Glycomine, a biotechnology startup working on a treatment for a rare genetic disorder, has raised a $115 million Series C round to push its lead program deeper into clinical testing.
The funding announced Wednesday for the San Carlos, California-based company will support development of its therapy GLM101, which is designed to treat phosphomannomutase 2-congenital disorder of glycosylation, or PMM2-CDG.
PMM2-CDG is the most common among a family of inherited disorders that cause errors in glycosylation, the biological process by which sugar chains called glycans are added to proteins. This type of malfunction can affect the structure and function of cellular proteins throughout the body, leading to an array of symptoms, developmental delays and impaired motor function. There are no approved treatments for the roughly 15,000 people who are estimated to have the condition in the U.S. and Europe, though a few programs are in clinical testing.
“That need is extremely high,” said Steven Axon, Glycomine’s CEO. “There's no other therapies for these patients. There's only supportive care.”
Glycomine aims to change that by delivering into the body a component used in glycosylation — mannose-1-phosphate — that’s deficient in people with PMM2-CDG.
The company’s approach is somewhat akin to the enzyme replacement therapies used to treat lysosomal storage disorders like Fabry or Pompe disease. Glycomine’s therapy is replacing a missing sugar molecule rather than an enzyme, however, and corrects a defective chemical process instead of clearing an accumulating toxin.
Last year, Glycomine released Phase 2 data showing the drug appeared to impact ataxia, a common symptom of PMM2-CDG, in a small group of patients. The Series C round will carry the company through a more rigorous, randomized and placebo-controlled Phase 2b study that should start in the middle of this year and enroll between 40 to 50 participants, Axon said.
The company hopes to report data in 2026 that “will really tell us whether the effect we were seeing in the earlier clinical program has been confirmed,” he said.
If those results are positive, Axon said the company may explore a partnership with a pharmaceutical company.
“Having this randomized controlled data and really knowing that the drug works and its path to approval would be highly derisking for a pharma partner,” he said.
Though Glycomine has not disclosed specifics about the rest of its pipeline, it’s developing drug candidates for other rare diseases outside of PMM2-CDG, according to Axon.
The company’s Series C funding was led by CTI Life Sciences Fund, funds managed by Abrdn, and Advent Life Sciences. Other backers include Novo Holdings, Sanofi Ventures and Abingworth. Glycomine previously raised $80 million across Series A and B rounds.
