Advanced therapies encompass gene therapy, somatic cell therapy, and tissue-engineered medicines offering innovative solutions for treatment of once incurable diseases and improving patient care. Encapsulated delivery mechanisms within the gene therapy space include adeno-associated viruses (AAV) and lentivirus viral vectors.
Messenger RNA (mRNA) therapeutics involve the production and purification of nucleic acids (e.g., DNA, RNA and pDNA) as well as encapsulation into delivery vehicles (e.g., lipid nanoparticles, LNP). The development and production of mRNA and vaccine products are continually evolving and rely on innovative purification technologies, process optimization, stringent quality control measures, and adherence to regulatory guidelines.
These workflows present several unique challenges due to the delicate nature of the molecules, process complexity, and rigorous safety and quality regulations. Furthermore, mRNA-based vaccines using LNP encapsulation have the added difficulty of low process yields.
With a combination of single-use and stainless solutions, VERDOT provides greater workflow flexibility and increased process efficiency with GMP technologies for commercial scale-up of advanced therapies and mRNA applications.