We're announcing HEM-001, our first hematology program—a novel small molecule approach to sickle cell disease designed entirely by our AI platform.

Sickle Cell Disease

Sickle cell disease (SCD) affects millions of people worldwide, causing severe pain crises, organ damage, and reduced life expectancy. While recent advances have brought new treatments, significant unmet need remains:

  • Crizanlizumab (anti-P-selectin antibody) reduces pain crises but requires monthly IV infusions
  • Voxelotor addresses sickling but has limited efficacy on pain
  • Gene therapies are promising but extremely expensive and complex to deliver

There's a clear need for oral, accessible therapies that address the underlying vaso-occlusive pathophysiology.

Our AI's Approach

Our AI Scientist analyzed the biology of vaso-occlusive crises and identified P-selectin as a compelling target—consistent with the clinical validation of crizanlizumab. But rather than pursuing another antibody, we asked: could we design a small molecule inhibitor?

The AI:

  1. Analyzed P-selectin structure and identified potential small molecule binding sites
  2. Generated novel chemical scaffolds predicted to inhibit P-selectin
  3. Optimized for oral bioavailability and drug-like properties
  4. Predicted patient populations most likely to respond

Program Differentiation

HEM-001 aims to deliver P-selectin inhibition through an oral small molecule with several potential advantages:

  • Oral administration: Daily pill vs. monthly IV infusion
  • Lower cost: Small molecules are inherently less expensive than biologics
  • Faster onset: Potential for use in acute pain crisis
  • Combination potential: Easy to combine with other SCD therapies

Our lead compounds show promising activity in biochemical and cellular assays.

Digital Patient Modeling

We're using our Digital Patient platform to:

  • Model vaso-occlusive crisis biology in silico
  • Predict which patients will respond best to P-selectin inhibition
  • Optimize dosing regimens for maximum efficacy
  • Identify potential biomarkers for clinical development

Progress and Timeline

HEM-001 has achieved several milestones:

  • Multiple novel chemical series with P-selectin inhibitory activity
  • Favorable ADMET profiles in computational and early experimental testing
  • Clear path to development candidate nomination

We're targeting IND-enabling studies within the next 18-24 months.

HEM-001 is in lead optimization. For partnership inquiries, contact partnerships@omic.ai

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