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Noncoding RNA Therapeutics Development

Noncoding RNA (ncRNA) therapeutics have shown potential application in the treatment of rare diseases. Our company has an interdisciplinary background in biology, immunology, chemistry, and nanotechnology, and has hired the world's top scientists to build an RNA-based therapy development platform. We have the capability to provide you with noncoding RNA therapeutics development services, including one-stop solutions that can meet any of your needs in rare disease gene therapy research.

Overview of Noncoding RNA Therapeutics

Noncoding RNA therapeutics use noncoding RNA molecules, which do not encode proteins but produce non-coding transcripts that regulate gene expression and protein function, to regulate gene expression and treat diseases. The main ncRNAs are the short miRNAs that have been well studied so far and the lncRNAs that have been discovered recently. They are associated with complex biological processes, such as immune cell development and function, neurodevelopment, and neurological diseases. Therefore, targeting these naturally occurring ncRNAs is a very promising therapeutic for various diseases.

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Fig.1 Biogenesis of several noncoding RNAs. (Ding, Lifeng, et al., 2021)

Noncoding RNA Therapeutics for Rare Diseases

Currently, a variety of RNA-based therapies, including ASOs, siRNAs, shRNAs, antimiRs, miRNA analogs, etc., have been developed. These advances have facilitated the development of ncRNAs as a novel class of therapeutic. There are already some examples of ncRNA therapeutics being used in rare disease research:

Table 1 Rare disease clinical trials with ncRNA therapeutics. (Huang, Cheng-Kai, et al., 2020)

Targeted miRNA Developmental Drug Chemistry/Mechanism Indication Sponsor/Collaborators Clinical Trial Identifier Phase
miR-16 Mesomir TargoMir Malignant pleural mesothelioma Asbestos Diseases Research Foundation/EnGeneIC Limited NCT02369198 Phase I
miR-34a MRX34 miRNA mimic Cancer/melanoma (advanced) Mirna Therapeutics NCT01829971 Phase I
miR-21 RG012 AntimiR Alport syndrome Sanofi Genzyme NCT03373786 Phase I

Our Services

Our research team is dedicated to overcoming the challenges faced in the development of noncoding RNA therapeutics, particularly the chemical modification of noncoding RNA and the development of delivery vectors suitable for the target organ and cell type. By focusing on promising preclinical advances, we hope to further advance the development of noncoding RNA interventions for the treatment of many types of diseases including rare diseases. Our services include but are not limited to:

siRNA Therapeutics Development

To improve the therapeutic efficacy of siRNA-based therapies and reduce the side effects of siRNA, our researchers provide customers with a variety of chemical modifications of siRNA and multiple delivery systems.

Ribozymes Therapeutics Development

Ribozymes show tremendous promise in the treatment of rare diseases. To help customers develop ribozyme therapeutic drugs more efficiently, our company is committed to providing ribozyme development and delivery services.

miRNA Therapeutics Development

With top technical talents and a complete platform, our company can target the selection and design of miRNAs and provide you with miRNA activity modulation and delivery services for miRNA therapeutics.

lncRNA Therapeutics Development

Our scientists are adept at using long non-coding RNA molecules to regulate gene expression to treat rare diseases, we provide our clients with tools and methods to study lncRNA therapeutics and design tissue- and cell-type-specific genomic drugs.

tRNA Therapeutics Development

Leveraging our deep understanding of tRNA biology and gene regulation, we employ state-of-the-art computational tools and experimental approaches to design tRNA therapeutics with enhanced efficacy and safety profiles.

Project Workflow

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With a research team with extensive expertise, our company is confident to provide clients with noncoding RNA therapeutics development services for rare diseases. We have the capabilities and resources to provide professional communication and problem-solving support to ensure that we can quickly respond to the changing needs of your rare disease therapy research projects. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.


  • Ding, Lifeng, et al. "Role of noncoding RNA in drug resistance of prostate cancer." Cell death & disease 12.6 (2021): 590.
  • Huang, Cheng-Kai, et al. "Preclinical and clinical development of noncoding RNA therapeutics for cardiovascular disease." Circulation research 126.5 (2020): 663-678.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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