According to AMRI, RNA-based therapeutics offer a new approach to health, but assessment tools are limited. As part of the agreement, The RNA Institute will provide RNA technology, new assays, and materials to support AMRI in its drug and diagnostics discovery programs.
“With the development of RNA-based therapeutics, biopharma requires assays and tools to characterize them for FDA approval and batch to batch stability,” Dr. Paul Agris, Director, The RNA Institute, told Outsourcing-Pharma.com.
“AMRI has the in-house expertise to develop these assays and tools for small molecule and protein-based therapeutics, and wanted to partner with RNA experts at The RNA Institute to develop and extend these tools for RNA-based therapeutics,” added Agris.
While The RNA Institute has the expertise to develop RNA-based analytical assays and tools, it does not have the facilities to provide services to biopharma.
“This is an ideal and first of its kind in RNA-based therapeutics for a collaboration that leverages the core competencies of The RNA Institute and AMRI for bringing a new class of drugs to market to treat and cure many diseases,” said Agris.
The collaboration is set to last multiple years.
“Since RNA therapeutics are at the beginning of their product life cycle and hold promise for the treatment of many intractable diseases, we expect the collaboration to grow as new drugs are discovered and requiring new tools and assays,” said Agris.
The “most significant” next steps for The RNA Institute will be working with pharmaceutical and biopharma companies to analyze their RNA drugs in development.
“Simultaneously AMRI will optimize for scalability of assays and tools so they can provide them as a more effective and efficient service to biopharma for their drug products,” added Agris.
Developing RNA-based therapeutics
RNA is a unique molecule, as it is produced on demand in the cell when needed before being immediately degraded.
“Of course we need reliable, robust, effective and efficient protocols to improve development and implementation of therapeutic applications,” said Agris, who said there are two unique RNA characteristics that pose challenges to creating analytical tools.
“It [RNA] is unstable when isolated from the cell and easily degraded by proteins on our fingers; and it can only be isolated from the cell in very small quantities for analysis,” he explained.
These characteristics create challenges in analytical tool development for assessing RNA-based therapeutics as it requires a laboratory environment devoid of proteins in order to maintain RNAs’ integrity when isolated from the cell, Agris explained.
The process also requires “extremely sensitive analytical tools that can measure the characteristics of RNA-based therapeutics in attomole quantities,” said Agris.