Erythropoietic porphyria

Disc Medicine Announces Collaboration with National Institutes of Health for Phase 2 Clinical Study of Bitopertin in Patients with Diamond-Blackfan Anemia (DBA)

Retrieved on: 
Tuesday, March 21, 2023
Diamond, National Heart, Lung, and Blood Institute, DBA, Head, Clinical trial, CRADA, Erythropoietic porphyria, Heme, Diamond–Blackfan anemia, Bangladesh Technical Education Board, Trial of the century, Development, NHLBI, IRON, Cooperative research and development agreement, Patient, NIH, Disease, Pharmaceutical industry, Disc

The study will be conducted and funded by the NIH under a Cooperative Research and Development Agreement (CRADA) and is expected to initiate mid-year 2023.

Key Points: 
  • The study will be conducted and funded by the NIH under a Cooperative Research and Development Agreement (CRADA) and is expected to initiate mid-year 2023.
  • “Disc now has ongoing development programs of bitopertin in both erythropoietic porphyria and DBA.
  • The phase 2 study will be a pilot, single-arm, dose-escalation trial of bitopertin in DBA patients who either have steroid-refractory and/or relapsed disease or are unable to tolerate systemic corticosteroids.
  • Upon completion of the main treatment period, patients may continue on extended treatment within the trial.

Portfolio Company Disc Medicine Enters Definitive Merger Agreement with Gemini Therapeutics

Retrieved on: 
Wednesday, August 10, 2022
Diamond, Chronic kidney disease, Sickle cell disease, Sodium- and chloride-dependent glycine transporter 1, Hepcidin, Erythropoietic porphyria, Inflammation, Industry, DBA, AbbVie, Microdeletion syndrome, Genetic disorder, Cancer, Medicine, Diamond–Blackfan anemia, Hope, Patient, TMPRSS6, Biotechnology, Degenerative disease, Disc, Anemia, Polycythemia, Iron overload, Ruxolitinib, Hematologic disease, Thalassemia, Hereditary haemochromatosis, Pharmaceutical industry, HJV

Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders.

Key Points: 
  • Disc Medicine is a clinical-stage biopharmaceutical company that is dedicated to transforming the lives of patients with hematologic disorders.
  • Disc is building a portfolio of innovative, first-in-class therapeutic candidates that affect fundamental pathways of red blood cell biology.
  • Disc Medicine is committed to developing treatments that empower and bring hope to the many patients who suffer from hematologic disease.
  • Disc has a clinical-stage development pipeline composed of investigational product candidates that affect heme biosynthesis and iron metabolism.

Disc Medicine Expands Hematology Pipeline with Worldwide Licensing Agreement for Bitopertin, a First-in-Class Modulator of Heme Synthesis

Retrieved on: 
Thursday, May 27, 2021
Porphyrias, Chemical compounds, Branches of biology, Chemistry, Glycine reuptake inhibitors, Bitopertin, Heme, Erythropoietic porphyria, Sodium- and chloride-dependent glycine transporter 1, Cytochrome P450 reductase

CAMBRIDGE, Mass., May 27, 2021 /PRNewswire/ --Disc Medicine, Inc. announced today that it has entered into an exclusive worldwide licensing agreement withF.

Key Points: 
  • CAMBRIDGE, Mass., May 27, 2021 /PRNewswire/ --Disc Medicine, Inc. announced today that it has entered into an exclusive worldwide licensing agreement withF.
  • Hoffmann-La Roche Ltdfor the development and commercialization of bitopertin, an orally administered GlyT1 inhibitor with demonstrated effects on the heme biosynthesis pathway.
  • "Importantly, the well-established clinical safety profile of bitopertin will allow us to move rapidly into patient studies of several serious, hematologic diseases."
  • Erythropoietic porphyrias (EPs) are a family of ultra-rare, debilitating and potentially life-threatening diseases caused by mutations that affect the heme synthesis pathway.