Quark has a broad pipeline of siRNA drug candidates based on Quark’s proprietary RNAi technology that includes novel siRNA structures and chemistry which the Company believes provide it with freedom to operate in the siRNA intellectual property arena. Quark also has IP covering non-invasive delivery of siRNA to target tissues including the eye, inner ear, kidney, lung, spinal cord and brain.

Among the pipeline programs:

Hearing Loss

We have developed synthetic siRNAs that protect auditory sensory cells from acoustic trauma and from chemotherapy-induced apoptosis. Quark is developing non-invasive strategies to enable delivery of synthetic siRNAs to the inner ear.Hearing loss is a major problem in the US and across the globe. It is an under-diagnosed and under-treated health issue. An estimated 28 million Americans have some degree of hearing loss.

Lung diseases

Lung transplantation
Acute lung injury (ALI)

Quark has demonstrated effective delivery of our synthetic siRNAs to the lung parenchyma and resident blood-born cells by inhalation. Promising results demonstrating amelioration of primary graft dysfunction (PGD) have been generated in animal models of lung transplantation, resulting in a prominent reduction in edema and bronchoalvelolar lavage cellularity and preservation of lung function evident from normalization of blood oxygenation.

Acute lung injury (ALI) and acute respiratory distress syndrome (ARDS) are 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 US each year with a 40-60% fatality rate. The current standard of care for ARDS includes placing patients on mechanical ventilators. In lung transplant patients, graft failure accounts for 31% of the early (30-day) mortality. Primary graft failure is ascribed to poor function of the allograft caused by ischemia-reperfusion injury.

CNS

Acute and Chronic Neurodegenerative Diseases

Preclinical programs are on-going for application of the Company’s synthetic siRNAs for the treatment of spinal cord injury and chronic neurodegeneration disorders like, e.g., amyotrophic lateral sclerosis (ALS) and Alzheimer’s disease.