CDG

Glycomine Closes $68 Million Series B to Advance into Clinical Trials a Novel Treatment for PMM2-CDG, a Rare Glycosylation Disease

Retrieved on: 
Wednesday, June 23, 2021
Professional services, Health, Finance, Clinical trials, Pharmaceutical, Biotechnology, Congenital disorders of glycosylation, PMM2, Genetic genealogy, Medical genetics, Branches of biology, Novo Holdings A/S, CDG, PMM2-CDG, Glycomine, Inc., Abingworth, Sanofi Ventures, RiverVest Venture Partners, Remiges Ventures, PMM2-CDG, GLYCOMINE, INC., ABINGWORTH, SANOFI VENTURES, RIVERVEST VENTURE PARTNERS, REMIGES VENTURES

The proceeds of the financing will be used to advance Glycomines lead drug candidate, GLM101, through initial clinical trials in patients.

Key Points: 
  • The proceeds of the financing will be used to advance Glycomines lead drug candidate, GLM101, through initial clinical trials in patients.
  • 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.
  • The Series B financing includes $35 million of new funds in addition to the $33 million announced in August 2019.
  • In addition, all previous Series B investors Novo Holdings A/S, Asahi Kasei Pharma Ventures, Mission BioCapital, Sanderling Ventures, and Chiesi Ventures participated.

Cerecor to Collaborate with Frontiers CDG Consortium on Pivotal Trial of CERC-801 for the Treatment of PGM1-CDG

Retrieved on: 
Tuesday, November 17, 2020
PGM1, Congenital disorders of glycosylation, Glycoprotein, CDG, Draft:SRD5A3 CDG

Andrea Miller, JD, MHA, President & Founder of CDG CARE, stated, We are truly excited to see the collaboration between FCDGC and Cerecor and this prospective trial in PGM1-CDG.

Key Points: 
  • Andrea Miller, JD, MHA, President & Founder of CDG CARE, stated, We are truly excited to see the collaboration between FCDGC and Cerecor and this prospective trial in PGM1-CDG.
  • CDG is an area of high unmet need where there are no approved therapies today.
  • CDG patients are born with a genetic defect that hinders their ability to utilize certain monosaccharides in the production of glycoproteins.
  • CDG CARE is a nonprofit 501(c)(3) organization founded by parents seeking information and support for a group of disorders known as CDG.

FDA Grants AT-007 Pediatric Rare Disease Designation and Orphan Designation for Treatment of PMM2-CDG

Retrieved on: 
Thursday, September 24, 2020
Congenital disorders of glycosylation, PMM2 deficiency, Health, Articles, PMM2, Branches of biology, Orphan drug, CDG

Food and Drug Administration(FDA) has granted AT-007 both Pediatric Rare Disease Designation and Orphan Drug Designation for treatment of PMM2-CDG.

Key Points: 
  • Food and Drug Administration(FDA) has granted AT-007 both Pediatric Rare Disease Designation and Orphan Drug Designation for treatment of PMM2-CDG.
  • PMM2-CDG is a debilitating rare disease caused by deficiency in the critical enzyme phosphomannomutase-2, required for systemic glycosylation of proteins.
  • PMM2-CDG causes multiple organ failure and severe disability, resulting in approximately 20% mortality in the first four years of life.
  • We are pleased that the FDA recognizes the transformative potential of AT-007 in treating patients with PMM2-CDG.