Platform Science Team Partner with Us
Cell & Gene Therapy · Regulatory Genomics

Designing
Genetic Control.
Precisely.

Locium — Generative AI · High-Throughput Validation

Every cell and gene therapy needs a regulatory element. None are optimized. Locium pairs generative AI with scaled MPRA validation to design synthetic promoters that are specific, compact, durable, and delivery-compatible.

Explore the Platform Partner with Us
Scroll
The Problem

Every gene therapy needs
a regulatory element.
None are currently optimized.

Promoters and enhancers control where, when, and how much a therapeutic gene is expressed. Today's standard elements — CMV, CAG — drive expression broadly, causing off-target toxicity, immune responses, and silencing in vivo. Locium designs the missing layer: synthetic regulatory elements that are cell-type-specific, compact enough for AAV, and durable after delivery.

Cell-Type Specific

Expression targeted to the right cell type — reducing off-target toxicity and enabling safer, lower-titre delivery.

Compact

Synthetic promoters under 400 bp, preserving precious AAV cargo space for larger therapeutic genes.

Durable

Synthetic sequences engineered to resist epigenetic silencing — maintaining therapeutic expression long-term.

Delivery-Compatible

Designed for AAV, LNP, and engineered cell therapies — validated across modalities before handoff.

The Locium Platform

Design · Validate · License

Three integrated pillars — a proprietary knowledge base, generative AI models, and in-house high-throughput validation — create a closed-loop engine for fit-for-purpose regulatory element design.

01
Regulatory Knowledge Base

One of the most comprehensive multi-modal regulatory maps ever built: activity profiles of 3M+ regulatory DNA elements across hundreds of human cell and tissue types, with 800k+ sequences profiled for transcriptional activity. This proprietary dataset is the foundation for all AI training and design.

Epigenomics · MPRA Atlas · Training Data
02
Generative AI Design

Advanced AI models — CNN for convolutional sequence features, Transformer for long-range attention context, and Diffusion for generative sequence sampling — generate and score hundreds of thousands of synthetic promoters for any partner-defined target. Built on our CODA and DNA-Diffusion platforms (Nature 2024, Nature Genetics 2026).

DNA-Diffusion · CODA · Generative AI
03
High-Throughput Validation

In-house MPRA technology measures hundreds of thousands of regulatory designs in parallel — across neuron, liver, muscle, immune, and heart systems. Demonstrated cell-type-specific expression in mouse embryo and brain in vivo, and therapeutic proof-of-concept in human leukemia cells.

MPRA · In Vivo · Cell-Type Specific
How it Works

Partner input to
validated promoter portfolio

Partners specify their program needs — we deliver experimentally validated, licensable synthetic regulatory elements ready for therapeutic vector integration.

1

Specify Your Target

Define the cell type or tissue, delivery vehicle (AAV, LNP, cell therapy), required expression level, size constraints, and any stimulus-response needs. Partner data and indication-specific systems plug directly into the pipeline.

2

AI Generates & Scores Candidates

Our DNA design engine generates hundreds of thousands of synthetic promoter candidates, scoring each for predicted specificity, expression level, compactness, and delivery compatibility — before any wet lab work begins.

3

MPRA Validation at Scale

Top AI candidates enter our high-throughput MPRA pipeline — functional data from hundreds of thousands of sequences in parallel, returned in weeks. Focused in vivo testing confirms therapeutic-grade leads.

4

Receive a Validated Promoter Portfolio

Ranked, experimentally validated hits delivered with full functional data. Partners license field-of-use rights and take the element forward through IND, clinical trials, and commercialization. Locium earns upfront fees, milestones, and royalties.

Synthetic Promoter · Design Output
Validated · Neuron-specific · 380 bp · AAV-compatible
Predicted Specificity Score
Neuron 96.4%
Liver 1.8%
Muscle 1.2%
The Team

Founders behind
the science.

Locium was built by the scientists who created the field's foundational tools — regulatory atlases, MPRA technology, and AI-driven sequence design. Our team has collectively published the reference datasets, models, and validation methods that the entire gene therapy community relies on.

We partner with pharma, biotech, and academic gene therapy programs across AAV, LNP, and engineered cell therapy modalities. Partners own the IND, clinical trials, and commercialization — Locium earns upfront license fees, development milestones, and royalties.

Partner with Locium
Nature 2020 · DHS Regulatory Atlas
Nature 2024 · CODA Platform
Nature 2026 · MPRA at Scale
Nature Genetics 2026 · DNA-Diffusion
Get in Touch

Partner with us

Whether you're developing a gene therapy programme, exploring a research collaboration, or looking to license synthetic regulatory elements for your therapeutic vector — we'd like to hear from you.

info@locium.bio locium.bio