Glycomine, Inc., a biotech company developing therapies for orphan diseases, raised $115 million in a Series C round to progress its lead candidate, GLM101, into a Phase 2b clinical trial. The round was led by CTI Life Sciences Fund, abrdn Inc.-managed funds, and Advent Life Sciences, with ongoing support from existing investors including Novo Holdings, Sanofi Ventures, Abingworth, RiverVest, Sanderling Ventures, Chiesi Ventures, Remiges Ventures, and Asahi Kasei Ventures.

Glycomine will use the funding to advance GLM101 into a randomized, placebo-controlled Phase 2b safety and efficacy study.

GLM101, a pioneering mannose-1-phosphate replacement therapy, targets phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG), a rare, life-threatening genetic disorder with no approved treatments. The funding will support a randomized, placebo-controlled trial starting later this year. Glycomine’s ongoing Phase 2 study, involving over 20 patients in the U.S. and Europe, has shown promising results, including an average 11.9-point improvement in ataxia (measured by ICARS) among nine adult and adolescent patients over 24 weeks.

“We’re thrilled to partner with leading rare disease investors and appreciate our existing investors’ continued support,” said Steve Axon, CEO of Glycomine. “This funding advances our mission to deliver the first disease-modifying therapy for PMM2-CDG patients.”

Youssef Bennani, Ph.D., of CTI Ventures, praised Glycomine’s progress, while Dominic Schmidt, Ph.D., of Advent Life Sciences, highlighted the therapy’s potential to significantly improve ataxia, a major challenge for PMM2-CDG patients.