Date Posted: 10 September 2009
Quark Pharmaceuticals, based in Freemont California, have announced preliminary results of a study using a chemically modified siRNA to down regulate a gene known as "RTP801", identified through a gene discovery programme at the company's research facility. siRNA approaches to target a range of gene transcripts in human disease are subject to intense research worldwide both in academia and industry. Many of the large global pharmaceutical companies have made significant investments in the field and it is likely that ocular disorders will be among the first beneficiaries to avail of these new technologies.
Quark's results were announced by the company's Chief Scientific Officer, Dr, Elena Feinstein, in a presentation entitled "PF-04523655 (REDD14NP), an siRNA compound targeting RPTP801, penetrates retinal cells producing target gene knockdown and avoiding TLR3 activation". The siRNA compound under evaluation is being co-developed by Quark and Pfizer for patients with diabetic macular edema (DME) and age-related macular degeneration (AMD).
The reported studies were performed in collaboration with Dr. Jayakrishna Ambati at the Department of Ophthalmology and Visual Sciences, University of Kentucky. The objective of the study was to determine if a chemically modified siRNA could enter retinal cells and down regulate a target gene while avoiding immunological response. The studies performed both in vitro and in vivo confirmed both knockdown of the target and avoidance of immune system activation. Quark is currently awaiting Phase II results from a study using an siRNA to target the same gene, RTP801.
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