Transcripta Bio

Transcripta Bio is a California-based drug discovery company that measures disease and drug effects in the same units — transcriptomic data — so that compounds can be matched to the gene-expression signature of a disease. Rather than assessing biology one gene at a time, the company profiles the full transcriptome across cell types, characterizing what a disease does to cells and what a compound perturbs, then uses machine learning to identify molecules whose effects invert the disease signature.

The platform has three components. The Disease Signature Atlas profiles patient-derived tissue with single-cell RNA sequencing and bulk sequencing across the full transcriptome, fusing expression data with human genetics and mechanism to build validated disease signatures. The Drug-Gene Atlas uses DRUG-seq to measure the complete transcriptomic response of selected compounds at dose in disease-relevant human cells, including iPSC neurons, motor neurons, fibroblasts, muscle progenitors, cardiomyocytes and hepatocytes, rather than immortalized cancer lines. Conductor AI is a set of machine-learning models trained on both atlases that ranks reversal candidates by a reversal score, accounts for combinations and cell type, and performs virtual screening of billions of on-demand synthesizable compounds to design new molecules for a target signature; wet-lab validation results feed back into the models.

The company describes a five-program pipeline across two modalities (signature reversal and single-gene targeting): autism spectrum disorder (IND-enabling studies), facioscapulohumeral muscular dystrophy (candidate selection), Huntington's disease and myotonic dystrophy (both lead optimization, targeting MSH3 suppression to address somatic repeat expansion), and Leigh syndrome (preclinical). Its most advanced work involves entrectinib, an FDA-approved oncology drug being repositioned for a genetically defined ASD subtype linked to 19q12 deletions, described in a peer-reviewed Scientific Reports publication.

Founding story

The company states it was founded by people personally waiting on better answers from drug discovery, around the conviction that seeing biology at the resolution where disease occurs makes the choice of medicine tractable. Co-founder, CEO and Chief Scientific Officer Chris Moxham previously worked on transcriptomics-driven discovery at Eli Lilly and Fulcrum Therapeutics.

Business model

Transcripta Bio develops its own therapeutic programs using an internal discovery platform and also co-discovers with external organizations, partnering with pharma, biotech, disease foundations, academic labs and research centers to apply the Disease Signature and Drug-Gene atlases to additional programs. It has entered non-equity collaborations, including funded drug screening with a patient advocacy group and a research collaboration with Microsoft Research.

Traction

The company reports more than 200 million experiments completed in its labs over roughly two years prior to its April 2025 unveiling, early results across several rare diseases without existing treatments, a five-program pipeline with autism spectrum disorder in IND-enabling studies, a peer-reviewed Scientific Reports publication on entrectinib repositioning in 19q12 autism spectrum disorder, inclusion in the TIME100 Most Influential Companies of 2024, and roughly $34 million in total disclosed funding.

Latest developments

Transcripta Bio raised $24 million in follow-on Series A funding from new investors Mayo Clinic and Omnimed alongside existing backers JAZZ Venture Partners, BlueYard Capital and life sciences family offices, bringing total disclosed funding to about $34 million. The capital is earmarked for completing IND-enabling studies and preparing for clinical trials in autism spectrum disorder and FSHD.