Sirnaomics, Inc. is becoming a leading company in RNAi therapeutics through use of three key technologies:

(A) In Silico design followed by high throughput (HTP) selection of siRNA inhibitors;
(B) Enhanced API efficacy through the use of multi-targeted siRNA cocktails;
(C) Clinically viable siRNA delivery in vivo using nanoparticle carriers.

These technologies form the basis of Sirnaomics ‘R4D’ therapeutic platform: Rapid Design, Discovery, Development and Delivery (of siRNAs for therapeutic development).

1. To identify the most potent siRNA inhibitors, we have developed a proprietary algorithm for siRNA sequence prediction against known gene targets. These siRNAs have optimal thermodynamics, enhanced RISC binding, lack immune stimulating motifs, have minimized “Off-Target” potential, and are homologous between human and mouse (to facilitate ease of development). The individual siRNA sequences and their combinations allows unique composition of matter patent protection. A robotic screening platform allows rapid in vitro validation of the individual siRNAs as well as the optimal combination of these for maximal potency and efficacy.

2. Human diseases are often complex syndromes involving contributions from multiple genes. To address this aspect of human disease etiology and to take advantage of chemical simplicity (uniformity) of siRNA design, a proprietary algorithm allows development of a “Tri-Blocker” design of a cocktail of siRNA duplexes targeting up to three disease-causing genes in one drug API. The siRNA API validated in cell culture is further tested and validated in a mouse model for its therapeutic property using efficient in vivo siRNA delivery systems.

3. We have developed and licensed several clinically viable and clinically approved nanoparticle technologies for use as delivery vehicles for siRNAs to appropriate targets in vivo. These include polymer and liposome-based siRNA carriers for therapeutic applications.

• SNANO-1 is a branched cationic polymer for enhanced siRNA delivery in various animal tissues.

• SNANO-2 is a clinically validated polymer for delivery of therapeutic compounds including siRNA.

• SNANO-3 is a clinically approved polymer for delivery of therapeutic agents including siRNA.

• SNANO-4 is a Pegylated cationic polymer for local delivery of siRNAs in vivo.

• SNANO-5 is a liposomal carrier for nucleic acid and siRNA delivery in vivo.

• SNANO-6 is a bio-degradable polymer for siRNA therapeutic application.

The combinations of design, discovery, development and delivery technologies for multi-targeted siRNAs differentiates Sirnaomics from its competitors in the RNAi therapeutic space and provides the company with a significant competitive advantage in rapid migration of our products from bench to bedside.

Therapeutic Programs

Sirnaomics, Inc. has worked with its scientific founders and collaborators in developing siRNA therapeutic protocols using the multi-targeted siRNA cocktail approach. Several such protocols have been tested in animal disease models with very promising results for therapeutic applications. Strong ties with the Chinese biomedical community allows the progression of these products through high quality preclinical and clinical studies at low cost.

The company currently has 3 key therapeutic programs in development:

1. The company’s leading therapeutic product, STP601, is aimed at Age-related Macular Degeneration (AMD), Proliferate Diabetic Retinopathy (PDR) and Herpetic Stromal Keratitis (HSK). This product has just completed a pre-IND meeting (May 14, 2008) with the USFDA and received a green light for IND enabling studies. The product will be delivered through intravitreous administration. Currently, the entire market is about US$4 billions and will reach to US$13 billions in 10 years, and at the time RNAi therapeutics itself will reach to US$700 millions.
2. The second therapeutic product, STP702, for seasonal and pandemic influenza infection, has demonstrated potent anti-H5N1 (avian influenza) activity in a mouse model. This product has shown to have both prophylactic and therapeutic benefit when administered through intranasal inhalation. Data from these studies has been used to submit a proposal in response to an NIAID Broad Agency Announcement (BAA) seeking a therapeutic platform for biodefense applications. Our submission sought ~$11 million for development of this product through preclinical studies. A subsequent grant (UO1; ~$7M) has also been submitted for R&D of siRNA therapeutics for influenza and other respiratory viral infections. This consortium grant leverages the strengths of Sirnaomics scientific founders and management team in the use of RNAi to treat respiratory viral infections. These therapies will position the company to be able to deliver valuable therapies when the next pandemic hits. The market for seasonal flu is about US$300 million a year while the development of STP702 is to address the biodefense application and potential threat of pandemic flu.
3. The third product, STP705, is an siRNA therapeutic cocktail for facilitating scarless wound healing. Using topical administration in a mouse model this cocktail has been shown to significantly accelerate wound healing while also demonstrating a marked decrease in scar formation. We are currently pursuing this product for use in battlefield medicine and are seeking collaborations with the armed forces. The wound healing market is currently estimated at $6billion worldwide and is expected to increase to $11b by 2012.

We are continuing to evaluate opportunities where siRNA therapeutics combined with our delivery agents can be used to penetrate new markets. Therapeutic programs in collaboration with several other institutions include validation and development of siRNA cocktails in treatment of brain cancer (glioblastoma Multiforme), prostate cancer, Melanoma, non-small cell lung cancer and breast cancer, treatments to assist in solid organ transplantation, treatments for other respiratory infectious diseases, etc.

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