The assay technology developed by Zacharon has potential beyond the company’s own internal therapeutic development programs. This technology has the potential to enhance drug development and guide clinical decision-making for diseases in which glycans are implicated in disease pathogenesis.
Sensi-Pro™ Substrate Assay
Lysosomal storage diseases, a family of approximately forty inherited diseases, result from enzyme deficiencies which cause substrates such as lipids or glycoproteins to accumulate in the lysosome, leading to serious and often fatal systemic disease . The family of lysosomal storage diseases includes Gaucher Disease, Fabry Disease, the Mucopolysaccharidoses (MPS), and other disorders.
The development, commercialization, and clinical optimization of therapies for MPS and other lysosomal storage diseases has been challenging due to the limitations of the available clinical and diagnostic measures. Clinical endpoints can have limited utility due to the characteristics of the disease. Several biomarker methods have been developed, however success has been constrained by limitations in sensitivity, dynamic range, and other factors [2-4].
To address this unmet need, Zacharon Pharmaceuticals has developed the Sensi-Pro™ Substrate Assay, a method capable of measuring substrate accumulation with outstanding selectivity and broad dynamic range.
Unique capabilities of the Sensi-Pro™ Substrate Assay
- Highly selective measurement of only the substrate material accumulating due to the deficient enzyme
- Very wide dynamic range, allowing the sensitive comparison of alternative doses
- Universal capability to measure substrate accumulation in a variety of sample types (blood, cerebrospinal fluid, synovial fluid, tissue, and urine)
- Small sample size requirements (less than 100 microliters)
- Characterization of the substrates, allowing for differential diagnosis and thorough analysis of dose response
- Readily adaptable to high-throughput panel-based methods (ex: drug discovery)
- Could be used to simultaneously screen for many lysosomal storage disorders (i.e. newborn screening)
- Applicable to MPS and other lysosomal storage diseases
Potential applications for the Sensi-Pro™ Substrate Assay
- Enhanced clinical development of novel therapies (improved dose / response analysis, patient selection, early indicator of product efficacy, and increased potential to achieve target product profiles)
- Improved evaluation of novel dosing strategies (dose optimization, intrathecal administration, etc.)
- Accelerated pre-clinical development of novel therapies (animal tissue studies, in vitro analysis)
- Identification and classification of undiagnosed patients via panel-based newborn screening methods
Glycans have been strongly implicated in other diseases such as cancer. Thus, Zacharon's diagnostic methods have potential as markers for disease severity, progression, and therapeutic response beyond lysosomal storage disease.
Zacharon Pharmaceuticals is engaging development and commercialization partners for these and other applications. Business development contact information is below.
Director, Business Development
5626 Oberlin Drive, Suite 100
San Diego, CA 92121
(858) 768-7815 office
(858) 459-9756 fax
- Scriver, ed. The Metabolic & Molecular Bases of Inherited Disease. 8th ed. 2001.
- Wraith, E., Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur J Pediatr, 2008(167): p. 267-277.
- Randall, D.R., et al., Heparin cofactor II-thrombin complex: a biomarker of MPS disease. Mol Genet Metab, 2008. 94(4): p. 456-61.
- Fuller, M., et al., Disease-specific markers for the mucopolysaccharidoses. Pediatr Res, 2004. 56(5): p. 733-8.