Projects

Alpha-Mannosidosis

The Zymenex pipeline includes Lamazym, for the treatment of the lysosomal disease Alpha-Mannosidosis. This disease is due to a deficiency of the Laman enzyme, affects approximately 500 patients world wide and the project is in late pre-clinical development.

Alpha-Mannosidosis is caused by an inability to break down certain carbohydrates, leading to the accumulation of oligosaccharides (carbohydrate molecules) in the lysosomes. It is believed that reducing the level of accumulated mannose-containing compounds would alleviate the symptoms associated with the condition.

The most severe form of the disease, the infantile phenotype (type 1) leads to rapid progressive mental retardation and typically death between 3 and 12 years of age. The juvenile-adult phenotype (type 2) is characterized by a milder and more slowly progressive course with survival into adulthood.

Zymenex collaborated with a number of European scientific groups under EU grants "Euraman" from 2003 to 2006 and "HUE-MAN from 2007 to 2009 to develop this enzyme and has, together with the scientific group, published proof-of-principle data (Hum. Mol. Genetics, 2008, vol 17, no 22: 3437-3445). Zymenex and its clinical and scientific collaborators received a new EU grant "ALPHAMAN" of € 5.9 million, under the 7th Framework 2010 www.alpha-man.eu to continue developing Lamazym and to perform clinical trials Phases 1 through 3 in alpha-Mannosidosis patients. Lamazym has received Orphan Drug Designation in both Europe and the US.

Globoid Cell Leukodystrophy (Krabbé)

Krabbé disease is a rare, inherited degenerative disorder of the central and peripheral nervous systems. It is characterized by the presence of globoid cells (cells that have more than one nucleus), the breakdown of the nerve's protective myelin coating, and destruction of brain cells. Krabbé disease is a leukodystrophy with an impairment of the growth or development of the myelin sheath, the fatty covering that acts as an insulator around nerve fibers, and cause severe degeneration of mental and motor skills. Myelin, which lends its color to the "white matter" of the brain, is a complex substance made up of at least 10 different enzymes. Krabbé disease is caused by a deficiency of the enzyme galactocerebrosidase, an essential enzyme for myelin metabolism. The disease most often affects infants, with onset before age 6 months, but can occur in adolescence or adulthood. Symptoms include irritability, unexplained fever, limb stiffness, seizures, feeding difficulties, vomiting, and slowing of mental and motor development. Other symptoms include muscle weakness, spasticity, deafness, and blindness. Infantile Krabbé disease is generally fatal before age 2. Bone marrow transplantation has been shown to benefit mild cases early in the course of the disease. Generally, treatment for the disorder is symptomatic and supportive.

Aspartylglucosaminuria

Aspartylglucosaminuria is an inherited disease belonging to the larger family of metabolic disorders called ‘lysosomal storage diseases'. Patients with this condition are unable to produce enough of the enzyme aspartylglucosaminidase, which is involved in the breakdown of molecules called glycoproteins within cells. As a result, glycoproteins accumulate in tissues, causing cell damage, particularly in the nerves and bones.

Patients with the disease have developmental delays (such as in speech and movement) in the early years of life, followed by a gradual decline in mental abilities in later years. Patients may also have problems with their bones, such as abnormalities in the curvature of their spine and in their facial features.

Aspartylglucosaminuria is a debilitating and life-threatening disease because of the decline in mental functions and other problems such as epilepsy and infections.

Technology

The company’s core knowledge is within protein technology, with expertise in molecular cloning, fermentation/purification, analytical development, preclinical and clinical testing and regulatory affairs. Zymenex has proprietary intellectual property in the field of Rare Metabolic Diseases.

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