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Tuesday, August 25 • 3:40pm - 4:00pm
[0123] Small Molecules for Treatment of Retinal Degenerative Diseases

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Limited Capacity seats available

Retinitis Pigmentosa (RP) is a family of progressive retinal degenerative diseases that effects small populations. The diseases are associated with many different genes hindering drug development – there are currently no treatments. We have hypothesized that metabolic stress is downstream to many of the gene mutations. Recently, a high throughput screen (HTS) was developed under conditions that mimic RP.[i]   Hits from this primary screen were then subjected to a second assay that measures mitochondrial flux capacity, addressing the oxidative stress component affiliated with this neurodegenerative process. Two of these hits, CB11 and CB12, come together to form a pharmacophore from which novel chemical entities were synthesized.  From these efforts, a small panel of analogs were developed and tested as a means to optimize protection of mitochondria from metabolic stress.  Achieving cellular protection via the cell’s “power house” offers a novel approach towards treating this disease and the potential for addressing other pathologies where mitochondria are part of the degenerative process. 

[i]           Beeson, Craig Cano; Rohrer, Baerbel; Perron, Nathan R. Compositions and methods for the treatment of degenerative diseases  PCT Int. Appl. (2011), WO 2011119869 A1 20110929

 


ST Lecturers
avatar for Christopher Lindsey

Christopher Lindsey

MitoChem Therapeutics Inc.
Director MitoChem Therapeutics Inc.,  Medical University of South Carolina  



Tuesday August 25, 2015 3:40pm - 4:00pm
MCC Theater