Glycomine Raises $115M to Advance First Disease-Modifying Therapy for PMM2-CDG
San Carlos, CA | April 2025 — Glycomine, Inc., a biotechnology company focused on rare and underserved genetic conditions, has announced a $115 million Series C financing round. This funding will accelerate the clinical development of GLM101, the company’s lead candidate and the first-ever disease-modifying therapy in development for PMM2-CDG, the most common congenital disorder of glycosylation.
What is PMM2-CDG?
Phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG) is a rare, life-threatening genetic condition caused by mutations in the PMM2 gene. This gene encodes an enzyme necessary to produce mannose-1-phosphate, a building block essential for glycosylation— the process by which sugar chains are added to proteins. When this process breaks down, as it does in PMM2-CDG, patients experience a wide range of symptoms including developmental delays, ataxia (a lack of coordination), neurological issues, and potentially fatal organ complications. There are currently no approved treatments.
A Novel Approach: Mannose-1-Phosphate Replacement
Glycomine’s GLM101 is a first-in-class mannose-1-phosphate replacement therapy. It aims to bypass the defective enzyme by directly delivering mannose-1-phosphate to patient cells, restoring the glycosylation pathway and correcting underlying dysfunctions. This approach holds the potential to address the root cause of the disease, rather than simply managing symptoms.
Clinical Progress and Proof of Concept
In a Phase 2 open-label study, GLM101 showed encouraging early clinical results, particularly in addressing ataxia—a key challenge for PMM2-CDG patients. Among nine adolescent and adult participants, the therapy led to an average 11.9-point improvement on the International Cooperative Ataxia Rating Scale (ICARS) over 24 weeks. These improvements mark a significant milestone in establishing clinical proof of concept.
The study is now expanding to include pediatric participants, and the new funding will support a randomized, placebo-controlled Phase 2b trial, set to begin later this year.
Backed by Rare Disease Expertise
The round was led by CTI Life Sciences Fund, abrdn Inc., and Advent Life Sciences, with continued support from top-tier investors including Novo Holdings, Sanofi Ventures, and Abingworth, among others. Dr. Youssef Bennani (CTI) and Dr. Dominic Schmidt (Advent) have joined Glycomine’s Board of Directors.
“This financing will enable us to advance GLM101 into a randomized, placebo-controlled trial later this year—an important step toward bringing the first disease-modifying therapeutic to patients with PMM2-CDG,” said Steve Axon, CEO of Glycomine.
Cosmael ThinkLab Commentary
This financing round and clinical progress reflect an important trend in precision rare disease therapeutics: replacing missing or defective biochemical building blocks rather than relying solely on gene or protein-level repair. If successful, GLM101 could become a paradigm-setting treatment in metabolic and glycosylation disorders, and pave the way for similar approaches in other rare inherited conditions.
Source: Glycomine, Inc. Press Release
For more information: www.glycomine.com