Working on a biotechnology platform intended to identify anti-infectives and immune modulators, the Genma Biosciences platform supported investigation of RNA repeat diseases such as myotonic dystrophy and lysosome based diseases, enabling insights re. drug mechanism of novel chemical molecules. Using the well-established genetic model C. elegans in a high throughput phenotypic screening format Genma Biosciences is tackling discovery of novel therapies and drug targets. By engineering C. elegans to mimic human disease and then screening for compounds or mutations in gene targets that correct the disease in living animals, Genma is able to rapidly identify the best lead compounds for advancement into the clinic. Gemmas technology platform was originally developed to identify anti-infectives and immune modulators/ The effort is now to use a similar approach to investigate RNA repeat diseases such as myotonic dystrophy and lysosome based diseases such as NCL. A working assumption is that therapies discovered through its whole animal approach could potentially have broader application to other diseases such as Alzheimers disease, ALS and inflammatory bowel disease.
