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RNA-based Therapy Development Platform

After RNA enters cells, it is regulated and expressed, thereby achieving the purpose of treating diseases. With many years of experience and a proven track record of quality, innovation, and customer support, our company provides comprehensive RNA based therapy development services for leading and emerging biopharmaceutical companies and scientific research institutions to accelerate your gene therapy research for rare diseases.

Introduction to RNA-based Therapies

RNA-based therapy is an approach to treating disease by sequence-specific interference with RNA expression in target tissue cells. RNA-based 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.

RNA-based Therapy Development Platform-1

Fig.1 Schematic diagram of RNA cellular functions. (Dykstra, P. B., et al., 2022)

RNA-based Therapies for Rare Diseases

RNA-based therapy has been highly successful in the treatment of rare diseases, especially neurological and hepatic diseases. The most commercially successful drug to date is Nusinersen, the world's first precision targeted treatment for SMA. Since 2018, Alnylam's Onpattro (Patisiran) has been the first siRNA drug approved by the FDA for the treatment of TTR protein amyloidosis, and Givlaari, Oxlumo and other siRNA drugs have been approved for the treatment of rare diseases. An increasing number of RNA therapeutics and drugs have been used in the study of rare diseases.

Our Services

RNA can be used as both a drug and a target. Our company's RNA-based therapy development platform provides you with different types of RNA synthesis and modification for rare disease gene therapy, diagnostic biomarkers or therapeutic targets, as well as RNA delivery services to help you deliver RNA drugs across the cell membrane to their targets. Our services include but are not limited to:

  • RNA Oligonucleotides Synthesis and Modifications

RNA oligonucleotides are short nucleotide sequences of RNA that can be used to treat rare diseases or in experimental research including gene chips, electrophoresis, and fluorescence in situ hybridization. We usually use chemical or enzymatic methods to provide you with RNA oligonucleotide synthesis services, including but not limited to:

miRNA Mimics Synthesis Double-stranded RNA (dsRNA) Synthesis
miRNA Inhibitor Synthesis Bridged Nucleic Acid Synthesis
miRNA Agomir Synthesis RNA Longmer Synthesis
miRNA Antagomir Synthesis Circular Oligonucleotide Synthesis
siRNA Synthesis Large Scale Oligonucleotide Production
mRNA Synthesis Antisense Oligonucleotide Synthesis
Single-stranded RNA (ssRNA) Synthesis Others

Based on your research and development needs, our company provides you with the following optional RNA oligonucleotide modification services:

SPO3 5' PO4 3' NH2 C7 Spacer 18 5' Dual Biotin 3' FAM
3' or 5' Digoxin 3' PO4 5' NH2 C12 dSpacer 5' PC Biotin 3' TET
3' DABCYL 3' C3 S-S NH2 C6 dT 5' Biotin 5' HEX 3' or 5' TAMRA
Ferrocene 5' C6 S-S Spacer C3 3' Biotin 5' Cy3 Internal TAMRA
dI 3' or 5' NH2 C3 Spacer C6 5' Biotin TEG 5' Cy5 5' or Internal Biotin dT
dU 5' NH2 C6 Spacer 9 3' Biotin TEG 5' FAM Others
  • RNA Purification

Different purification services are provided according to oligonucleotide length, modification, or sequence composition, which takes approximately 1-2 days, including but not limited to:

  • RNase-Free PAGE/SEC
  • RNase-Free IE HPLC
  • RNase-Free RP-HPLC
  • RNase-Free Dual HPLC
  • RNase-Free PAGE follow by HPLC
  • RNA Delivery Biomaterials Development

Lipid-based delivery systems

Our company is dedicated to helping our customers create lipid delivery systems with a variety of different chemical structures, including micelles, liposomes, and lipid nanoparticles (LNPs).

Polymer and polymer-based nanoparticles

Our company helps customers develop a variety of RNA delivery polymers, such as dendrimers. We I mprove the efficiency of the polymer to deliver RNA into the cell by modulating the polarity, degradability, and molecular weight of the polymer.

In addition, delivery systems such as peptide nanoparticle delivery system (PNP), exocrine delivery, bacteriophage, and bacterial micro-cell delivery are also options.

To ensure that we can quickly respond to your rare disease research needs, we have the capabilities and resources to provide one-stop RNA-based therapy development services, including but not limited to:

Project Workflow

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With a research team with extensive expertise, our company is confident to provide clients with RNA based gene engineering 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.

Reference

  • Dykstra, P. B., et al. "Engineering synthetic RNA devices for cell control." Nature Reviews Genetics 23.4 (2022): 215-228.

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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