Northern Therapeutics Inc. is a private Canadian biopharmaceutical company committed to the discovery and development of novel cell and gene therapies to extend and enhance the quality of the lives of people suffering from chronic life-threatening pulmonary disorders, in the absence of toxicity and major side effects.

Northern Therapeutics has developed a novel method for selective gene transfer and regenerative cell therapy targeting the pulmonary vasculature with the hope of repairing damaged lung tissues in a number of respiratory and cardiopulmonary conditions, including PAH, Acute Respiratory Distress Syndrome (ARDS), Cystic Fibrosis and Emphysema. This technology involves the isolation of patient’s own cells and further growth in cell culture followed by the introduction of a therapeutic transgene into the cells. These genetically engineered cells are then injected back into the pulmonary circulation, and carried to the lung, where they are efficiently filtered and engrafted at the level of the smallest arteries. In this location, they are able to release the therapeutic gene products, which then can act on the pulmonary microcirculation, as well as participate directly in the regeneration of the damaged lung vessels. The feasibility and efficacy of this method of “cell-based gene therapy” is supported by a number of publications from our Research Team.*

The ‘first’ clinical trial in humans utilizing this novel approach was initiated in Q3 of 2005 utilizing ‘endothelial progenitor cells (EPC)’ as the cell type of choice, which pre-clinical data has shown will optimize efficacy. This Phase IIa clinical trial seeks to establish the safety of cell-based gene transfer of human eNOS in patients with refractory PAH that have failed all conventional treatments.

* Zhao YD, Courtman DW, Deng Y, Kugathasan L, Zhang Q, Stewart DJ. Rescue of Monocrotaline-Induced Pulmonary Arterial Hypertension Using Bone Marrow-Derived Endothelial-Like Progenitor Cells. Efficacy of Combined Cell and eNOS Gene Therapy in Established Disease. Circ Res. 2005;96:442-450.