| Name | Title | Contact Details |
|---|
We are a genome editing company developing genetic medicines for the fight against rare pediatric diseases. GeneRide™ is a promoterless and nuclease-free approach to gene insertion which may improve the safety profile of AAV based gene therapies With GeneRide™, the therapeutic gene is site-specifically integrated into the genome allowing durable gene expression in dividing cells and mature tissues. Furthermore, therapeutic gene expression is regulated by the targeted locus so production can be limited to specific cell types.
Airway Therapeutics has extensive expertise in protein development for applications in the lungs and pediatrics.
Mohawk Shared Services is a Burlington, ON-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Founded in 2004 and located in Marshfield, Wisconsin, PreventionGenetics is a CLIA and ISO 15189:2012 accredited clinical DNA testing laboratory.
Exonics Therapeutics was launched in February 2017 to advance the research of our scientific founder, Dr. Eric Olson and his laboratory at the University of Texas Southwestern Medical Center (UTSW) to develop treatments for patients with neuromuscular diseases. Dr. Olson is one of the world’s leading experts in the study of muscle cells and the application of gene editing to treat these types of diseases. In particular, Dr. Olson’s laboratory has used adeno-associated virus (AAV) to deliver a CRISPR/Cas9 technology that can identify and repair exon mutations to restore the production of dystrophin, a protein that helps stabilize and protect muscle fibers. Dystrophin is the protein missing in boys with Duchenne. The loss of dystrophin causes Duchenne.