# Transcripta Bio

> Drug discovery at the resolution of biology. Using the power of the
> transcriptome to discover revolutionary medicines.

Detailed page content available in markdown:
- [Home](/index.md)
- [Platform](/platform.md)
- [Who We Are](/who-we-are.md)
- [Careers](/careers.md)
- [Contact Us](/contact-us.md)

Transcripta Bio is a biotechnology company based in Palo Alto, California.
Named to TIME100 Most Influential Companies of 2024.

## Core Thesis

The transcriptome — the full landscape of gene expression — is the most
information-dense, mechanistically grounded window into biology. It lets
us answer two fundamental questions: given a disease, what has gone wrong
in the cells of the patient? Given a molecule, what does it change in
those same cell types?

Every disease and every molecule produces a unique transcriptomic
signature in human cells. Our platform maps both — then uses AI to find
molecules whose signature can restore a disease state to healthy.

## Platform

Our platform is built around three capabilities:

- **Disease Signature Atlas**: Maps disease from patient-derived tissue with
  scRNA-seq (and bulk transcriptomics for breadth), across the full
  transcriptome. We build validated signatures — fusing expression, human
  genetics, and mechanism — so they capture what causes a disease, not just
  what correlates with it.
- **Drug-Gene Atlas**: Disease understanding guides which compounds are worth
  testing; DRUG-seq then measures each compound's full transcriptomic
  response, at dose, in the cell types where the disease manifests.
- **Conductor AI**: A system of machine-learning models trained on both
  atlases. It identifies compounds — alone or in combination — whose
  transcriptomic effect reverses a disease signature, and screens billions of
  on-demand, synthesizable molecules in silico to find novel candidates worth
  making.

[Platform Overview](https://www.transcriptabio.com/platform)

## Structural De-Risking

We attack the root causes of clinical failure before the clinic:

- **Starting from the right target**: Target selection grounded in causal
  human genetics — where the evidence establishes disease causality.
- **Matching against real patient biology**: Disease signatures derived from
  actual patient transcriptomes at single-cell resolution. Drug response
  measured in disease-relevant human cell models.
- **Pre-validated starting chemistry**: Every molecule input already
  demonstrates potent, selective, cell-based activity.
- **A tiered path to the clinic**: FDA-approved compounds, late-stage
  clinical assets, and novel molecules deliver staggered proof-of-concept
  readouts.

## Pipeline

A tiered pipeline of FDA-approved compounds, late-stage clinical assets,
and novel molecules — designed for staggered proof-of-concept and capital
efficiency. Five disease programs spanning neurology and neuromuscular
disease:

- **Autism Spectrum Disorder** — Signature Reversal — IND Enabling Studies
- **Facioscapulohumeral Muscular Dystrophy (FSHD)** — Signature Reversal —
  Candidate Selection
- **Huntington's Disease** — Single Gene Targeting (MSH3) — Lead Optimization
- **Myotonic Dystrophy** — Single Gene Targeting (MSH3) — Lead Optimization
- **Leigh Syndrome** — Signature Reversal — Preclinical

## Team

Led by Co-founder and CEO Chris Moxham, PhD (more than two decades of
experience, a dozen molecules to clinic across 7 therapeutic areas). Our
team includes scientists, engineers, and leaders in computational biology,
chemistry, and drug discovery.

- [Who We Are](https://www.transcriptabio.com/who-we-are)

## Contact

- [Contact Us](https://www.transcriptabio.com/contact-us)
- [Careers](https://www.transcriptabio.com/careers)
- [News](https://news.transcriptabio.com/) (Substack)
- [LinkedIn](https://www.linkedin.com/company/transcriptabio/)