Articles from Glycomine, Inc.

Glycomine, Inc., a biotechnology company focused on developing transformative new therapies for orphan diseases, today announced a $115 million Series C financing to advance its lead candidate, GLM101, into a Phase 2b clinical trial. The financing was led by CTI Life Sciences Fund, funds managed by abrdn Inc., and Advent Life Sciences, alongside continued investment from existing investors, Novo Holdings, Sanofi Ventures, Abingworth, RiverVest Venture Partners, Sanderling Ventures, Chiesi Ventures, Remiges Ventures, and Asahi Kasei Ventures.

Glycomine, Inc., a clinical-stage biotechnology company focused on developing new therapies for serious unserved orphan diseases, today announced the appointment of Joshua Grass as Chairperson of the Board. Mr. Grass succeeds Christopher Starr, Ph.D., who will continue to support the company as a scientific and clinical advisor.

Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for GLM101, a mannose-1-phosphate replacement therapy in development for the treatment of patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).

Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, announced the presentation of summary findings from its ongoing Phase 2 clinical study (GLM101-002) in adult patients with PMM2-CDG at the Rare Disease Day 2024 Symposium, CDG Scientific and Family Conference, that took place March 1-3 in San Diego.

Glycomine, Inc., a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, announced the first pediatric patient has been dosed in a multi-center open-label Phase 2 study of GLM101, a mannose-1-phosphate replacement therapy, in patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).

Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, announced the presentation of summary findings from its ongoing natural history study at the American Society for Human Genetics (ASHG) 2023 Annual Meeting taking place in Washington, DC. The findings included genotype characterization, combined with the progression of key biomarkers, over two to four years.

Glycomine, Inc., a clinical-stage biotechnology company focused on developing new therapies for serious orphan diseases, announced the appointment of Rose Marino, M.D. as Chief Medical Officer. Dr. Marino has more than 20 years of collective experience in academic medicine and clinical development in the pharmaceutical industry, with specialized training in pediatrics and pediatric endocrinology.

Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, announced the presentation of summary findings from its ongoing natural history study at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium taking place August 29 to September 1 in Jerusalem. The findings included baseline values of key coagulation, liver, metabolic, and endocrine measurements and their evolution over two to four years.

Glycomine, Inc., a clinical-stage biotechnology company focused on developing new therapies for orphan diseases, today announced that the first patient has been dosed in a multi-center open-label Phase 2 study (ClinicalTrials.gov Identifier: NCT05549219) of GLM101, a targeted mannose-1-phosphate replacement therapy, in adult patients with phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG).

Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, today announced that the company has received U.S. Food and Drug Administration (FDA) clearance of an Investigational New Drug (IND) application for GLM101 for the treatment of PMM2-CDG and has initiated dosing healthy volunteers in a Phase 1 clinical study. GLM101 is a mannose-1-phosphate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG Type Ia. PMM2-CDG is the most prevalent congenital disease of glycosylation but has no FDA-approved treatments.

Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, today announced the publication in Molecular Genetics and Metabolism of a key finding from a natural history study of phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), a rare pediatric orphan disease. In this study, the levels of morning cortisol and adrenocorticotropic hormone (ACTH) were measured in a cohort of patients and found to be significantly below normal, indicating PMM2-CDG patients are at risk for secondary adrenal insufficiency. These data provide key insights to improve standard of care, as early recognition of adrenal insufficiency and initiation of glucocorticoid replacement therapy and stress dosing could be lifesaving. The authors conclude that morning cortisol and ACTH levels should be evaluated at least annually for all patients with PMM2-CDG. If abnormal, a low dose ACTH stimulation test should follow to evaluate the hypothalamus, pituitary, adrenal (HPA) axis.

Glycomine, Inc., a biotechnology company focused on developing new therapies for orphan diseases, today announced that it has closed a $68 million Series B financing. The proceeds of the financing will be used to advance Glycomine’s lead drug candidate, GLM101, through initial clinical trials in patients. GLM101 is a novel substrate replacement therapy in development to treat phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), a rare disease representing a critical unmet medical need.