AAV Edge System

A suite of tools for end-to-end payload design and gene therapy production.

OVERVIEW

A flexible, one-stop-shop for AAV tools

The AAV Edge System is the first comprehensive platform to enable end-to-end optimization, allowing developers to choose the modules that best address their design and production needs from a singular access point. Choose from payload design tools that enable precise and tissue-specific control of gene expression, improve manufacturability, and increase potency, and an AAV production system that includes a clonal suspension-adapted GMP-banked HEK293 cell line, flexible plasmid design, and model-guided processes to achieve up to E12 vg/mL titers.

Payload Design

Tissue-specific promoters

Enhance safety and efficacy with AI-designed, in vivo validated TSPs.

Sequence optimization

Increase expression using advanced model-guided DNA sequence optimization.

GOI silencing during production

Improve manufacturing efficiency and avoid GOI toxicity.

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Production System

Robust clonal HEK293 cell line

Fast growing, GMP, suspension-adapted HEK293 cell line.

Model-guided PD

Leverage model-guided processes tested across multiple scales.

Transient plasmid system

Optimized 2-plasmid system for transient AAV production.

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We provide tools for you to develop a cell line

We will ship the software, cells, and genes you need to design your cell line in-house.

You design DNA

You use our software to design and optimize genetic constructs using best-in-class parts and models.

We build DNA

We will ship the software, cells, and genes you need to design your cell line in-house.

You transfect cells

You transfect and generate stable cell lines and use our software for data analysis.

You request producer cells

You request a producer cell line for a biologic or gene therapy.

We do cell line development

We do cell line development and process development at our site.

We share progress

We share progress and data with you using  our software.

We ship you producer cell banks

We ship you clonal cell banks and transfer the process to your site.

Payload Design

GOI silencing during production

Improve manufacturing efficiency and avoid GOI toxicity.

Tissue-specific promoters

Enhance safety and efficacy with AI-designed, in vivo validated TSPs.

Sequence optimization

Increase expression using advanced model-guided DNA sequence optimization.

Learn more

Bioproduction

Robust clonal HEK293 cell line

Fast growing, GMP, suspension-adapted HEK293 cell line.

Model-guided PD

Leverage model guided processes tested across multiple scales.

Transient plasmid system

Optimized 2-plasmid system for transient AAV production.

Learn more

Production system performance

High titers across serotypes

Our 2 plasmid system achieves up to E12 vg/mL titers. High performance demonstrated across multiple serotypes with eGFP payload.

High performance across multiple CAR transgenes

E12 titer with eGFP using 2-plasmid system

2-plasmid system outperforms 3-plasmid system across both titer and % full, while reducing GMP plasmid cost.

anti-BCMA Car LV

8.3E7 TU/mL

Yescarta CAR LV

1.1E8 TU/mL

Kymriah CAR LV

1.5E8 TU/mL

Kymriah case study:
LV Edge Packaging System vs transient

The LV Edge Packaging outperforms commercially-available systems.

Titers: Codon optimization of the CDS results in a 5-fold improvement in functional titer and 10-fold improvement of the infectivity ratio. As expected, both LV Edge Packaging and standard four-plasmid systems both show increased titers from sequence optimization.

Variability: Lentiviral production from a single-plasmid transfection results in reduced inter-batch variability versus the standard four-plasmid process. Importantly, the observed reduction in variability is maintained across the different sequence-optimized transfer plasmids.