Drugs to regulate hepcidin expression within the body

Disc Medicine Inc. is focused on developing innovative therapies for hematologic diseases, particularly those related to heme synthesis and iron metabolism. The company has established a pipeline of first-in-class therapies, including bitopertin, an oral selective inhibitor of glycine transporter 1 (GlyT1), which aims to treat erythropoietic porphyrias by modulating heme synthesis. This therapy is designed to address rare genetic disorders caused by dysregulated heme production. Additionally, Disc Medicine is advancing DISC-0974, an injectable monoclonal antibody that inhibits hemojuvelin (HJV) to suppress hepcidin production, targeting various forms of anemia, including those associated with myelofibrosis and chronic kidney disease.

The company’s research and development strategy emphasizes the modulation of critical biological pathways that influence red blood cell (RBC) development. By targeting heme biosynthesis and iron homeostasis, Disc Medicine aims to create therapies that can significantly impact patient outcomes. Their approach is grounded in validated biological functions and genetic evidence, ensuring that their therapeutic targets are relevant to human physiology. This commitment to understanding and manipulating the underlying mechanisms of hematologic diseases positions Disc Medicine as a leader in the field, with a focus on delivering meaningful therapies to patients suffering from these conditions.

United States

Disc Medicine is a biopharmaceutical company dedicated to transforming the lives of patients with hematologic disorders. We are building a unique portfolio of innovative, first-in-class therapeutic candidates that affect fundamental pathways of red blood cell biology.

Genetic Diseases

Hematologic Diseases

Disc Medicine Inc

erythropoietic porphyrias

X-linked protoporphyria

Bitopertin is an investigational, clinical-stage, orally-administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis.&nbsp;

Bitopertin

Anemia

Chronic kidney disease

Myelofibrosis

DISC-0974 is an investigational anti-hemojuvelin monoclonal antibody (mAb) and is designed to suppress hepcidin production and increase serum iron levels in patients suffering from anemia of inflammation.

DISC-0974

Blood cancer

Cancer

Polycythemia vera

DISC-3405 (formerly MWTX-003)

Rare genetic disease

Bitopertin is an investigational, clinical-stage, orally-administered inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate heme biosynthesis.

Age-related macular degeneration

Wet age-related macular degeneration

Gemini’s &nbsp;lead program, GEM103, is a pioneering precision medicine approach, &nbsp;targeting trial enrichment with genetically-defined patients. GEM103 &nbsp;targets a genetically-defined subset of age-related macular degeneration &nbsp;(AMD) patients with complement dysregulation. Of the 15 million dry AMD &nbsp;patients, approximately 40% (or six million) have variants in the &nbsp;complement factor H (CFH) gene. Such loss-of-function variants are &nbsp;associated with increased dry AMD disease risk. GEM103 is believed to be &nbsp;the first ever recombinant native complement modulator, full-length &nbsp;recombinant complement factor H (rCFH) protein. When delivered by &nbsp;intravitreal injection, GEM103 has the potential to address unmet &nbsp;medical need in genetically-defined subsets of AMD patients by &nbsp;circumventing dysfunctional CFH loss-of-function variants and slowing &nbsp;the progression of their retinal disease.
<a href="https://investors.geminitherapeutics.com/news/news-details/2021/Gemini-Therapeutics-Announces-FDA-Fast-Track-Designation-Granted-for-GEM103-for-the-Treatment-of-Dry-Age-Related-Macular-Degeneration-AMD-in-Patients-with-Complement-Factor-H-CFH-Loss-of-Function-Gene-Variants/default.aspx">Source</a>

Disc Medicine Inc

Company Details