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核心技术

Our team uses a consistent, validated approach to develop novel siRNA based therapeutics for significant markets with high unmet clinical need. Using a proprietary algorithm for siRNA sequence prediction against known gene targets we can rapidly identify siRNAs for use against traditionally "non-drugable" targets.


Our delivery technologies allow us to select the best approach to ensure efficient and safe delivery of siRNAs to their target tissues. Histidine-Lysine peptide copolymer (HKP, US 7,070,807 B2) is the proprietary delivery system for our leading siRNA therapeutic candidate. It is a biodegradable and can be self-assembled with siRNA into nanoparticles having average size of 150 nM in diameter. Human diseases are often complex and multiple genes can be involved in etiology. To address this aspect of human disease etiology and to take advantage of the chemical simplicity (uniformity) of siRNA design, a proprietary algorithm generates a combination of siRNA targeting disease-causing genes in one drug product. 













STP705, Hypertrophic Scar Prevention and Reduction:  


Our lead product candidate STP705 is an Anti-fibrosis compound and our first indication in humans will be Hypertrophic scar reduction and prevention. Hypertrophic scarring is an abnormal condition that can occur after any injury to the skin and most commonly after surgical procedures. This abnormal scarring results in scars that are large, red, raised and pruritic. If this type of scarring occurs on the face or over joints such as the hips, knees, shoulders, or hands, it can be both disfiguring and debilitating.  There is no FDA-approved therapeutic for hypertrophic scar prevention and reduction and currently used off label treatments typically have been show to have minimal impact in terms of efficacy with very high recurrence rates. Pathophysiology of hypertrophic scars entails a prolonged inflammatory and proliferative phase of wound healing after injury. Among various cytokines promoting hypertrophic scar formation, TGF-β1 is known as a key regulator of the aberrant fibrogenic response while COX-2 acts as potent pro-inflammatory and proliferative mediator. STP705 (Cotsiranib) targets both TGFβ1 and Cox-2 gene expression and has been tested in mouse and swine skin excision wound models, and human hypertrophic scar tissue implant mouse models. STP705 has achieved simultaneous silencing of TGF-Beta1 and COX-2 expression as well as down regulation of fibrogenic markers like alpha SMA, hydroxyproline, Collagen 1 and Collagen 3 and fibroblast apoptosis in multiple animals models as well as a human hypertrophic scar model and STP705 has demonstrated therapeutic benefit of scar prevention and reduction in animal models.


The market opportunity for STP705 is estimated at $4.5-5B. Each year, 42 million surgical skin procedures are performed and 62 million scars are formed in the US. Approximately 93 million people in the US are living with scars and about 169 million scars can be characterized as hypertrophic or keloids. If STP705 is successful at preventing or reducing scars in clinical studies, we anticipate that it could gain share rapidly in a currently underserved market.  Our pipeline compounds are currently focused on systemic anti-fibrosis diseases and oncology.  

3gen