RNA Interference (RNAi)

RNAi has emerged as a powerful tool for therapeutic gene silencing....Its potential for therapy is indisputable, considering that one can use this mechanism to silence genes implicated in carcinogenesis and metastasis.

​RNA interference (RNAi) is a natural mechanism used by the cell to regulate its molecular fate. RNAi can be compared to a system of switches on a circuit-board, which ultimately direct how information flows inside the cell. Specifically, in RNAi, short double-stranded RNA oligonucleotides inhibit the expression of genes with complementary nucleotide sequences, similar to the way a key and lock interact. RNAi has emerged as a powerful tool for therapeutic gene silencing because of its potency, modularity, broad applicability (virtually any gene can be silenced), and potential for exquisite specificity (single nucleotide). Its potential for therapy is indisputable, considering that one can use this mechanism to silence genes implicated in carcinogenesis and metastasis. 

Transforming the diagnosis and treatment of cancer

TransCode Therapeutics, Inc., an RNAi Company, is focused on the discovery, development and commercialization of a pipeline of innovative diagnostics and therapeutics for the detection and treatment of cancer, with an initial focus on metastasis. ​TransCode’s vision is to be the leader in RNAi in oncology employing data-driven cancer diagnostics as well as optimized targeted treatment solutions for each individual cancer patient that results in a significant improvement in their outcome.

Using RNAi - Individualized Patient Approach

               Strategic Drivers

• Significantly Improve Cancer Outcomes - Initial Focus on Metastasis  
• Data Driven - Translational Bioinformatics
• Early Diagnostic Detection of Disease
• Targeted Delivery of Cancer Therapeutics​

​Conventional therapies aimed at treating primary cancer oftentimes are ineffective at treating metastatic cancer, resulting in poor outcomes in patients diagnosed with metastatic disease. ​​

Improve Cancer Outcomes

• A need for differential treatment is paramount
• Initial goal is to significantly improve treatment outcomes in patients with late stage disease​
• ​Individual patient treatments based on mutation type, stage of disease and biomarker expression
• Optimize dose and treatment duration for each individual patient

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Image-guided Delivery Platform

• Image-guided treatment combines knowledge of drug bioavailability & outcome​
• Delivery-based therapeutic platform - backbone of therapeutic pipeline
• Therapeutics targeting specific biomarkers in multiple cancer types
• ​Combination therapies as a multipronged approach to treatment

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Data Driven 

• Identify key biomarkers associated with primary and metastatic cancers
• Apply highly specific biomarker diagnostic tools for cancer detection and staging
• Assimilate biomarker expression data, stage of disease, tumor type, aggressiveness of disease 
• Translational bioinformatics - data to inform therapeutic decision making and early intervention​

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Early Detection 

​The staple of reducing mortality due to cancer has been early detection. 

• Utilize novel diagnostic biomarkers for the presence of metastasis
• Develop predictive biomarkers for early diagnosis of metastasis and disease progression
• Identify patients at increased risk of progression
• Stratify tumors based on aggressiveness to better inform treatment 

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2. Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature. 2001;411(6836):494-8.
3. McCaffrey AP, Meuse L, Pham TT, Conklin DS, Hannon GJ, Kay MA. RNA interference in adult mice. Nature. 2002;418(6893):38-9.