Science
Restoring Homeostasis
Our name honors Santiago Ramón y Cajal, the scientist and artist whose pioneering work transformed biology and revealed the intricate architecture of life. We carry his spirit forward by developing medicines that restore balance to the body’s most essential and complex networks - beginning with iron homeostasis, a tightly regulated system, the disruption of which drives serious systemic and neurological diseases.
Iron Balance at the Core of Biology
Iron is fundamental to human biology, powering cellular energy production, red blood cell creation, oxygen transport, and brain function. Because of its central role, iron homeostasis is regulated with extraordinary precision. When that balance breaks down, the consequences are far-reaching. Iron deficiency, iron overload, and functional iron deficiency (where iron is pathologically trapped within cells or tissues) have each been recognized as major causes of morbidity and mortality worldwide. Preserving iron balance across a lifetime remains one of medicine’s most persistent and unsolved challenges.
Systemic Disease
Disrupted iron handling underlies a range of serious systemic conditions. In anemias of inflammation, chronic inflammation traps iron within tissues, starving the hematopoietic system and preventing red blood cell production. Patients are left fatigued and anemic despite adequate or elevated body iron stores. In iron overload disorders, including hereditary hemochromatosis, beta-thalassemia, and transfusional iron overload, excess iron accumulates in organs over time, leading to progressive toxicity, long-term complications, and increased mortality. Together, these conditions illustrate the broad and damaging impact of disrupted systemic iron homeostasis.
Neurological Disease
Iron is essential in the brain, supporting mitochondrial energy production, neurotransmitter synthesis, and myelination. Even small disruptions can impair neuronal health and brain function. In Parkinson’s disease, growing evidence suggests that functional iron deficiency, due to chronic inflammation, genetic risk, and environmental factors, drives mitochondrial dysfunction, impaired dopamine synthesis, and neuronal loss. Similar imbalances may contribute to Friedreich’s ataxia and other neurodegenerative diseases. Impaired iron handling has long been recognized as a hallmark of neurodegeneration, underscoring the importance of restoring iron balance in the central nervous system.
A New Class of Medicines: Iron Mobilizers
Cajal is pioneering a new therapeutic approach with iron mobilizers - small molecules that transport iron across membranes and into the compartments where it is required.
By moving iron down natural concentration gradients, they offer a physiologic means of restoring equilibrium. This mechanism enables iron mobilizers to bypass disease-driven bottlenecks, including chronic inflammation, altered iron transporter expression, and lysosomal dysfunction, that disrupt normal iron regulation. We are advancing both peripherally restricted and brain-penetrant compounds, building a pipeline to address diseases rooted in iron imbalance across the body and brain. With this first-in-class mechanism, we aim to correct iron dysregulation at its source.
Our Lead Indication: Anemias of Inflammation
Anemia is a common complication of chronic inflammatory diseases, including chronic kidney disease, myelofibrosis, cancer, and inflammatory bowel disease, and is associated with worse outcomes and higher mortality.
Inflammation elevates hepcidin, a hormone that suppresses the iron transporter ferroportin, blocking both intestinal absorption and tissue recycling of iron. As a result, iron supply to the hematopoietic system is restricted, impairing red blood cell production. Iron mobilizers restore iron absorption and release iron sequestered in tissues, offering the potential for a more effective, durable, and oral therapy.
Transform the future with us
