Safer, more effective advanced therapies are defined by tailored expression of the transgene. Together with you, we select the screening systems and criteria to develop the promoter your therapy needs, and validate it experimentally. We have serviced dozens of leading biopharma, some of whom have taken our promoters into clinical-stage therapies.
Gene therapies depend on three components: the vector, the therapeutic gene, and the promoter that expresses it. Safety and efficacy hinge on precise control over where, when and how much the gene is expressed. A vector can be targeted, but delivery is never perfect, and it does not control how strongly or how long the gene stays active. The promoter, often the least considered component, is the ultimate gatekeeper of on-target, durable expression. A suboptimal promoter can stall a therapy in the clinic, so getting it right from the start matters.
Using SuRE™, we functionally measure millions of human genome-derived regulatory elements directly in the relevant cell types, generating experimental data that goes beyond computational prediction. The screen is shaped with you around the on- and off-target cell types, expression levels, model system and inducibility your therapy requires.
Because we measure rather than predict, we can develop promoters even where no prior data exists, such as a novel small-molecule-inducible switch, and screen at scale directly in vivo, in the model that reflects your therapy. The elements we deliver are validated, and yours to own.
Human genome-based, experimentally validated regulatory elements readily available for evaluation and licensing for your cell or gene therapy program.
Controlled expression in defined cell types (e.g. pan-CNS, glial, cardiac), minimal off-target activity.
Triggered and doseable by cell state, compounds to make your therapeutic gene active only where and when needed.
Stable, high-level expression for consistent transgene activity over time.
Saturation mutagenesis to boost activity or manufacturability, reduce background, remove CpG sites, or shrink size without losing expression.
What a project looks like depends on the customer’s needs. Typically, it involves four steps:
Find the genomic locations where your transgene performs reliably.
AIM™ maps integration sites at scale while in parallel measuring expression for each site. This reveals where expression is stable, controlled, and best suited to your system.
Find the regulatory sequences that give your gene the right expression behavior.
SuRE™ screens promoters, enhancers and regulatory variants at scale to identify what delivers the right strength, specificity and control.
We imagine there could be some questions you want to ask us. Discover the most frequently asked questions about this subject right here.
Let’s start with your goals.
Share your challenge, question or ambition. We will help you explore how tailored gene expression can improve the safety and effectiveness of your therapy.
