March 2, 2016 9:35am

From its In Vivo protein replacement platform programs for MPS I and MPS II; resulted in statistically significant improvements in cognitive function in treated animals 


 

SGMO presentation of new preclinical data in disease models from its In Vivo Protein Replacement Platform programs for MPS I and MPS II.

 

The Bottom Line: The data demonstrate that the IVPRP approach enabled stable production of therapeutic levels of replacement enzyme from the liver into the circulation and secondary tissues, including the brain, resulting in significant reduction in biomarkers of the disease and, importantly, statistically significant improvements in cognitive function in treated animals.

Behavioral data from animals was tested in the Barnes Maze, a tool used to measure spatial learning and memory, demonstrated statistically significant preservation of cognitive learning in MPS I and MPS II mice treated with the ZFP Therapeutic, compared to untreated mice.

Supporting these data, increased levels of the hIDUA and hIDS (statistically significant at high dose) enzymes were detected in the brain tissue of treated animals.

The data further demonstrated that therapeutic hIDUA and hIDS produced in the liver could be transported across the blood brain barrier at levels that were able to effect meaningful change in cognitive function.

 

SGMO closed at $5.48 and is DOWN -$0.07 at the open… early dta but with a positive indication which needs more development.