Conference Day One | Wednesday, July 30

7:30 am Check-In & Coffee Networking

8:25 am Chair’s Opening Remarks

Optimizing Preclinical Development to Accelerate Progression to the Clinic

8:30 am Panel Discussion: Improving Safety & Efficacy Data by Rethinking Large Animal Models

  • Ralf Schmid Associate Director - Pre-Clinical Research, Novartis AG
  • Lorelei Stoica Executive Director - Nonclinical Research, Ultragenyx Pharmaceutical Inc.
  • Samir Koirala Head of Genetic, Neurodevelopmental & Epilepsy Disorders, Biogen
  • Daniel Carlson Chief Scientific Officer, Recombinetics

Synopsis

  • Outlining the holdbacks of non-human primates from a cost, time, and ethical standpoint
  • Emphasizing how utilizing pig models for safety and efficacy tests addresses the challenges presented by non-human primate use
  • Highlighting that regulators do not always require the use of non-human primate models

9:30 am Redefining Preclinical Strategy: Leveraging Humanized Mouse Models to Accelerate Regulatory Pathways for CNS Gene Therapies

Synopsis

  • Highlighting engineered AAV capsids that effectively cross the blood-brain barrier by targeting human receptors
  • Engineering capsids with specificity to a human receptor can render humanized mice highly effective for biodistribution, toxicity, and efficacy studies that are both translationally relevant and cost-efficient
  • Streamlining to a single-species preclinical model reduces development time, ethical burden, and barriers to IND submission

10:00 am Lonza | Expertise Partner Presentation

10:30 am Morning Refreshment Break & Speed Networking

11:30 am Accelerating Clinical Impact with Novel AAV Vectors Designed for Enhanced Delivery to Eye, Muscle, & CNS

  • Eric Kelsic Co-Founder & Chief Executive Officer, Dyno Therapeutics

Synopsis

  • Applying AI to design AAV capsids with optimized transduction efficiency for eye, muscle, and CNS delivery to facilitate enhanced clinical outcomes
  • Validating capsid performance in NHPs to ensure safety, efficacy, and effective translation of novel capsids
  • Demonstrating delivery to specific cell populations to motivate accelerated development of more effective treatments for rare and common diseases

Evaluating Factors Impacting Immunogenicity & Dosing to Navigate Safety Concerns

12:00 pm Exploring the Benefits of Direct Administration to Lower Dose & Cost

  • Robert Lin Chief Executive Officer, Avista Therapeutics

Synopsis

  • Evaluating systemic and direct routes of administration for vector therapy
  • Outlining the workflow to implement direct administration for CNS indications
  • Elevating the safety and cost benefits of direct administration

12:30 pm Roche | Hosting Partner Position

1:00 pm Lunch Break & Networking

2:00 pm Understanding Immunosuppression for AAV Vectors to Improve Safety

  • Kruti Patel Associate Director - Adeno Associated Virus Immunology, Solid Biosciences

Synopsis

  • Exploring modifications to AAV vectors which facilitate immune suppression
  • Emphasizing how strategies to eliminate immunogenicity improve vector efficacy
  • Highlighting how immunosuppression can also enable a reduction in dose

Identifying & Utilizing Effective Payload Design to Improve Vector Efficacy

2:30 pm Optimizing Overall Vector Efficacy with Promoter Selection & Engineering

  • Daniel Cohen Head of Vector Optimization, Spark Therapeutics, Inc.

Synopsis

  • Outlining how promoter engineering enhances transgene expression
  • Understanding how promoter strength and vector dose affect the predictability of gene therapy
  • Exploring effects of promoters on ON-target versus OFF-target gene expression

3:00 pm Afternoon Break & Networking

3:30 pm Engineering Payload to Increase Expression Durability & Lower Doses

  • Zhongdong Shi Vice President & Head of Research & Development, Frontera Therapeutics

Synopsis

  • Highlighting current developments and challenges in AAV gene therapy for Hemophilia A
  • Emphasizing how engineering the FVIII protein to generate hyperfunctional mutants could enhance expression durability and improve therapeutic efficacy
  • Demonstrating an efficient approach to generate hyperfunctional transgenes, enabling lower therapeutic doses and improved durability compared to firstgeneration AAV gene therapies

4:00 pm Developing Site-Specific Gene Integration Technologies to Reverse Genetic Disorders in a Mutation-Agnostic Manner

  • Daniel Gao Post-doctoral fellow, The Broad Institute of MIT & Harvard

Synopsis

  • Exploring programmable gene integration technologies to enable mutation-agnostic editing strategies
  • Engineering integrases for efficient, site-specific gene integration using phage-assisted continuous evolution and rational protein design
  • Optimizing editor components and delivery vectors to enhance the therapeutic potential of gene integration technologies

4:30 pm Exploring How Fine-Tuning Expression Levels Better Addresses Specific Indications

Synopsis

  • Understanding that different indications require different transgene expression patterns
  • Exploring the possibility of transient but durable expression to treat different indications
  • Reviewing design techniques which influence expression levels

5:00 pm Chair’s Closing Remarks

5:05 pm Close of Conference Day One

REGISTER NOW
FULL EVENT GUIDE
CONFERENCE DAY 2 →