Scientist using a microscope for close-up research in a laboratory.Scientist using a microscope for close-up research in a laboratory.

Targeting disease drivers to create transformational medicines.

PROGRAM
INDICATION
PRECLINICAL
EARLY CLINICAL
LATE CLINICAL
PROGRAM
Porphyria
Biologic
INDICATION
Erythropoietic Porphyrias
(EPP and XLP)
PRECLINICAL
EARLY CLINICAL
LATE CLINICAL
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Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP) are inherited disorders of heme metabolism resulting in the building of the immediate precursor to heme, a molecule called Protoporphyrin IX (PPIX). Once in circulation, PPIX drives painful skin photosensitivity, liver injury, and damage in other tissues.



Our approach is to directly remove PPIX from circulation using our proprietary porphyrin-scavenging technology. By removing this disease-causing molecule, we hope to create a potent disease-modifying treatment for patients.
PROGRAM
Heme
Biologic
INDICATION
Oncology‍, Heme Injury
PRECLINICAL
EARLY CLINICAL
LATE CLINICAL
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We are developing heme-sequestering proteins for indications driven by excess free heme, including kidney injury, hemolysis, and cancer.

Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP)

EPP and XLP are rare genetic disorders that affect the body's ability to produce heme leading to an excessive buildup of protoporphyrin IX (PPIX), a heme intermediate. PPIX is highly toxic and photoreactive causing liver damage and severe, painful skin reactions upon exposure to sunlight.



With no approved disease-modifying treatments, patients take extreme measures to avoid sunlight. Upon sunlight exposure, patients may experience disabling pain attacks that can last for days and cause chronic skin lesions and scarring. Additionally, many patients experience liver disease, with severe cases requiring transplantation.
Back view of a child reaching toward bubbles in a bright, sunlit garden, symbolizing hope and resilience for patients with rare genetic disorders like EPP and XLP.
Close-up of a researcher in gloves handling a blood sample, symbolizing the study of heme's role in disease progression and innovative therapeutic approaches.

Heme in disease

Excess heme released due to tissue damage or disease exerts toxic effects on multiple organs. Free heme—that is, heme not bound to proteins—is toxic because it induces oxidative stress through the generation of reactive oxygen species (ROS). Under normal conditions, proteins such as haptoglobin and hemopexin neutralize free heme; however, these defenses can be easily overwhelmed during disease. Consequently, heme-induced injury in various pathological states may lead to renal, cardiac, and hepatic damage.

Additionally, an increased rate of heme consumption and metabolism fuels cancer growth and progression. As a critical component of the electron transport chain, heme is essential for mitochondrial energy production. Several cancer types have been shown to rely on enhanced heme metabolism, actively modulating heme pathways to meet their heightened energy demands.

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A smiling woman enjoying the sunlight by the beach, symbolizing hope and the transformative impact of innovative medical treatments.