*All times are in EDT

9:20 am Chair’s Opening Remarks


9:30 am Developing Next Generation Vectors to Treat Pediatric Patients

  • John Xiong Director, Head of In Vivo Pharmacology, LogicBio


  • Current vector technologies are not suitable for liver targeted indications in pediatric patients as hepatocyte proliferation results in loss of transgene expression
  • By precisely inserting a corrective transgene into the genome through the GeneRide technology, durable transgene expression can be achieved
  • Highlighting current data on GeneRide as the next generation of gene therapy technologies

10:00 am Assessing the Big Pharma Approach to Next Generation AAV Development & Implementation


  •  Understanding the dynamics between big pharma and other key stakeholders when reviewing external opportunities for improved technologies/new modalities
  • Working with external organizations as well as internal efforts in the context of assessing translatability of vectors
  •  What key criteria do big pharma use to assess which vectors to use and progress in gene therapy development?

10:30 am Morning Break & Networking

10:35 am


11:00 am Characterizing the Proteomic and Epigenomic Landscape of AAV

  • Nicole Paulk Assistant Professor, Biochemistry & Biophyiscs, University of California San Francisco


  • First, publicly available, unbiased ‘omics characterization of AAV
  • Detailed comparison of biochemical differences in AAV by production platform
  • Key insights for choosing a vector production platform

11:30 am Transcriptome-Based Quantification of AAV Variants with Single- Cell Resolution

  • Leah Byrne Assistant Professor of Ophthalmology, University of Pittsburgh


  • Although the choice of vector is critical for successful clinical translation, accurate and quantitative comparison of AAVs, especially in primates, remains challenging
  • We have developed scAAVengr, a single-cell AAV engineering pipeline to simultaneously quantify and rank efficiency of competing AAV vectors across all cell types in parallel, in the same animals
  • Transcriptome based-quantification of AAV-mediated gene expression allows for precise evaluation of AAV performance and determination of vector tropism, as well as the development of novel AAV vectors for rapid and efficient clinical translation

12:00 pm A human iPSc Platform to Model Gene Therapy Vector Safety

  • Michael Themis Reader, PI of Gene Therapy and Genotoxicity, Brunel University London


• We have developed a human specific gene therapy safety platform for LV and AAV vectors
• Our platform avoids the bias provided by in vivo and cell line based models
• The platform is modular and uses a liver based pharmacotoxicological cellular component coupled with molecular assays that measure the known vector associated genotoxicity contributors to oncogenesis
• Outreads are subjected to simple OMICS tests using positive and negative control vectors

12:30 pm Lunch & Networking

1:30 pm Characterization of rAAV Key Quality Attributes Generated From A Highly Optimized, Hela 3.0 Producer Cell Line (PCL) Production Platform


  • HeLa 3.0 Producer Cell Lines were generated via genetic modification of existing, highly optimized PCLs
  • HeLa 3.0 PCLs generated high titer, high quality rAAV for multiple programs
  • HeLa 3.0 PCLs demonstrate high genetic stability during manufacturing

2:00 pm Virtual Roundtables: Developing Translatable Vectors: Key Considerations


Even if a vector is designed which shows high efficiency and targeting, there are still barriers in translating this effectively to the clinic. Whether thinking about complexity of the vector and therefore ease of manufacturing, or understanding if performance in mouse models is an accurate indicator of performance in humans, several factors influence what will make for a successful vector to proceed into clinical trials. This set of roundtables will explore some of the key considerations when developing not only a specific and effective vector, but a translatable one.

Predicting Translatability Assessing Manufacturability Navigating Regulatory Considerations



2:30 pm Afternoon Break & Networking


3:00 pm Strategies to Tackle Gene Therapy for Large or Alternatively Spliced Genes

  • Hemant Khanna Associate Professor, University of Massachusetts Medical School


  • Current AAV vectors are limited in capacity
  • Several therapeutic targets would require presence of functional genes which are too large for AAV
  • Addressing AAV payload size through developing minigenes is a promising option for several diseases, including Duchenne Muscular Dystrophy

3:30 pm Developing Dual Vector Systems to Deliver Larger Genes to Target Cells

  • Vivian Choi Head of Global Gene Therapy Research, Takeda


  • Dual vector system is an approach to expand the packaging size of AAV vectors by exploiting the natural molecular mechanism of AAV vector genome concatemerization
  • Pros and cons of using a dual AAV vector system versus single AAV vectorsystems and other viral and non-viral delivery systems
  • Approaches that could increase efficiency of dual vector system described in the field
  • Considerations for developing dual vector as a drug product: Product characterization, potency, fill & finish considerations and clinical

4:00 pm Chair’s Closing Remarks

4:10 pm End of Day Two & Close of Conference