mRNA therapeutics have emerged as a revolutionary force in medicine, riding the wave of success initially generated by mRNA vaccines for COVID-19. However, as the field progresses from vaccines to protein replacement therapies and beyond, it faces distinct hurdles, particularly in the realm of delivery.
Challenges of mRNA Drug Development
Unlike smaller RNA molecules like siRNA or antisense oligonucleotides, mRNA molecules are significantly larger and inherently unstable. This instability arises from their natural function as transient messengers carrying genetic information from DNA to ribosomes for protein synthesis. In the context of drug development, this instability poses challenges in manufacturing, storage, and delivery.
Enzymatic production, a key step in mRNA synthesis, can lead to variations in size and composition, complicating quality control and reproducibility. Moreover, the large size of mRNA molecules hinders their efficient delivery to target cells and tissues. The body’s natural defense mechanisms readily recognize and degrade foreign RNA, further compounding the delivery problem.
Delivery: The Key Hurdle
Traditional approaches employed for smaller RNA molecules, such as chemical modifications or targeting ligands, often prove ineffective for larger mRNA molecules.
Nanoparticle delivery systems have emerged as a promising solution. These tiny carriers encapsulate mRNA, shielding it from degradation and facilitating its uptake by target cells. Lipid nanoparticles (LNPs), for instance, played a pivotal role in the delivery of mRNA vaccines. However, further advances in nanoparticle design and formulation are necessary to address issues like targeted delivery, controlled release, and potential toxicity.
Addressing the Delivery Challenge: Areterna’s Contribution
Areterna distinguishes itself as a comprehensive hub for mRNA raw materials and services. Going beyond its role as a cost-effective supplier of mRNA products and services, we empower researchers with a range of integrated solutions.
For mRNA-LNP research, Areterna stocks a list of ready-to-use ‘Tool’ mRNAs for LNP encapsulation and delivery research, including eGFP mRNA, Flue mRNA, OVA mRNA and Fluc-eGFP mRNA.
To facilitate effective delivery, we provide expert mRNA-LNP encapsulation services, offering a choice between established high-quality formulas (SM102, ALC0315, MC3) or the exploration of our diverse library of innovative lipid combinations.
Customers requiring customized mRNA-LNP may benefit from our AI-assisted LNP screening system, which uses a high-throughput platform to expedite the identification of optimal lipid nanoparticle formulations.
The Path Forward
By offering these resources, Areterna empowers researchers to overcome delivery challenges and accelerate the development of innovative mRNA-based therapies. Advances in nanoparticle technology, coupled with the exploration of novel delivery strategies and the contributions of companies like Areterna, hold the key to unlocking the full potential of this groundbreaking therapeutic modality.
References:
- Nature Biotechnology article on mRNA therapeutics (https://www.nature.com/articles/s41587-022-01491-z)
- News-Medical article on mRNA drug challenges (https://www.news-medical.net/news/20240124/Balancing-efficacy-and-safety-The-challenges-of-mRNA-drugs-and-vaccines-in-modern-medicine.aspx)