8:30 am Chair’s Opening Remarks

Driving Therapeutic Discovery & Development with Synthetic Biology Tools

8:45 am Designing Synthetic Vectors to Genetically Engineer Previously Intractable Prokaryotes to Program the Microbiome

Synopsis

• Leveraging synthetic biology to engineer viral vectors to tune microbes for the in vivo production of synthetic biology-based therapeutics
• Discussing how synthetic biology tools have been used to engineer bacteriophages for optimized payload delivery tailored to a specific prokaryotic genus
• Using de novo vector technology to engineer bacteria to express therapeutic proteins at the right concentration, at the right time

9:15 am Leveraging CRISPR Automation for a New Era of Design

Synopsis

  • Applying software engineering principles to scale genome engineering efforts
  • Industrializing CRISPR through versatile cell and gene editing technology platforms
  • Making therapies accessible by supporting discovery to clinic research

9:30 am Building CRISPR 2.0 as a Programmable Search Engine to Drive Next-Generation Diagnostics and Therapeutics

Synopsis

• Leveraging the diversity of nature to identify novel CRISPR enzymes, integrate new features, and engineer CRISPR 2.0 with novel functions
• Developing programmable CRISPR enzymes to advance applications across disease detection and treatment
• Discussing how CRISPR 2.0 enzymes are opening new fields of research and driving possibility in synthetic biology

10:00 am Using Synthetic Biology to Create the Next Generation of Drug Discovery Platforms to Modulate Proteins and Target Complex Drug Mechanisms

Synopsis

• Creating cell-based platforms to produce and functionally select therapeutic candidates for drug development
• Engineering genetic circuits to leverage functional intracellular screening opposed to in vitro approaches
• Describing how synthetic cell-based platform enable discovery of compounds with novel modes of action

10:30 am Morning Refreshments & Speed Networking

Exploring a Roster of Synthetic Biology Foundational Technologies to Deploy in Therapeutic Development

11:15 am Programming T Cells Enabled by Precise Delivery of Large and Modular Gene Circuits with Multiple Therapeutic Function

Synopsis

  • Providing an overview of precise, large gene circuit delivery to overcome limitations of viral and random approaches
  • Advancing sense and respond receptors and gene circuits to enable novel therapeutic behaviors
  • Implementing host cell expression modifications to enhance cell persistence and function

11:45 am Leveraging the Ginkgo Platform for Genetic Medicines

Synopsis

  • An introduction to mammalian work at Ginkgo
  • New single cell tools to address AAV production
  • Pooled screening approaches to novel CAR designs
  • Discovery of safe harbor sites for precision cell engineering

12:00 pm Advancing Synthetic Promoter Design to Direct Behavior in Specific Cell Lines

Synopsis

• Controlling action in specific settings using smart sensors and synthetic promoters to maximize safety and specificity of mechanisms
• Leveraging bioinformatics to discover novel synthetic promoters to elicit greater transcriptional control
• Developing synthetic promoters to drive complete on/off expression and flexible gene regulation to drive transcription more precisely
• Discussing the impact of synthetic promoter design on AAV vector design to enable precise cell and tissue specificity

12:30 pm Lunchtime & Networking

Advancing Discovery & Preclinical Development of Synthetic Units

1:30 pm Developing Synthetic Biology-Based Microbiome Therapeutics

Synopsis

• Developing a computer-aided drug design platform to support oral drug delivery
• Creating synthetic microbiome therapeutics with a describable mechanism of action
• Targeting the lower gastrointestinal tract with control over therapeutic dosing
• Discussing how the use of synthetic biology provides broad clinical applicability

2:00 pm Developing Assays to Validate Synthetic Promoter Candidates Identified with Bioinformatics Tools

Synopsis

  • Leveraging bioinformatics to identify promising candidates to discover novel synthetic promoters and elicit greater transcriptional control
  • Determining the biological output of a novel promoter and whether an assay indicates clinical usefulness
  • Designing assays which select for inputs and outputs of synthetic components and ensuring assays indicate important biological function
  • Investigating which secondary targets are impacted by synthetic modules, the different ways this disrupts genes, and how modulating a gene impacts phenotypes

2:30 pm Panel Discussion: Providing Strategic Frameworks to Identify and Validate Areas Where Synthetic Biology Will Make a Therapeutic Impact

  • Tasuku Kitada Co-Founder, Director, President, Head of R&D, Strand Therapeutics
  • Ron Weiss Professor of Biological Engineering, MIT
  • Janice Chen Co-Founder & CTO, Mammoth Biosciences

Synopsis

• Defining areas of unmet need in therapeutic development and medical applications such as cell therapies, and how synthetic biology can advance development
• Providing an overview of the synthetic biology technologies available, and how they might plug therapeutic gaps
• Introducing a rational criterion for implementing synthetic units, considering what, when, where, why, how, and how much?
• Deciding when and where a living organism or pathway discovered is going to be effective, and prioritizing what to progress in development

3:00 pm Afternoon Platform Presentation & Networking

Manufacturing Complex Synthetic Therapeutics & Demonstrating Safety & Efficacy

3:30 pm Optimizing Gene Engineering, CMC, Manufacturing, and Scale Up and Sharing Regulatory Learnings to Bring Cell-Based Therapies with De Novo Function into Phase I Clinical Trials

Synopsis

  • Creating efficacy assays which demonstrate that novel synthetic circuits work and induce the desired outcomes in vivo
  • Demonstrating that an assay is appropriate and communicating a novel mechanism of action to regulators
  • Ensuring therapeutics are non-immunogenic and have minimal residual synthetic DNA
  • Engineering cell-based therapies with complex genetic circuits, and scaling up manufacture for clinical trials

4:00 pm Accelerating Innovation Cycle -Applying Synthetic Biology to Autologous Cell Therapy

  • Qi Cai Director Cell Biology, Kite: A Gilead Company

Synopsis

  • Introduction of autologous cell therapy
  • Synthetic Biology’s contribution and potential for the development of the autologous cell therapy

4:30 pm Chair’s Closing Remarks

End of Day 1