Event Recap

AAV Innovation Summit 2024

Optimizing genome integrity through AI and vector design

November 6
8:30am-1pm EST
Cambridge, MA

Watch Form Bio and leading experts from industry and academia’s half-day summit on AAV innovation. This exclusive event brought together top minds in gene therapy to explore the latest innovations, challenges and industry trends.

Interested in attending the  March 2025 AAV Summit in Raleigh, NC.?

Register for 2025 Summit

Watch the 2024 Summit

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3:57

Advancing Gene Therapy Through Collective Innovation

Mark Swendsen, Chief Revenue Officer at Form Bio kicks off the inaugural AAV Innovation Summit by introducing the speakers, the AAV working group, and  the key industry challenges, and how the community is advancing with robust digital infrastructure.

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33:56

Understanding AAV Vector Genome Integrity

Phillip Tai, PhD, Assistant Professor, Horae Gene Therapy Center, UMass Chan Medical School, explores the development and application of advanced sequencing methodologies to address the challenges of heterogeneity in AAV vector design.

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18:37

Designing rAAV Vectors Using In Silico Vector Modeling

Adam Cockrell, PhD, Sr. Director Vector Biology at Solid Biosciences delves into the characterization and design of AAV vectors, focusing on the use of in silico technologies and vector modeling to expedite early research and improve therapeutic development.

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16:31

CDMO Perspective: AAV Manufacturing Challenges & the Role of AI

Nithya Jesuraj, PhD, Director, Commercial R&D at National Resilience explores the challenges in AAV manufacturing and the role of AI in addressing them.

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24:56

AI & In Silico Tools for Faster, More Predictable AAV Development

Amicia Elliott, PhD, Product Director - Life Sciences at Form Bio explores the transformative potential of AI and in silico computational tools in accelerating and enhancing the predictability of AAV-based gene therapy development.

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34:20

Standardized Nomenclature & Reporting for Long-Read Sequencing & Analysis in rAAV Vectors

In this panel discussion, members of the AAV Working Group delve into the current challenges in gene therapy, the use of long-read sequencing for genome integrity and vector design, FDA's requirements for genome integrity measurements in IND applications, and more.

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23:02

State of the Gene Therapy Industry

Tim Hunt, CEO at Alliance for Regenerative Medicine discusses the industrialization of the cell and gene therapy field, highlighting the role of AI and digital innovation in advancing the sector.

Speakers

Phillip Tai, PhD

Phillip Tai, PhD

Assistant Professor, UMass Medical School
Tim Hunt

Timothy Hunt

CEO, Alliance for Regenerative Medicine
Adam Cockrell, PhD

Adam Cockrell, PhD

Sr. Director, Vector Biology, Solid Biosciences
Nithya Jesuraj, PhD

Nithya Jesuraj, PhD

Director, Commercial Development, Gene therapy, National Resilience
Nadia Sellami, PhD

Nadia Sellami, PhD

Segment Lead, CGT, PacBio
Amicia Elliott, PhD

Amicia Elliott, PhD

Product Director, Life Sciences, Form Bio
Alpha Diallo, PhD

Alpha Diallo, PhD

Head of Solutions Science, Form Bio

Partners

Preliminary Agenda

  • 8:30 AM
    Check-in & coffee
  • 9:00 AM
    Welcome Remarks
  • 9:15 AM
    Understanding AAV Vector Genome Heterogeneity
    • Phillip Tai, PhD - Assistant Professor, UMass Medical School
  • 9:45 AM
    designing recombinant aav vectors using in silico vector Modeling
    • Adam Cockrell, PhD - Sr. Director, Vector Biology, Solid Biosciences
  • 10:15 AM
    CDMO Perspective: AAV Manufacturing Challenges and the Role of AI
    • Nithya Jesuraj, PhD - Director, Commercial Development, Gene therapy, National Resilience
  • 10:30 AM
    AAV Working Group Panel - Standardized Nomenclature and Reporting for Long-Read Sequencing and Analysis in rAAV Gene Therapy Vectors
    • Adam Cockrell, PhD - Sr. Director, Vector Biology, Solid Biosciences
    • Alpha Diallo, PhD, Head of Solutions Science, Form Bio
    • Nadia Sellami, PhD, Segment Lead, CGT, PacBio
    • Phillip Tai, PhD - Assistant Professor, UMass Medical School
    • Nithya Jesuraj, PhD - Director, Commercial Development, Gene therapy, National Resilience
  • 11:15 AM
    AI & in silico tools for faster, more predictable AAV Development
    • Amicia Elliott, PhD - Product Director, Life Sciences, Form Bio
  • 11:40 AM
    Gene Therapy State-of-the-Industry
    • Timothy Hunt - CEO, Alliance for Regenerative Medicine
  • 12:00 PM
    LUNCH / NETWORKING
  • 1:15 PM
    Summit Concludes

Areas of Focus

Utilizing long-read sequencing for AAV Genome Integrity Analysis

Advancing long-read sequencing analysis with open-source tools (bioRxiv publication)

Leveraging AI and in silico tools to accelerate AAV Gene Therapy Development

Who Should Attend?

Executives who work in AAV gene therapy sector

Scientists who work in AAV therapeutic development, including preclinical and manufacturing

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