Quark has a broad pipeline of siRNA drug candidates based on internally developed novel structures. These programs, in preclinical studies, are based on the Company’s fully integrated drug development platform that spans therapeutic target identification to drug development. Quark’s RNAi technology includes novel siRNA structures and chemistry providing Quark with freedom to operate in the siRNA intellectual property arena, as well as the ability to deliver siRNA non-invasively to target tissues including the eye, ear, kidney, lung, spinal cord and brain.
Among the pipeline programs:
Lung diseases
Acute lung injury (ALI)
Lung transplantation
Our proof of concept studies have shown that Quark’s siRNA can be efficiently delivered by inhalation to rodent and non-human primate lungs. Direct lung delivery of Quark’s siRNA drug candidate lead to a prominent knockdown of its target gene in the LPS-induced lung inflammation model in mice and has shown prominent anti-inflammatory activity, reverting LPS-induced pathological features. Results from this model and pilot studies in ischemia- reperfusion injury in rat suggest that inhibition of this Quark target by siRNA [s1] is a potential preventive or therapeutic method for lung indications including acute lung injury and lung transplantation
Acute lung injury (ALI) and acute respiratory distress syndrome (ARDS) are acute life-threatening forms of hypoxemic respiratory failure and a leading cause of morbidity and mortality in critically ill patients. An estimated 150,000 to 250,000 adults are diagnosed in the U.S.A each year with a 40-60% fatality rate. The current standard of care for ARDS includes placing patients on mechanical ventilators in intensive care units. In lung transplant patients, graft failure accounts for 30.5% of the early (30 days) mortality. Primary graft failure is ascribed to poor function of the allograft, caused by ischaemia-reperfusion injury.
Tumors in the lung
Lung cancer
Metastases in the lung
Inhaled siRNA, unlike siRNA administered intravenously, efficiently enters not only normal lung cells but also tumor cells growing in lungs. The Company’s siRNA to a proprietary target gene sensitizes established tumors to chemo- and radiotherapy. Further preclinical studies are under way.
Lung cancer is the leading cause of cancer-related mortality in both men and women, worldwide. Its prevalence is second only to that of prostate cancer in men and breast cancer in women.
CNS
Acute and Chronic Neurodegenerative diseases
Preclinical programs are on going for non-invasive delivery of the Company’s novel siRNA.:
To the spinal cord in models of spinal cord injury (SCI) and amyotrophic lateral sclerosis (ALS)
To the brain, in models of Alzheimer’s disease.