Focusing on the fundamentals:
heme and iron
We are deliberate in targeting pathways that play fundamental and
well-validated roles in red blood cell (RBC) biology and whose functions
have been confirmed in humans. We believe this focus enables us to
develop therapies that will have a meaningful impact on patients’ lives.
Our R&D strategy focuses on pathways that will have a clear impact on red blood cell development (or erythropoiesis). Our programs are designed to modulate two critical processes required for functional RBCs: heme biosynthesis and iron metabolism. By manipulating these central components of erythropoiesis, our programs can address a wide range of hematologic diseases.
Importantly, we select our molecular targets based on confirmed biological function in humans. Each of our programs is supported by genetic or clinical evidence validating the mechanism of action in humans. We believe this provides the best insight into predicting the biology of our targets and potential for therapeutic benefit to patients.
Our Technology Platforms
We have established distinct programs to modulate these
fundamental pathways. We affect heme synthesis by
targeting the supply of intracellular glycine, which
is a key building block of heme. Our iron homeostasis
technology targets hepcidin, the master regulator of iron
availability in the body, and we have developed separate
programs designed to suppress and induce hepcidin production.
Heme Synthesis Modulation
Heme is a critical component of hemoglobin and is required
for erythropoiesis. It is synthesized through a multi-step
enzymatic process that requires a high demand of intracellular
glycine, a necessary precursor for heme. Our lead program,
bitopertin, limits the uptake of glycine by developing erythrocytes
via glycine transporter 1 (GlyT1) and by doing so, has been
shown in the clinic to control heme synthesis. We intend
to develop bitopertin to treat erythropoietic porphyrias,
a family of diseases caused by dysregulated heme synthesis,
and other conditions associated with abnormal erythropoiesis.
Hepcidin Pathway Modulation
Iron is a critical component of hemoglobin and required for
erythropoiesis. Hepcidin is the primary regulatory hormone
of iron homeostasis and plays a central role in controlling
how iron is absorbed, stored and utilized. Iron metabolism
is a critical and carefully controlled process and when it
becomes dysregulated, results in a wide range of serious,
debilitating and potentially fatal conditions. Disc is a leader
in the development of therapeutics that target the hepcidin
pathway, with distinct programs designed to suppress
hepcidin (increase iron) or increase hepcidin (reduce iron)
to fully address the range of hematologic disorders
associated with iron dysfunction.