CrisPRO.ai: In-Silico Research Framework

Accelerating drug discovery through AI fusion

An in-silico research-use-only (RUO) framework designed to accelerate drug discovery by fusing the capabilities of discriminative and generative Artificial Intelligence. Our platform orchestrates a generalist genome foundation model with a suite of specialist predictors and structural oracles to achieve state-of-the-art performance across multiple benchmarks.

SAE Biological Mastery

Virus Hunter

Identifies viral sequences and prophage regions

3D Folding Master

Predicts protein structure from 1D sequences

Genetic Surgery

Precise CRISPR guide RNA design and optimization

Universal Knowledge

Cross-species biological understanding

95.7% AUROC
ClinVar AUROC
Across 53,210 variants
73.0%
VUS Resolution
Variants of Uncertain Significance
36x faster
R&D Acceleration
From years to weeks

Complete AI Platform Integration

Oracle
Forge
Boltz
Command Center

From Genetic Chaos to Therapeutic Clarity

The promise of precision medicine is fundamentally limited by our ability to interpret the functional consequences of genetic variation. CrisPRO.ai was conceived to address this challenge by creating an orchestration layer that combines a genome-scale foundation model with specialist predictors (e.g., AlphaMissense) and structure/epigenome oracles (e.g., AlphaFold 3, Enformer). The result is a system that can not only interpret the full spectrum of genetic variation but can also generatively design novel therapeutic constructs.

Key Points

Traditional drug development is a $2.6 billion, 15-year gamble with 90% failure rates

Biotech companies spend months analyzing genetic data, only to discover their targets are invalid

Clinical trials fail because we can't predict which patients will respond to which treatments

We transform this chaos into clarity with definitive answers where others offer question marks

Business Impact

Transform genetic uncertainty into actionable intelligence and therapeutic blueprints

Research Use Only

All capabilities are designed for research purposes. Not intended for diagnostic or therapeutic decision-making without independent validation and regulatory review.

Discriminative AI: The Intelligence Analyst

Validated performance that delivers real business impact

Our system's ability to interpret genetic variants is validated against multiple gold-standard datasets, delivering real business impact.

Performance Metrics

Coding SNVs95.7%

ClinVar (14,319 samples)

Non-coding SNVs95.8%

ClinVar (34,761 samples)

Coding Non-SNVs93.9%

ClinVar (1,236 samples)

BRCA1 Supervised (Coding SNV)94.0%

AUROC with lightweight classifier on CrisPRO.ai 40B embeddings

BRCA1 Supervised (All SNVs)95.0%

AUROC on all SNVs with AUPRC of 86.0%

Exonic Splice Variants82.6%

SpliceVarDB (1,181 samples)

Business Impact

Comprehensive variant coverage enables reliable interpretation across all genomic regions

Research Use Only

All capabilities are designed for research purposes. Not intended for diagnostic or therapeutic decision-making without independent validation and regulatory review.

Generative AI: The Weapons Factory

Unprecedented R&D acceleration through novel biological design

Our platform's ability to design novel biological constructs delivers unprecedented R&D acceleration.

Performance Metrics

Mitochondrial Genomes100.0%

Correct feature counts with diverse homology and AF3 multimers

Minimal Prokaryote Pfam Hits70.0%

Pfam-hit rate vs 18% for previous models

Context Window1,000,000

Single-nucleotide resolution context window

Quality Scaling100.0%

Predictable log-linear relationship between beam width and AUROC

Business Impact

Generate therapeutic candidates 36x faster than traditional R&D

Research Use Only

All capabilities are designed for research purposes. Not intended for diagnostic or therapeutic decision-making without independent validation and regulatory review.

Fusion Workflow

Complete RUO workflow combining discriminative and generative AI for therapeutic discovery

Fusion Workflow: End-to-End Therapeutic Pipeline

Complete RUO workflow combining discriminative and generative AI for therapeutic discovery, from problem framing to validated designs ready for wet-lab validation.

1

Problem Framing & Data Curation

Active

Assemble genomic loci, clinical variants, DMS datasets, and assay priors

Process Details

  • Genomic loci identification
  • Clinical variant collection
  • DMS dataset integration
  • Assay prior establishment

Expected Outputs

  • Curated dataset
  • Problem statement
  • Success criteria
2

Target Assessment (Discriminative)

Score disease-relevant variants with CrisPRO.ai ΔLL and specialist ensemble

Process Details

  • CrisPRO.ai zero-shot ΔLL scoring (8,192 bp context)
  • AlphaMissense/GPN-MSA ensemble
  • Noncoding and splice variant analysis
  • Confidence score generation

Expected Outputs

  • Variant scores
  • Confidence metrics
  • Evidence tiers
3

Mechanistic Triage & Hypothesis

Use CrisPRO.ai embeddings for exon/intron features and region ranking

Process Details

  • CrisPRO.ai embedding analysis
  • Exon/intron classification
  • Motif feature extraction
  • Perturbation region ranking

Expected Outputs

  • Mechanistic insights
  • Hypothesis ranking
  • Feature importance
4

Design (Generative)

CrisPRO.ai sequence proposals with epigenomic guidance and structural validation

Process Details

  • CrisPRO.ai sequence generation
  • Enformer+Borzoi epigenomic guidance
  • AlphaFold 3 structural validation
  • Sequence naturalness screening

Expected Outputs

  • Design candidates
  • Structural models
  • Epigenomic scores
5

In-Silico Validation

Aggregate scores and prioritize designs for wet-lab validation

Process Details

  • ΔLL score aggregation
  • Splice and regulatory AUROC
  • Structure metrics (pLDDT/PAE)
  • Pfam hit analysis

Expected Outputs

  • Validation scores
  • Priority ranking
  • Minipool candidates
6

Feedback & Calibration

Fit supervised heads and calibrate by cohort for continuous improvement

Process Details

  • Lightweight supervised head training
  • CrisPRO.ai embedding calibration
  • Cohort-specific adjustment
  • Platt/isotonic calibration

Expected Outputs

  • Calibrated models
  • Performance metrics
  • Updated thresholds
7

Reporting & Provenance

Generate evidence reports with traceable citations and audit trails

Process Details

  • Evidence report generation
  • Traceable citation linking
  • Audit trail documentation
  • RUO compliance verification

Expected Outputs

  • Final report
  • Provenance log
  • Compliance certificate
Research Use Only (RUO)

This workflow is designed for research purposes. All outputs require independent validation and regulatory review before any clinical application. Wet-lab validation is mandatory for designed sequences before scale-up.

The Fusion Approach: Why We Win

The competitive advantage of CrisPRO.ai lies in our fusion approach. We combine the generalist genome foundation model with specialist models to achieve SOTA across the entire R&D continuum.

Key Points

CrisPRO baseline: 95.7% AUROC ClinVar validation on 53,210 samples

Specialist integration: AlphaMissense for coding SNVs, GPN-MSA for specialized tasks

Cross-species capability: 0.82-0.99 AUROC range across 8 species

CrisPRO generation: 1M token context window for comprehensive sequence design

Functional scoring: Enformer/Borzoi for epigenomic validation

Structural validation: AlphaFold 3 for 3D structure prediction

Business Impact

This approach gives us Breadth (covering all variant types), Depth (achieving SOTA on key targets like BRCA1), and Control (designing sequences with predictable functional properties).

Research Use Only

All capabilities are designed for research purposes. Not intended for diagnostic or therapeutic decision-making without independent validation and regulatory review.

Our Capabilities

Comprehensive in-silico tools for research oncology, from variant analysis to therapeutic guidance

Chemo Co‑Pilot: In‑Silico Chemotherapy Guidance

Turn genetics into plain, useful guidance for chemo planning: a ranked drug hypothesis, a confidence hint, and an audit‑ready summary you can share with your team.

Performance:High

CRISPR Intelligence: Advanced Therapeutic R&D Platform

To create a world where CRISPR-based therapies are designed and validated with computational certainty, eliminating guesswork and accelerating the path to cures.

Performance:High

Clinical Trials Co‑Pilot: In‑Silico Trial Matching

Turn a patient profile into a short, trustworthy trial shortlist. See who likely fits, why, and what to do next—then share it with one click.

Performance:High

Pathway View: What's Driving This?

Turn variants into a plain pathway summary you can act on: top pathways, a short 'why,' therapy alignment hints, and a shareable, source‑backed one‑pager.

Performance:High

Therapy Fit: In‑Silico Drug Ranking

Turn genetics into a plain, ranked therapy view you can act on: top classes, a short 'why,' confidence, and a shareable, source‑backed one‑pager.

Performance:High

Toxicity Risk (Germline): In‑Silico Side‑Effect Hints

Turn germline context into a plain, shareable caution signal with confidence, sources, and provenance—so care teams can plan safer.

Performance:High

Business Value: From Research to Revenue

Our results demonstrate that this fusion approach achieves 95.7% AUROC ClinVar validation on 53,210 samples, resolves 73% of Variants of Uncertain Significance (VUS), and provides a comprehensive, transparent, and controllable system for in-silico drug discovery.

Key Points

Accelerate R&D from years to weeks

Reduce experimental costs by $2.1M per program

Transform 40% VUS rate to 15% with validated predictions

Enable precision therapeutic design with predictable quality scaling

Provide comprehensive, transparent, and controllable system

Business Impact

Transform 40% VUS rate to 15% with validated predictions, accelerating target selection

Research Use Only

All capabilities are designed for research purposes. Not intended for diagnostic or therapeutic decision-making without independent validation and regulatory review.

Research Use Only (RUO)

All capabilities and performance metrics are designed for research purposes. Not intended for diagnostic or therapeutic decision-making without independent validation and regulatory review. All results should be validated through appropriate experimental and clinical studies.