Glycomine raises $115M to see if it can treat rare glycosylation disorder

Title

Glycomine Secures $115M to Advance First Disease-Modifying Therapy for Rare PMM2-CDG Disorder

Keywords

Glycomine
GLM101
Congenital Disorders of Glycosylation (CDG)
PMM2-CDG
Rare diseases
Mannose-1-phosphate replacement therapy
Series C financing
Phase 2b clinical trial
Ataxia
Biopharmaceuticals
Orphan drug development

Key Facts

Glycomine, a California-based biotech firm, has raised $115 million in a Series C financing round to advance its lead drug candidate, GLM101, into a Phase 2b clinical trial targeting phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG)1 3 4 7 10.
PMM2-CDG is the most common and severe congenital disorder of glycosylation, a rare, multisystem genetic disease often diagnosed in infancy, causing a wide range of symptoms including severe neurological dysfunction and a high early mortality rate4 7.
There are currently no approved treatments for PMM2-CDG. The disease is caused by deficiency of the enzyme phosphomannomutase 2, which disrupts protein glycosylation pathways essential for normal cellular function3 7.
GLM101 is a first-in-class, lipid nanoparticle–delivered mannose-1-phosphate replacement therapy designed to bypass the faulty PMM2 enzyme and restore normal glycosylation1 4 8.
Initial Phase 2 data has demonstrated promising clinical improvements, particularly in ataxia (loss of muscle coordination), with an average 11.9-point improvement on the International Cooperative Ataxia Rating Scale (ICARS) after 24 weeks in adults and adolescents1 3 4 7.
The Series C funding will enable Glycomine to start a randomized, placebo-controlled Phase 2b trial enrolling 40-50 patients from age two through adulthood, with data readout anticipated in mid-20264 10.
GLM101 has received Orphan Drug, Rare Pediatric Disease, and Fast Track designations in both the U.S. and Europe1 3 8.
Glycomine is also exploring additional approaches, such as mRNA-based therapies, but is currently focused on advancing GLM101 as the first potential disease-modifying treatment for PMM2-CDG4.
The financing round was led by CTI Life Sciences Fund, abrdn Inc., Advent Life Sciences, and included major industry investors like Novo Holdings, Sanofi Ventures, Abingworth, and others3 7.
If successful, GLM101 could represent a breakthrough for patients and families affected by this devastating and currently untreatable rare disease1 3 4 7.

This breakthrough positions Glycomine at the forefront of innovation in rare genetic disorders, with the potential to transform outcomes for PMM2-CDG patients who currently have no approved therapeutic options.