Projects

Velmanase alfa for treatment of Alpha-Mannosidosis

Velmanase alfa (Recombinant Human Alpha Mannosidase) is an enzyme replacement therapy for treatment of the lysosomal disease Alpha-Mannosidosis. The project is currently under regulatory review, as a Market Authorization Application (MAA) has been submitted to EMA.


Alpha-Mannosidosis is due to a deficiency of the Alpha Mannosidase enzyme and affects approximately 500 patients worldwide. It is caused by an inability to break down mannose containing 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.


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 velmanase alfa and to perform clinical trials Phases 1 through 3 in alpha-Mannosidosis patients. Velmanase alfa has received Orphan Drug Designation in both Europe and the US.

Recombinant human aspartylglucosaminidase (rhAGA) for the treatment of Aspartylglucosaminuria

Aspartylglucosaminuria is an inherited disease belonging to the larger family of metabolic disorders called ‘lysosomal storage diseases'. Patients with this condition have a mutation in the gene coding for the enzyme aspartylglucosaminidase, which is involved in the breakdown of molecules called glycoproteins within cells. As a result, the enzyme is inactive and aspartyl containing oligosaccharides 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 motor and mental abilities in later years. Recurrent infections are also common. 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.

 

Recombinant human galactocerebrosidase (rhGAL) for treatment of Krabbe disease

Krabbe disease (Globoid Cell Leukodystrophy) 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. Krabbe 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. Krabbe 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 Krabbe 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.

 

Technology

The company’s core knowledge is within protein technology, with main expertise in molecular cloning, cell propagation, protein purification, analytical development, protein characterization, formulation development, and preclinical testing. Clinical development and regulatory affairs are pursued jointly with Chiesi R&D. Zymenex has proprietary intellectual property in the field of Rare Metabolic Diseases

 

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