Rare Disease · AI Drug Repurposing

A World Where No Disease
Is Too Rare.

We build a rare disease drug pipeline using AI. Our CREST engine identifies repurposing candidates from approved drugs — faster, cheaper, and with higher clinical success rates.

7,000+
Known rare diseases
95%
Have no approved treatment
300M
Patients worldwide
The Problem
The bottleneck isn't the algorithm. It's the economics.
Traditional drug development costs $2.6B and takes 15 years. For rare diseases with tiny patient populations, the ROI math never works. Drug repurposing changes the equation.
$2.6B
Average new drug development cost
15yr
Average development timeline
3–6yr
Repurposing timeline — using drugs with known safety profiles
$243B
Global orphan drug market (2026)
Core Technology
CREST Engine
Cell-type Resolved Expression Screening for Therapeutics — our proprietary algorithm that matches rare disease gene expression patterns against a curated library of approved drug signatures.
01

Disease Signature

Calculate the gene expression change pattern from patient transcriptome data — which genes go up, which go down.

02

Drug Library Matching

Compare against a curated library of approved drug signatures using proprietary drug-disease scoring.

03

Reversal Ranking

Rank drugs that reverse the disease pattern. Statistical validation via 10,000-iteration permutation test (p=0.001).

04

Candidate Delivery

Deliver ranked repurposing candidates with evidence packages — ready for regulatory and IP strategy.

CREST engine drug ranking output
CREST output — ranked drug candidates by CREST score across rare disease signatures
Validation

Validated Results

Sirolimus × Progeria (HGPS)
Rank 4 / 1,170
Top 0.3% — blind recovery of a clinically confirmed drug. Permutation p = 0.001.
Spironolactone × Duchenne MD (DMD)
Rank 10 / 1,170
Independent validation on a second rare disease with published efficacy evidence.
Pipeline Validation
3 diseases · 6 engine-validated candidates
HGPS, DMD, NF1 validated. CREST independently identified drugs already in clinical trials — blind recovery across multiple diseases.
Platform
Pipeline-first. Services bridge the gap.
While our pipeline matures, CREST powers a suite of intelligence services — generating early revenue and validating the engine with real-world client data.
W
Intelligence

ORPHERA Watch

Competitive intelligence for rare disease drug repurposing. Track orphan designations, clinical trials, patents, and publications — automatically.

FDA Orphan Drug DB monitoring
ClinicalTrials.gov alerts
KIPRIS + Global patent tracking
Monthly competitive landscape report
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C
Regulatory

ORPHERA Compass

Regulatory pathway navigator for repurposed drugs. Input a drug–disease pair, get a structured route analysis with patent and clinical cross-checks.

505(b)(2) / ODD pathway simulation
Real-time patent barrier check
Prior clinical data availability
Decision memo auto-generation
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S
Evidence

ORPHERA Signal

Instant Go/No-go cards for drug–disease combinations. Literature, patents, clinical data, and competitive status — scored and standardized.

Standardized evidence cards
Go/No-go scoring (transparent logic)
Batch screening (10+ combos)
Comparison view across candidates
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AI Research Agent

ORPharma

Rare disease-native AI research agent. Fine-tuned on rare disease literature, omics, and regulatory data. Covers literature, omics, experimental design, and regulatory strategy.

PubMed · ChEMBL · ClinicalTrials On-premise available Custom fine-tuning
Early Access →
✓Fine-tuned on rare disease literature, omics, and regulatory data — HGPS, DMD, NF1 and growing
✓Live tool-calls to PubMed, ChEMBL, ClinicalTrials — not static training data
✓Regulatory strategy — ODD, 505(b)(2), NCIE pathway guidance built in
✓On-premise deployment for IRB-compliant environments
✓Custom fine-tuning on your proprietary pipeline data
Why Orphera
Built different from day one.
Every competitor feeds the same public databases into their models. Our approach is fundamentally different.
01 / Method

Cell-type resolved analysis

We match disease signatures at the cell-type level, not tissue-level bulk data. This produces 250× stronger signal — the key factor behind Sirolimus rank 4.

02 / Rigor

Built-in quality gate

Our pipeline self-diagnoses. If patient expression patterns are inconsistent (CREST score < 0.8), the system automatically flags the disease as not suitable — preventing false predictions.

03 / Focus

Rare disease specialized

Not a general-purpose AI drug discovery platform. Every design decision — from data curation to regulatory knowledge — is optimized for orphan drug economics.

04 / Coverage

Full value chain coverage

Discovery (CREST) → Intelligence (ORPHERA Watch) → Regulatory (ORPHERA Compass) → Evidence (ORPHERA Signal). No competitor covers the full rare disease repurposing pipeline.

Every child deserves a chance.

We partner with biotech companies, patient foundations, and investors. For custom drug-disease screening, ask about our FFS discovery contracts.

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Accelerating hope for rare disease patients.
A child looking up with hope