Articles from GondolaBio

GondolaBio, a clinical-stage biopharmaceutical company developing novel medicines for patients living with genetic diseases, and the Children’s Tumor Foundation (CTF), NF’s global drug discovery engine focused on neurofibromatosis and schwannomatosis (collectively known as NF), today announced a partnership to advance GondolaBio’s preclinical neurofibromatosis type 1 (NF1) program.

GondolaBio, a clinical-stage biopharmaceutical company developing novel medicines for patients living with genetic diseases, on behalf of its affiliate Portal Therapeutics, today announced positive data from its Phase 2a GATEWAY trial of PORT-77 in patients with erythropoietic protoporphyria (EPP). PORT-77 is a novel investigational oral, small molecule ABCG2 inhibitor designed to treat EPP and X-linked protoporphyria (XLP), two rare genetic disorders characterized by severe, lifelong photosensitivity and liver complications.

GondolaBio, a clinical-stage biopharmaceutical company developing novel medicines for patients living with genetic diseases, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Orphan Drug Designation and Fast Track Designation to its affiliate, Portal Therapeutics, for PORT-77, an investigational oral, small molecule ABCG2 inhibitor, for the treatment of EPP and XLP.