# Immunotherapeutic for ATTR/AL Cardiac Amyloidosis

> **NIH NIH R43** · AURORA BIO INC · 2020 · $249,752

## Abstract

Cardiac amyloidosis is characterized by myocardial accumulation of protein fibrils in the heart and the
most common types are wild-type and hereditary transthyretin (ATTR) and light-chain (AL) amyloidosis. It is a
severe, progressive and often lethal disorder. We believe it is possible to develop a pan amyloid therapeutic
that can treat all cardiac amyloidosis and can target patients irrespective of whether they have AL, wild-type or
mutant ATTR amyloidosis. We have identified a novel family of synthetic, polybasic peptides that specifically
detect a unique version of heparan sulfate in amyloid deposits and binds to the surface of diverse protein amy-
loid fibrils (Fig 1). Heparan sulfate, which is a major and ubiquitous component of all amyloid deposits is struc-
turally distinct from the heparan sulfate normally found in the extra-cellular matrix. It is present in amyloid in a
much higher density and is hypersulfated, and can therefore be specifically targeted. The peptides, p5 and the
elongated form p5+14, were shown to bind to amyloid deposits in vitro and in vivo in a murine model. A radio-
labeled version designated 124I-p5+14, is currently being developed as a pan-amyloid imaging agent for the
detection, quantification and monitoring of multi-organ amyloidosis including cardiac amyloidosis in human
subjects.
 We propose to develop and characterize a humanized version of the p5 antibody-fusion (termed hIgp5),
in which the p5 peptide is fused directly to the humanized antibody light chain. The new antibody-peptide fu-
sion construct will be quantitatively evaluated in various in vitro ATTR and AL amyloid binding studies. Addi-
tionally, we will employ florescence based methods of measuring their ability to induce uptake of amyloid in
vitro. Mice bearing localized fluorescent human amyloidomas, which can be non-invasively monitored by opti-
cal imaging, will be used for in vivo studies.
 Our goal is to develop a pan amyloidosis therapeutic agent to 1) bind all types of amyloid 2) leverage
multiple binding sites 3) remain highly specific 4) serve as a backbone for therapeutics and 5) utilize for imag-
ing as a disease biomarker and a biomarker to monitor outcomes from therapeutic intervention. Our research
strategy could lead to a pan amyloid antibody therapeutic that is highly effective in clearing amyloid fibrils from
the heart and a trailblazer to a transformative therapy for cardiac amyloidosis patients.

## Key facts

- **NIH application ID:** 10081324
- **Project number:** 1R43HL154918-01
- **Recipient organization:** AURORA BIO INC
- **Principal Investigator:** Suganya Selvarajah
- **Activity code:** R43 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $249,752
- **Award type:** 1
- **Project period:** 2020-09-16 → 2021-08-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10081324

## Citation

> US National Institutes of Health, RePORTER application 10081324, Immunotherapeutic for ATTR/AL Cardiac Amyloidosis (1R43HL154918-01). Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/grant/nih/10081324. Licensed CC0.

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