Drug discovery, redesigned

A disease is a signature.
We find the drug
that reverses it.

Most drug discovery looks at biology one gene at a time. We measure all of it — every gene, every cell, every change a drug makes — so the right medicine becomes a match, not a guess.

The platform's principle

Everything lives inone shared language.

Whatever we measure — what a disease does to cells, what a drug does to cells — it lands in the same place: transcriptomic data. The disease and the drug become directly comparable.

The shared coordinate system
Transcriptomic space
Gene expression vectors
across cells & conditions
a neuron
measure every gene
Its signature — a pattern across thousands of genes

No single gene tells the entire story. The whole pattern is the cell's fingerprint — every cell type has its own, and any cell, healthy or diseased, can be read the same way and compared.

So we can ask two questions:

01
Given a disease…

What’s the signature?

Sometimes a single broken gene; more often a coordinated shift across thousands — in specific cell types, in a pattern unique to that disease.

02
Given a molecule…

What does it perturb?

Not just on its intended target. Every gene the compound changes — profiled with DRUG-seq, in disease-relevant cells.

…and read the answers in the same units.
The platform

Map disease. Profile drugs. Find the match.

01
Disease Signature Atlas

Map every disease

Patient-derived tissue profiled with scRNA-seq, across the full transcriptome — the signature of each condition.

02
Drug-Gene Atlas

Profile the right drugs

Disease understanding tells us which compounds are worth testing — then DRUG-seq measures the full transcriptomic effect of each, in the cell types where the disease manifests.

03
Conductor AI

Match — and discover

Machine-learning models that find reversal candidates — and search billions of synthesizable compounds for new ones.

A Transcripta researcher walking a colleague through transcriptomic analysis
Why this exists

The people behind the platform.

Chemists, biologists, and machine-learning researchers rarely work this close together. At Transcripta they do — because matching a drug to a disease signature only works when all three are in the same room.

Pipeline

Five programs. Two modalities. One platform.

Conditions where decades of drug development have come up short: neurodevelopmental, neurodegenerative, and rare muscular diseases. Each shares complex transcriptomic dysregulation that traditional approaches can't address.

↪ Expand any program — its signature reverses toward healthy

Genetically diverse forms of ASD converge on shared transcriptomic signatures — a pattern of synaptic hyperexcitability across specific neuronal cell types. By targeting the shared signature rather than individual mutations, our platform can address a broad population through a single therapeutic approach.

FSHD involves dysregulation across hundreds of genes in muscle tissue — not a single broken gene, but a broad transcriptomic shift. This makes it ideally suited for a signature-based approach: characterize the full disease pattern, then find compounds that reverse it.

MSH3 drives the somatic repeat expansion underlying Huntington’s progression. Our Drug-Gene Atlas identified compounds that suppress MSH3 expression — a discovery made possible by measuring drug effects across the full transcriptome, not just a single readout.

Like Huntington’s, Myotonic Dystrophy is driven by somatic repeat expansion mediated by MSH3. Our Drug-Gene Atlas revealed that compounds suppressing MSH3 expression are effective across both diseases — demonstrating how full-transcriptome characterization can unlock a single therapeutic approach for multiple conditions sharing the same biological driver.

Leigh Syndrome produces a distinct transcriptomic signature reflecting mitochondrial dysfunction across multiple cell types. The rarity and severity of the disease make a signature-based drug matching approach especially valuable — it enables rapid identification of therapeutic candidates from existing compound libraries.

Co-discover with us
Bring a program to full resolution.

We partner with pharma, biotech, and academic teams to apply the Disease Signature and Drug-Gene atlases to new programs — and find the compounds that reverse disease.

Explore the data
Our CRISPRi perturbation dataset — open on Verily Exchange.

Hundreds of gene perturbations, profiled at full transcriptomic resolution. Open to academic researchers and industry partners.