapshot of the clonal repertoire because the small number of cells that can be tested and highly invasive when solid tissues are studied

• Blood plasma-derived cell-free DNA can be used to study and monitor the composition and fate of genetically modified cells residing in solid tissues without the need to perform invasive solid biopsies and provides a more accurate picture of the clonal repertoire at the whole organismal level

3:00 pm Chair’s Closing Remarks & End of Conference

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