From rare-disease research to applied AI

ORPHARMA supports research questions. Intelligence organizes evidence. Foundry develops AI models and practical systems for inspection and image analysis.

WHERE WE BEGAN

Why? Why revisit existing medicines?

Small patient populations make new rare-disease treatments costly and slow to develop. ORPHERA began by exploring new possibilities for existing approved medicines.

We organize candidates and evidence through gene-expression analysis and explainable mechanism assessment. Foundry applies this AI development experience to inspection and image-analysis workflows in other organizations.

~10,000Known rare diseases
95%Rare diseases without an FDA-approved treatment

Source: NIH/NCATS · 2023 fact sheet

RESEARCH & DEVELOPMENT

Rare-disease research and model evaluation

The figures below describe retrospective CREST analysis and ORPHARMA model evaluation. They do not measure Foundry application performance.

TECHNOLOGY 01

CREST · Gene-expression-based candidate discovery

CREST compares disease and drug gene-expression patterns to prioritize candidates for further research.

Retrospective screening placed drugs studied in preclinical and clinical research within the top 1%.

10th
of 1,170 candidatesTop 0.85%

Spironolactone × Duchenne muscular dystrophy (DMD)

These retrospective rediscovery cases connect computational rankings with prior research, suggesting potential for prioritizing new drug-repurposing candidates.

Internal retrospective comparison with prior research; not validation of predictive performance on new candidates or of clinical efficacy.

TECHNOLOGY 02

ORPHARMA · Explainable mechanism assessment model

ORPHARMA · After trainingGPT-5.6-solORPHARMA · Before training

Standard testn = 119

Gene-split testn = 296

Drug-split testn = 249

Gene & drug-split testn = 46

On smaller screens, scroll horizontally to compare all conditions.

Absolute scores · All bars use a 0–100% scale

ORPHERA · THREE AREAS

Research, assess, and put AI to work

Tools for rare-disease research, evidence analysis, and AI development for your organization.

01 · RESEARCH AI

ORPHERA ORPHARMA

A research AI agent that connects literature, drug, and clinical evidence to support candidate exploration and study planning. In development

Concept of android hands actively typing with both hands beneath a frontal monitor showing ranked drug candidates and indication-specific numerical scores
AI-generated research concept · Fictional text and data, not an actual product screen.

02 · RESEARCH ANALYSIS

ORPHERA Intelligence

Watch tracks research developments, Compass examines regulatory pathways, and Signal organizes drug- and disease-specific evidence.

Three separate A4 report sheets on a desk showing research summaries, experimental data and evidence review
AI-generated report concept · Text and data are fictional, not actual research findings.

03 · CUSTOM AI

ORPHERA Foundry

We develop and fine-tune models, build agents and AI solutions for defect inspection, video and image analysis, and document search.

Foundry workflow: organizational data and work criteria, custom AI development, and tools for everyday work
AI-generated application concept · Not a deployed product or performance claim.