Glycomine is focused on developing innovative replacement therapies for rare genetic diseases, particularly targeting conditions like PMM2-CDG (phosphomannomutase 2-congenital disorder of glycosylation). The company’s lead product, GLM101, is a mannose-1-phosphate replacement therapy designed to address the underlying enzyme deficiency caused by mutations in the PMM2 gene. By delivering mannose-1-phosphate directly into cells, GLM101 aims to restore the disrupted biochemical pathways and improve the quality of life for patients suffering from this debilitating condition. The therapy is currently undergoing clinical trials in the U.S. and Europe, with promising initial results indicating significant clinical benefits, particularly in reducing ataxia, a common symptom of PMM2-CDG.

Glycomine's approach to treatment emphasizes the use of replacement therapies that can restore missing functions in patients with rare genetic disorders. The company is committed to advancing its clinical pipeline, with GLM101 being the first drug developed from its platform. The ongoing Phase 2 clinical study has shown that GLM101 is safe and well-tolerated, with no serious adverse events reported. Glycomine has received Orphan Drug Designation in both the U.S. and Europe, as well as Rare Pediatric Disease Designation in the U.S., underscoring the potential impact of its therapies on patients with limited treatment options.

United States

Glycomine is developing orphan drugs for serious rare disorders of metabolism and protein misfolding for which no other therapeutic options exist. The company's approach is to use replacement therapies – substrates, enzymes, or proteins – and to target those molecules to clinically relevant cellular compartments. Glycomine’s lead drug candidate, 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.

Glycomine

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