The structure of human lysosomal acid alpha glucosidase is critical for understanding the disease process of Pompe’s disease. Several studies have revealed the role of Lysosomal acid alpha glucosidase in the degradation of glycogen into glucose. A mutation in this enzyme leads to Pompe’s disease, a rare autosomal-recessive metabolic disorder that destroys lysosomes and muscle cells. The condition can lead to cardiac and hepatomegaly and muscle weakness.The enzyme is synthesized normally but has a mutation in the Asn-233 amino acid. It assembles in the endoplasmic reticulum, but fails to reach the lysosome and fails to enter the Golgi complex. This gene defect prevents it from circulating in the blood stream. It also causes the disease to become life-threatening.
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Growing research &development activities coupled with the number of clinical trials and pipeline products is expected to boost the lysosomal alpha glucosidase market in near future. For instance, in 2017, Valerion Therapeutic, a clinical-stage biotechnology company specialized in the development of therapies for orphan genetic diseases, initiated phase 1/2 clinical trials for its candidate drug, VAL-1221 in patients with late-onset of Pompe disease. VAL-1221 combines company’s proprietary antibody-mediated delivery technology with recombinant human acid alpha-glucosidase, offering improved glycogen clearance and better patient outcomes.
Additionally, JCR Pharmaceuticals Co. Ltd., evaluated its candidate genetically engineered enzyme, JR-162, a therapeutic enzyme product for Pompe disease. As on August 2017, JR-162 was in preclinical stage. JR-162 is developed by applying J-Brain Cargo, an advanced technology by the company that allow the delivery of intravenously administered drug compounds of large molecular weight such as protein to cross blood brain barrier.
Further, in Feb 2018, Amicus Therapeutics, patient-centric biotechnology company focused on discovering, developing and delivering novel high-quality medicines for rare metabolic disease, is investigating ATB200/AT2221 in patients with Pompe disease and is in Phase1/2 clinical study. ATB200 is recombinant human acid alpha-glucosidase enzyme co-administered with AT2221, a pharmacological chaperone. In 2016, Genzyme, a unit of Sanofi, initiated phase III clinical trial for NeoGAA, a second generation alglucosidase alfa enzyme based enzyme replacement therapy for Pompe disease. In Feb 2018, Audentes Therapeutics, Inc., a biotechnology company focused on developing and commercializing gene therapy products, conducts phase 1/2 clinical trial for AT982, to treat Pompe disease.
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Moreover, in 2012, Oxyrane collaborated with VIB researchers from Ghent University and Vrije Universiteit Brussels, have developed a new technology that enables a more efficient approach to ERT production for LSD treatments. Further, Oxyrane, company involved in enzyme replacement therapies (ERTs) for the treatment of lysosomal storage diseases (LSDs), also has a pipeline product named OXY 2810 for Pompe disease.
Regionally, the lysosomal alpha glucosidase market has been segmented into North America, Latin America, Europe, Middle East, Asia Pacific and Africa. Europe is projected to be lead market over the forecast period due to higher rate of incidence in these region. For instance, according to Feb 2018 published article of BMJ Paediatrics Open journal, the incidence of infantile Pompe disease in the Maroon community of France is roughly 50 times higher than elsewhere in the world.
The global market of lysosomal alpha glucosidase market is led by Genzyme
Corp., a unit of Sanofi. The player with pipeline product includes Pharming Group N.V., Valerion Therapeutic, Audentes Therapeutics, Inc. and Oxyrane.
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Table of Content
Chapter 1 Industry Overview
1.3 Research Scope
1.4 Market Analysis by Regions
1.5 Lysosomal Alpha Glucosidase Market Size Analysis from 2022 to 2028
11.6 COVID-19 Outbreak: Lysosomal Alpha Glucosidase Industry Impact
Chapter 2 Global Lysosomal Alpha Glucosidase Competition by Types, Applications, and Top Regions and Countries
2.1 Global Lysosomal Alpha Glucosidase (Volume and Value) by Type
2.3 Global Lysosomal Alpha Glucosidase (Volume and Value) by Regions
Chapter 3 Production Market Analysis
3.1 Global Production Market Analysis
3.2 Regional Production Market Analysis
Chapter 4 Global Lysosomal Alpha Glucosidase Sales, Consumption, Export, Import by Regions
Chapter 5 North America Lysosomal Alpha Glucosidase Market Analysis
Chapter 6 East Asia Lysosomal Alpha Glucosidase Market Analysis
Chapter 7 Europe Lysosomal Alpha Glucosidase Market Analysis
Chapter 8 South Asia Lysosomal Alpha Glucosidase Market Analysis
Chapter 9 Southeast Asia Lysosomal Alpha Glucosidase Market Analysis
Chapter 10 Middle East Lysosomal Alpha Glucosidase Market Analysis
Chapter 11 Africa Lysosomal Alpha Glucosidase Market Analysis
Chapter 12 Oceania Lysosomal Alpha Glucosidase Market Analysis
Chapter 13 South America Lysosomal Alpha Glucosidase Market Analysis
Chapter 14 Company Profiles and Key Figures in Lysosomal Alpha Glucosidase Business
Chapter 15 Global Lysosomal Alpha Glucosidase Market Forecast (2022-2028)
Chapter 16 Conclusions
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