Transcripta Bio

Transcripta Bio is a biotech company that facilitates breakthrough drug discoveries for rare diseases. The company's platform empowers patient organizations to orchestrate a comprehensive research strategy, allocate resources, and accelerate execution by outsourcing early R&D steps. These include collecting and banking tissue samples, creating cell lines and model organisms, running drug screens, and matching patients with existing biotech assets.

Transcripta Bio's proprietary Drug-Gene Atlas maps molecular structures and drug actions to the entire cell transcriptome, revealing therapeutic candidates that rebalance gene expression to treat diseases. The company leverages artificial intelligence (AI) modeling through its Conductor AI suite to discover and predict the effects of new drugs at the transcriptomic scale.

By focusing on diseases where causality is unequivocally established by human genetics, Transcripta Bio utilizes the universe of small molecules that have consistently led to successful drugs. The company's approach dramatically reduces the time and money required for drug discovery, not just for rare diseases but for any drug that treats disease by changing gene expression. As of April 2024, Transcripta Bio had moved five FDA-approved drugs into single-patient studies for new indications, which would otherwise have taken five to eight years.