Glycomine is a clinical-stage biotech based in San Carlos, California, focused on developing transformative therapies for rare orphan diseases. Its lead candidate, GLM101, is a first-in-class mannose-1-phosphate replacement therapy designed to deliver mannose-1-phosphate into cells and bypass disease-causing PMM2 mutations to restore N-glycosylation. GLM101 has received Orphan Drug Designation in the U.S. and E.U., and Rare Pediatric Disease and Fast Track designations in the U.S. The company has enrolled more than 20 patients across Europe and the U.S. in an ongoing Phase 2 open-label study and has initiated dosing in pediatric patients. Data from the Phase 2 open-label study showed an average 11.9-point improvement on the ICARS among nine adult and adolescent patients over 24 weeks, providing clinical proof of concept for improvement in ataxia. Glycomine recently announced a $115 million Series C financing to support advancing GLM101 into a randomized, placebo-controlled Phase 2b safety and efficacy study later this year. Glycomine is a biotechnology company based in San Carlos, California, developing therapies for orphan diseases. Its lead candidate, GLM101, is a mannose-1-phosphate substrate replacement therapy designed to deliver mannose-1-phosphate intracellularly to bypass PMM2 enzyme deficiency and restore N-glycosylation. Preclinical studies have shown GLM101 can restore disrupted glycosylation pathways in PMM2-CDG. GLM101 has received Orphan Drug Designation in the U.S. and Europe and Rare Pediatric Disease Designation in the U.S. Glycomine plans to use the Series B proceeds to advance GLM101 into initial clinical trials and confirm clinical potential across PMM2-CDG genotypes. The company focuses on replacement therapies targeted to clinically relevant cellular compartments for rare metabolic and protein-misfolding disorders. Glycomine focuses on developing orphan drugs for serious rare monogenic disorders of metabolism and protein misfolding. The company’s approach combines replacement therapies—substrates, enzymes, or proteins—with delivery vehicles made of bio-nanomaterials or ligands to target molecules to the cell interior of relevant organs. Glycomine intends to use the new capital to advance its substrate replacement therapy for PMM2‑CDG (CDG‑1a) into early-stage clinical studies. The company raised $33m in a Series B financing led by Novo Holdings A/S. In connection with the financing, Peter McWilliams, Ph.D., moved from acting CEO to full-time CEO. Kenneth Harrison, Ph.D., of Novo Ventures and Chris Starr, Ph.D., joined the company’s board. Glycomine is a San Francisco, CA–based biotechnology company developing a new generation of replacement therapies for rare monogenic disorders of metabolism and protein misfolding. Its approach combines replacement therapies—substrates, enzymes, or proteins—with intracellular delivery vehicles consisting of bio‑nanomaterials or targeting ligands to deliver molecules into clinically relevant organs. The company is led by CEO Agnes Rafalko, PhD, with Christopher Starr, PhD serving as Executive Chairman. Glycomine raised $12m in a Series A financing to advance its pipeline. The company intends to use the funds to complete IND‑enabling preclinical studies and initiate clinical studies of a substrate replacement therapy in patients with Congenital Disorder of Glycosylation Type Ia (CDG‑Ia). Proceeds will also be used to accelerate discovery efforts toward an enzyme replacement therapy for N‑glycanase deficiency (Ngly1).

Total raised

$228M

Funding rounds

4

Latest round

Series C

Latest activity

Apr 2025