SAN FRANCISCO, Nov. 16, 2016 -- Glycomine, Inc. a biotechnology company developing a new generation of replacement therapies for rare diseases, today announced that it has raised $12 million in a Series A financing led by Sanderling Ventures and supported by Chiesi Ventures as well as existing high net worth individuals and patients. Glycomine will use the proceeds from this financing to complete IND enabling pre-clinical studies and initiate clinical studies of its substrate replacement therapy in patients with Congenital Disorder of Glycosylation Type Ia (CDG-Ia), and to accelerate discovery efforts towards an enzyme replacement therapy for N-glycanase deficiency (Ngly1).

"Glycomine's approach to a new generation of replacement therapies focuses on intracellular delivery and targeting of treatments to clinically relevant organs," said Agnes Rafalko, PhD, Glycomine's Chief Executive Officer. "We are very grateful for the funding and support we have received from our investors, patients' families, and doctors as we embark on the development of the first-ever replacement therapy for CDG-Ia." The therapy has the potential to significantly improve both patients' quality of life and lifespan. "CDG-Ia is a debilitating disorder with no treatment and Glycomine's approach to therapy is our only hope for these patients," said Prof. Jaak Jaeken of University of Leuven, Belgium, the mastermind behind the discovery of Congenital Disorders of Glycosylation.

Replacement therapies continue to play an important role in orphan diseases. "One of the advantages of substrate or enzyme replacement therapies is having the ability to adjust the therapeutic dose according to patients needs and disease severity," said Gregory Enns, MD, of Stanford Medicine who is also a member of the company's Clinical Advisory Board.

"Glycomine's approach reminds me of the emergence of therapies for lysosomal storage disorders," said Christopher Starr, PhD, Glycomine's Executive Chairman and previously co-founder and C-level executive of BioMarin and Raptor. "Mucopolysaccharidosis I (MPS I) was the first of its class to be successfully treated with enzyme replacement therapy and led the way to BioMarin's pipeline growth in other MPS and lysosomal storage disorders. CDG-Ia is one of the first out of more than 100 unaddressed disorders of glycosylation to be potentially treated with a substrate replacement therapy."

In conjunction with Glycomine's Series A financing, the company announced that Peter McWilliams, PhD, MBA, Managing Director at Sanderling Ventures and Giacomo Chiesi, MBA, Managing Partner at Chiesi Ventures joined the company's Board of Directors. "Sanderling is delighted to invest is such a deeply passionate, experienced, and highly talented management team," said Peter McWilliams. "Together with the expertise of Chiesi Ventures we aim at accelerating the development of long-awaited and truly important therapies for patients in immense need."

About Congenital Disorder of Glycosylation Type Ia

Congenital Disorder of Glycosylation Type Ia (CDG-Ia) is an inherited metabolic disorder caused by a deficiency of an enzyme called phosphomannomutase 2 (encoded by the gene PMM2). As a result, the body fails to produce glycoproteins, which are proteins decorated with carbohydrates often referred to as sugar trees. CDG-Ia affects many systems of the body including the nervous system. Approximately 20% of children with the disease die within the first few years of life, often due to infection, liver problems, or heart disease. Others with CDG-Ia may live into adulthood and most are wheelchair bound throughout their life, unable to speak and live independently. There is no cure or therapeutic option for >1000 children diagnosed with the disorder. For more information about the disease and patient network, please visit CDG Community Alliance and Resource Exchange at

About Sanderling Ventures

Founded in 1979, Sanderling is among the oldest investment firms dedicated to building new life science and biomedical companies. Sanderling's unique approach combines a specialized investment focus with active management and long-term commitment to building sustainable important companies. The firm seeks to ensure the highest rates of return for its investors and the creation of sustainable new companies for the communities in which it invests. Since its inception, Sanderling has supported over 90 biomedical companies from very early stage start-ups, through commercial development. For further information please visit the firm's website at

About Chiesi Ventures

Chiesi Ventures is a venture capital firm focused on the area of rare and orphan disorders. Its goal is to complement the strategic interest of the Chiesi Group by investing in early stage development opportunities. Chiesi Ventures also aims to accelerate the expansion of the Chiesi network in the US and EU among universities, venture capital investors, rare disease patient organizations and entrepreneurial companies developing treatments for rare diseases. To learn more, please visit

About Glycomine, Inc.

Glycomine is developing orphan drugs for serious rare monogenic disorders of metabolism and protein misfolding for which no other therapeutic options exist. The company's approach is to combine replacement therapies – substrates, enzymes, or proteins – with intracellular delivery vehicles consisting of bio-nanomaterials or ligands that target the molecules to the cell interior of clinically relevant organs. The company is based in San Francisco, California.