7:00 am Check-In & Coffee

7:50 am Chair’s Opening Remarks

Realizing The Potential Of Next-Generation Vectors

8:00 am Showcasing Less Liver-Tropic Development Efforts for IV-Administered Vectors

  • Leszek Lisowski Unit Head Of Translational Vectorology Unit, Children's Medical Research Institute

Synopsis

  • Outlining the latest rational design and natural variants methods for AAV vector engineering
  • Highlighting a range of vector bioengineering efforts
  • Covering liver, CNS, and ocular focused vector development, as well as T cells for CAR-T development

Vector Development Strategies to Overcome Immunogenicity & Toxicity Hurdles

8:30 am AAV Capsid Engineering to Modify the Immune Response

  • Jing Liao Director - Vector Operations, Alexion Pharmaceuticals

Synopsis

  • Which capsid components are predictive of a negative immune response?
  • Presenting a capsid engineering approach to dampen innate and adaptive immune responses
  • Outlining engineering methodology to derive the novel capsid variant
  • Analyzing In vivo performance and addressing human translatability challenges

9:00 am Next Generation Evolved and VHH- Modified AAV Vectors for Retinal Gene Transfer and Local Gene Delivery

Synopsis

  • Analyzing the transduction of multiplexed vectors for retinal gene transfer, following subretinal dosing at the single-cell level
  • Exploring Revvity’s novel direct-targeted AAV vector platform, developed by inserting a VHH domain into the cap protein
  • Optimizing production to generate yields of 1.4E14vg/l in suspension cells and enhanced in vitro and in vivo gene delivery
  • Showcasing promising correlations between vector biodistribution and target receptor expression in vivo in an exploratory multiplexed NHP dosing study

9:30 am Morning Refreshments & Speed Networking

Synopsis

Our speed networking is the ideal opportunity to get face-to-face time with many of the brightest

minds working in gene therapy and introduce yourself to the attendees that you would like to have

more in-depth conversations with. Benchmark against the industry leaders and establish meaningful

business relationships to pursue for the rest of the conference and beyond.

10:30 am Highlighting Better Tissue-Specificity Development Efforts for IV-Administered Vectors

Synopsis

  • Improving capsid and payload design efforts to increase tissue specificity
  • Decreasing vector sequestration by the liver: does liver de-targeting impact extrahepatic biodistribution?
  • Highlighting detargeting efforts of other off-target tissues (e.g. DRG detargeting for CNS targeted capsids)

11:00 am Engineering Human CNS Receptor-Targeted Capsids for Improved Antibody Evasion

  • Simon Pacouret Senior Research Scientist, The Broad Institute of MIT & Harvard

Synopsis

  • Developing human CNS receptor-targeted capsids for brain-wide gene delivery
  • Enhancing the resistance of human CNS receptor-targeted capsids to neutralizing antibodies (NAbs)
  • Testing key capsid candidates for manufacturability, function and NAb evasion

11:30 am scAAVengr Derived Intravitreal Vectors for Ophthalmic Gene Therapy

Synopsis

  • New data on Avista’s lead candidate capsids for intravitreal delivery
  • AI/Machine Learning techniques to identify and optimize capsids
  • Advancement towards clinical indications for Avista’s internal pipeline

12:00 pm Lunch & Networking

Capsid discovery & engineering 2.0

1:00 pm Transforming AAV Features with Ligand-Capsid Bioconjugation

Synopsis

  • Simple click chemistry enables great flexibility of ligand choices
  • Bioconjugation to add novel vector characteristics: example of cell-type specificity
  • Future applications of AAV bioconjugation

1:30 pm Machine-Guided Design of AAV Capsids for Ocular & CNS Indications

  • Eric Kelsic Founder and Chief Executive Officer, Dyno Therapeutics

Synopsis

  • Highlighting the latest data for Dyno eCap 1 and bCap 1 vectors as ocular and CNS delivery vectors respectively
  • Benchmarking progress with AI and ML approaches and applications
  • Outlining a roadmap of these innovative vectors to reach the clinic

2:00 pm AI-Powered Variant Discovery for Novel AAV Vectors

Synopsis

  • InsightMiner & CARE(Cell-specific AAV Research Engine)
  • On- & De-targeting tissues or cells
  • From plasmids to lead vectors

2:10 pm Updates on Capsida’s Next-Generation Engineered AAV Capsids for the Clinic

Synopsis

  • Platform advancements leading to breakthrough, IV-delivered engineered AAV capsids for the CNS
  • Generations of directed evolution yields highly targeted AAV capsids with increased therapeutic index
  • Potential for Capsida’s engineered AAVs to treat neurological disorders across all age groups

2:20 pm
Roundtable Discussion: A 101 in Primary Methods for Capsid Discovery & Engineering

Rational Design

Synopsis

Delve into the Latest Understanding of Capsid Structure-Function Relationships to Generate Tissue-Specific and Immune-Evading Capsids

Directed Evolution

Synopsis

Leverage the Most Effective in Vivo Models to Discover and Engineer Highly Translatable Vectors

Bioconjugation

Synopsis

Understand the Intricacies of Chemical Modifications to Stabilize Viral Vectors and Improve Their Tissue-Specific Target Binding Profiles

3:00 pm Afternoon Refreshments & Networking

3:45 pm Therapeutic Vector Evolution: Concept to Clinic

Synopsis

  • 4DMT’s approach to Directed Evolution
  • Translatable capsid characterization
  • Successful IND submissions

4:15 pm Discovery and Mechanism of Action of Cross-Species BBB-Penetrant Capsids

  • Brett Hoffman Senior Scientist II, Novel Capsid Discovery, Voyager Therapeutics

Synopsis

  • TRACER™ AAV capsid discovery platform enables rapid discovery of CNS tropic AAV capsids
  • Identification of Novel Cross-Species BBB-Penetrant capsid family
  • De-risking human translatability though receptor discovery

4:45 pm Panel Discussion: Evaluating Investor Interest in Next-Generation Gene Therapy Technologies

  • Lucia Faccio Partner, Sofinnova Partners
  • Campbell Murray Senior Research Analyst, Agent Capital
  • Amanda Placone Senior Director External Innovation - Gene Therapy and Gene Editing, Eli Lilly & Co.
  • Morten Sogaard President, Gene Therapy Research & Technical Operations, Astellas Innovation Management LLC

Synopsis

  • What innovation in gene therapy vector development makes for an attractive investment proposition?
  • What differentiating factors and data packages are big pharma looking for to acquire or collaborate with new startups?
  • Looking beyond rare disease: are investors confident that better delivery tools could enable gene therapy success in more prevalent conditions?

5:15 pm Chair’s Closing Remarks

5:20 pm End of Day One