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Biotech, GSK to Partner on Rare Inherited Cellular Diseases

Illustration of brain (NIDA)

(National Institute of Drug Abuse)

Angiochem, a biotechnology company in Montreal, Canada says it will collaborate with the global pharmaceutical company GlaxoSmithKline on treatments for lysosomal storage diseases, a type of rare, inherited conditions causing cellular dysfunctions. Under the agreement, Angiochem will be eligible for at least $300 million in payments.

Lysosomal storage diseases are a class of disorders caused by an enzyme deficiency from inherited gene mutations that occur in about one in every seven thousand births. The enzymes are needed for the metabolism of lipids or glycoproteins in cells, and the accumulation of undigested or partially digested substances leads to cellular dysfunctions. Lysosomal storage diseases affect the central nervous system, eyesight, and connective tissue and include Tay Sachs Disease, Fabry Disease, Gaucher’s Disease, Pompe Disease, Hunter syndrome, and similar disorders.

The agreement calls for Angiochem to adapt its engineered peptide compound technology, called EPiC, to develop an enzyme replacement therapy that addresses lysosomal storage diseases affecting the brain and central nervous system. Angiochem says the EPic technology can cross the blood-brain barrier using the body’s own receptors and treat these disorders.

GSK will gain the rights to further develop and commercialize drug candidates coming out of Angiochem’s research. The agreement allows for expanding the collaboration to cover other lysosomal storage disease targets.

Angiochem will be eligible for at least $300 million from GSK, starting with up to $31.5 million in initial cash, as well as research funding and other fees if GSK accesses the limited number of disease targets early in the collaboration. Angiochem is can also receive royalties on future sales of EPiC-enzymes that arise from the partnership.

Read more: GSK to Fund Canadian Life Sciences Start-Ups, Institutes

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