# Development of a novel disease-modifying glycan therapeutic for early at-home intervention of acute vaso-occlusive crisis in sickle cell disease

> **NIH NIH R44** · IHP THERAPEUTICS, INC. · 2023 · $301,480

## Abstract

PROJECT SUMMARY/ABSTRACT
Current therapies for sickle cell disease largely fail to meet patient needs, particularly for the management of
acute pain crisis associated with this devastating disease. Healthcare systems are largely ill-equipped for
managing patients in the midst of these extremely painful and life-threatening events, leaving patients with
medical distrust and no effective treatment option. We are therefore developing IHP-100 for at-home self-
management of pain crisis, thus empowering patients and providing significant freedom from disease. IHP-100
is a novel P-selectin and complement inhibitor that is administered by subcutaneous injection at the onset of
early symptoms of pain crisis. This is a major paradigm shift in care that builds on our recent understanding of
sickle cell pathology and incorporates direct patient feedback to provide a truly effective treatment.
IHP-100 is a novel glycan-based therapeutic, designed to target the key underlying pathology of vaso-occlusive
crisis (VOC). VOC is multi-factorial and includes both cellular adhesion via selectins as well as complement
activation. Glycans are a prime drug class for such complex pathologies given their pleiotropic activities. We
have therefore engineered IHP-100 as a single agent that potently inhibits P-selectin as well as complement.
IHP-100 is therefore highly differentiated both in its biological activity and in its treatment paradigm at the earliest
symptom of pain crisis.
We have shown that IHP-100 inhibits P-selectin mediated cell binding to inflamed endothelium, inhibits
complement-mediated cell lysis, and reduces vaso-occlusions in the Townes sickle cell disease mouse model
of VOC. In the proposed work, we will optimize the dose level for in vivo efficacy, transfer manufacturing and
analytical testing to a GMP facility, and complete non-clinical IND enabling dose-range finding studies. We will
also further investigate mechanisms of IHP-100 in a P-selectin-deficient Townes sickle cell mouse model, and
establish an adhesion-based biomarker strategy for clinical development.

## Key facts

- **NIH application ID:** 10603870
- **Project number:** 1R44HL167473-01
- **Recipient organization:** IHP THERAPEUTICS, INC.
- **Principal Investigator:** John Paderi John Paderi John Paderi
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2023
- **Award amount:** $301,480
- **Award type:** 1
- **Project period:** 2023-03-05 → 2023-09-01

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10603870

## Citation

> US National Institutes of Health, RePORTER application 10603870, Development of a novel disease-modifying glycan therapeutic for early at-home intervention of acute vaso-occlusive crisis in sickle cell disease (1R44HL167473-01). Retrieved via AI Analytics 2026-05-25 from https://api.ai-analytics.org/grant/nih/10603870. Licensed CC0.

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