Originally developed for therapeutic delivery of RNA, Mirus’ LPNCs have been adapted for a wide range of gene delivery applications.
The deal expands Gamma Biosciences' reach in gene therapy and next-generation vaccines, while supporting the launch of Mirus Bio's flagship TransIT VirusGen platform for manufacturing of adeno-associated viruses (AAV) and lentivirus-based viral vectors for gene therapy.
The investment will also help to further develop and commercialize TransIT VirusGen for in vivo delivery applications, according to the parties.
The financial details of the agreement were not disclosed. Current Mirus shareholders will retain a substantial equity ownership stake in the company.
Demand for efficient and scalable gene delivery technologies continues to increase as larger volumes of viral vector are required to support the growing number of gene therapies in development, said Gamma Bio, an investment platform supported by global investment firm KKR.
High productivity
When asked whether Gamma Bio see Mirus’ technology as game changing in terms of viral vector manufacturing, and, if so, in what way, Philip Vanek, CTO, Gamma Bio, told BioPharma-Reporter:
"To deliver the quantities of functional virus necessary for an adult somatic cell gene therapy - 10e15 or 10e16 functional virus particles per patient or more - manufacturers rely on highly efficient packaging of the viral componentry and the necessary functional gene in a production cell line such as HEK 293. Traditionally, manufactures have depended on conventional lipids or polyethylenimine (PEI) or combinations to balance delivery efficiency with low cellular toxicity. While these technologies do allow routine transfection at smaller scale, they are not optimized for high efficiency manufacturing at the scale necessary for larger format (200L plus) bioreactors.
"Mirus’s technology brings the best of all worlds: high efficiency viral particle production, as determined by functional viral titer and not simply viral genomes, with the necessary quality standards suitable for therapeutic manufacturing. The high productivity of the manufacturing platform, and the high quality of the manufactured virus is substantially improved when compared to competing technologies, finally making large scale viral manufacturing possible."
Cell engineering
And how do Mirus Bio’s engineering and delivery capabilities complement and extend Gamma Bio’s advanced therapy offering?
"Gamma invests in technologies that are readily deployable into most biologics, vaccine, cell and gene therapy manufacturing workflows. We take a process-level or systems-level view of the challenges faced by therapeutic developers, and work to improve efficiency and productivity in the near-term, while keeping our focus on innovative and disruptive future technologies to build upon the foundational platforms we have acquired.
"We are convinced that cell engineering, whether for biologics cell line development, or for future genetic manipulation of cells in vivo or ex vivo, will be foundational to next generation therapies. By adding Mirus to the Gamma portfolio, we provide a more comprehensive capability to our customers from cell line development, through cell culture scale-up (upstream manufacturing) and bioseparations (magnetic cell sorting and both conventional and affinity chromatography). So, we’re now able to provide a suite of unique cell manufacturing technologies spanning cell line development through final product formulation, all of which are designed for cGMP manufacturing at scale."
Scientific expertise
Mirus’s scientists are world class at biomolecular delivery into a wide variety of cells, he continued.
"Its IP protected technology is built upon novel biomimetic LPNCs that interact with cells in much the same way that viruses infect cells in vivo. By engineering these LPNCs in a way that maximizes the delivery of DNA, RNA or other biomolecules into cells, while minimizing cytotoxicity, and most importantly leverages the intracellular vesicle machinery to traffic the delivered molecules into cells – getting the right materials to the right part of the cell in an orchestrated fashion – improves the functional performance of almost any cell engineering workflow, including viral vector manufacturing."
While Mirus' TransIT VirusGen product has been available for RUO and process development applications for more than a year, with users demonstrating considerable productivity improvements, the cGMP manufacturing grade version is anticipated to launch in the third quarter of 2021, added Vanek.