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Development of RNA Therapies for Rare Diseases

Development of RNA Therapies for Rare Diseases

As an indispensable technology for the treatment of human diseases, RNA-based therapy provides an effective alternative for those diseases that are not suitable for traditional gene therapy. Our company is committed to providing a complete scientific pathway to help our customers develop antisense oligonucleotides (ASO), microRNA (miRNA), small interfering RNA (siRNA), and other therapies. Our comprehensive scientific services for the development of gene therapy products will contribute to the advancement of gene therapy for rare diseases.


RNA-based gene therapy is an approach to treating disease by sequence-specific interference with RNA expression in target tissue cells. RNA therapy can be used to target human and viral transcriptomes, regulate gene expression, alter mRNA splicing, target transcription, and more. Compared with traditional gene replacement therapy, targeted RNA therapy can avoid problems such as the transfer and regulation of exogenous DNA in vivo. Therefore, it became an increasingly popular method for the treatment of genetic diseases.

Theoretically, RNA therapies can be designed to silence the protein production process with the knowledge of the gene coding sequence of the disease-causing protein, and the development cost will be greatly reduced. This has natural adaptability for many rare diseases. Although RNA delivery to target organs and effective cell entry remains challenging, the advantages of targeting undruggable targets, fast production, long-term effect, and usefulness for rare diseases make the development of RNA therapies a worthwhile investment. Currently, RNA therapies have been highly successful in the treatment of rare diseases, particularly hepatic and neurological diseases.

Fig. 1 Diverse cellular molecules can be targeted by RNA therapy.

Fig. 1 Diverse cellular molecules can be targeted by RNA therapy. (Kim Y K, 2022)

Development of RNA Delivery Biomaterials

A growing number of RNA molecules are emerging as promising therapies for rare diseases. We help our customers explore and develop a variety of RNA delivery biomaterials to enable RNA therapies to treat various rare diseases.

  • Lipid-based delivery systems
    Our researchers are dedicated to helping our customers create lipid delivery systems with a variety of different chemical structures, including micelles, liposomes, and lipid nanoparticles (LNPs) to dramatically improve the efficiency of targeted delivery of RNA therapeutics.
  • Polymer and polymer-based nanoparticles
    We help our customers develop a variety of RNA delivery polymers, such as dendrimers. We improve the efficiency of the polymer to deliver RNA into the cell by modulating the polarity, degradability, and molecular weight of the polymer.
  • Peptide nanoparticle delivery system (PNP), exocrine delivery, bacteriophage and bacterial micro-cell delivery, and other delivery systems.

Our RNA Gene Therapy Development Services for Rare Diseases

RNA-based therapeutics can be classified into ASO, siRNA, aptamer, and mRNA depending on their structural characteristics and mode of action. We offer different RNA-based drug development services to our customers.

Development of ASO-Based Gene Therapies

Development of siRNA-Based Gene Therapies

Development of Aptamer-Based Gene Therapies

Development of mRNA-Based Gene Therapies

Development of Noncoding RNA Therapeutics

Development of RNA Editing-Based Gene Therapies


RNA therapies can be used in the treatment of a variety of rare diseases, including but not limited to:

  • mRNA therapy for methylmalonic acidaemia
  • mRNA therapy for spinal muscular atrophy (SMA)
  • mRNA therapy for acute intermittent porphyria

Our company has established an industry-leading gene therapy development platform that enables us to provide integrated solutions and complete scientific services to our customers, covering the entire development cycle of activities. In addition, our scientific experience and broad product portfolio give us a competitive advantage in the development of novel, breakthrough RNA therapeutics. If you are interested in our services, please contact us for more details.


  • Kim, Y. K. RNA therapy: rich history, various applications and unlimited future prospects. Experimental & Molecular Medicine, 2022, 54(4): 455-465.

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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