miRNA Therapeutics Development
MicroRNAs (miRNAs) are a class of small RNA molecules with a single-stranded structure that play important roles in development and cellular processes. Our company has extensive experience in the development of miRNA therapeutics and has been continuously building and improving gene therapy development platforms for many years. We provide you with comprehensive miRNA development services to support your research and development of rare disease therapy research and development.
Overview of miRNA Therapeutics
MicroRNAs (miRNAs) are generally between 21-23 nucleotides in length and regulate gene expression at the translation level. miRNAs have a wide range of targets and can regulate various biological functions including metabolism, growth, development, immunity, etc. Since Victor Ambros' laboratory discovered miRNAs as important molecules in gene regulation in 1993, miRNAs have received widespread attention in the field of biology and have become attractive tools in disease research and novel therapeutic methods.
Fig.1 Application of therapeutic miRNA. (Chakraborty, Chiranjib, et al., 2021)
miRNA in Cancers
miRNAs play an important role in tumor proliferation, invasion, and metastasis, miRNA-based gene therapy is emerging as a novel strategy for rare cancers, which usually includes two therapeutics strategies:
miRNA Inhibition Therapies
Response to oncomiR overexpression by inhibiting miRNA activity.
miRNA Restoration Therapies
Involves restoring miRNA activity when oncogenic miRNAs are inhibited.
miRNA Therapeutics for Rare Diseases
By improving the in vivo stability of therapeutic RNA molecules and designing optimal delivery systems, minimal toxicity can be released during miRNA therapy. Because of its unique advantages, miRNA therapeutics play a major role in rare disease research. There are already some cases of using miRNA therapeutics for the research and development of rare disease therapies:
Table 1 Rare disease development phase with miRNA therapeutics. (Chakraborty, Chiranjib, et al., 2021)
| Molecules | Targets | Diseases | Companies | Stages |
|---|---|---|---|---|
| RGLS4326 | miR-17 | For the therapeutics of Polycystic kidney disease (PKD) | Regulus Therapeutics | Phase I clinical study |
| MGN-4893 | miR-451 | For the therapeutics of disorders like abnormal red blood cell production | miRagen therapeutics. | Preclinical stage |
| MRG-107 | miR-155 | For the therapeutics of amyotrophic lateral sclerosis (ALS) | miRagen therapeutics | Completed preclinical trial and entered in clinical trial |
Our Services
With top technical talents and a complete platform, our company can target the selection and design of miRNAs and provide you with one-stop development services for miRNA therapeutics. In addition, we provide customized services according to your requirements and develop efficient and economical rare disease research solutions for you.
- miRNA Activity Modulation
- Providing a variety of strategies to therapeutically restore miRNA activity. For example, by using synthetic RNA duplexes containing chemical modifications to improve stability and cellular uptake.
- Using miRNA sponges or antisense oligonucleotides called antimiRs to suppress mature miRNAs. Providing a variety of chemical modifications of antimiR oligonucleotides to achieve effective silencing of dysregulated miRNAs in vivo.
- miRNA Delivery
Our company has viral vector and non-viral vector development platforms to support miRNA delivery services. At the same time, we provide two strategies to deliver miRNA replacement therapies in vivo:
- Formulated, synthetic, double-stranded miRNA mimics
- Viral constructs over-expressing the lost or down-regulated miRNA
Project Workflow
With the participation of multidisciplinary researchers with extensive project experience, our company is committed to providing one-stop development services to help you accelerate miRNA therapeutics development for rare diseases. If you are interested in our miRNA therapeutics development services, please feel free to contact us for more details and quotation information on related services.
Reference
- Chakraborty, Chiranjib, et al. "Therapeutic advances of miRNAs: A preclinical and clinical update." Journal of advanced research 28 (2021): 127-138.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.