8:45 am Chair’s Opening Remarks

Driving Therapeutic Discovery & Development with Synthetic Biology Tools

9:00 am Harnessing Synthetic Biology to Optimize T-Cell Function

Synopsis

• Outlining the areas of unmet need in therapeutic development in oncology
• Discussing how synthetic biology tools can be deployed to overcome current therapeutic limitations
• Building an R&D pipeline and planning to implement synthetic biology in development
• Providing an update on synthetic biology development at Kite, sharing experience of applying synthetic biology in cell therapy and outlining future developments

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

11:00 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

11:30 am Leveraging Synthetic Circuits to Modify T-Cell Therapies for Safety, and Multi-Functionality

Synopsis

• Designing complimentary constructs to the TCR to enhance control with synthetic genetic circuits
• Engineering cells which respond to dynamic temporal and spatial conditions in vivo with smart sensors to detect disease environment
• Developing logic gates to ensure disease tissue specificity, and regulator dials to tune therapies in vivo, creating a narrow dose window
• Engineering multiple synthetic units into T cells using viral and non viral vectors optimized for T-cell transduction

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 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

4:00 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

4:30 pm Implementing Next Generation Cellular Reprogramming for the Consistent and Scalable Production of Cells for Therapies

Synopsis

• Harnessing transcriptional regulation to modulate cell characteristics, and optimize function for therapeutic application
• Identifying cell therapy manufacturing bottlenecks which can be solved with synthetic biology
• Enhancing functionality with synbio tools

5:00 pm Programming Living Cells to Advance the Design and Manufacture of Biologics and Cell Therapies

Synopsis

• Leveraging computer-aided design to simulate and redesign genetic systems to create novel cell lines with engineered function
• Building a database of genetic parts to engineer novel function into cell lines using optimized vectors for delivery
• Creating GMP host cell lines for cell-based production of biologics and viral vectors using synthetic biology-based cells
• Leveraging machine learning to investigate large datasets from cell read-outs to design and investigate biological complexity

5:30 pm Chair’s Closing Remarks

End of Day 1