A Discussion on the Outsourcing of mRNA Production (BIO 2024)

At BIO 2024 in San Diego, Calif., which was held on June 3–6, Pharmaceutical Technology® Group’s Chris Spivey spoke with Christian Cobaugh, PhD, Vernal Biosciences’ CEO and founder, about the outsourcing landscape for messenger RNA (mRNA) production. Vernal’s simplified business model for manufacturing all stages of mRNA R&D galvanized the company into contributing comments to FDA’s Guidance on Platform Technology Designation Program for Drug Development (1), in which—once consistent with the statutory conditions required to be compliant with chemistry, manufacturing, and controls (CMC) processes—applicants can re-use certain foundational modules without having to go backwards and prove that module repeatedly. Cobaugh, along with Vernal’s senior director of commercial and technical operations, Grant Henderson, PhD, and guest contributor, Khaled Yamout, analytical sciences, quality, and manufacturing consultant at Y-Chem Consulting, shared their thoughts in an Aug. 1, 2024 blog article (2).

Looking at the field of mRNA service providers Cobaugh divides the landscape into two categories. “There [are] those that just generally do GMP [good manufacturing practice] for multi different types of drug products. and then there [are] more specialty companies that were set up to make nucleic acids. In our position [in] mRNA, we have several years of trial runs on the manufacturing technology platform, performing research-grade processing before we flipped over into GMP,” he said in the BIO interview with Spivey.

While innovation on specific steps were needed, one of the areas requiring the most improvements were the analytics, Cobaugh said, noting that “there's a lot of complexity there. Part of manufacturing is controlling what you're manufacturing, and, in order to do that, you've got to have unimpeachable analytical and QC [quality control] capabilities. And so that was something that kept me up at night, [but] no longer does. The number of platforms that we've got to use for release testing is quite daunting. and we've mastered pretty much all of them.”

Among the most promising of the new higher performing analytical approaches is next-generation sequencing (NGS). Cobaugh pointed out that “NGS offers … an opportunity to go deeper and pick off multiple critical quality attributes from the same test, which, along with consolidating platforms more broadly, presents significant cost of goods savings in the long run.”

Located in Vermont, Vernal has been manufacturing mRNA and lipid nanoparticle (LNP) products for clients since 2021. The company offers custom mRNA, mRNA sequence design, GMP mRNA and LNP–mRNA manufacturing, and LNP-formulated mRNA products. Vernal takes a stage-gated approach to offer GMP mRNA and LNP production services and recently upgraded its manufacturing space, QC lab, warehouse, and additional lab and office space in a total building area of 23,000 square feet. The company is also planning a new 20,000 square-foot GMP mRNA manufacturing plant, which it expects to bring on line in the near future.

References

1. FDA. Draft Guidance for Industry, Platform Technology Designation Program for Drug Development (CDER, CBER, May 2024).
2. Cobaugh, C.; Hendersonm G.; Yamout, K. FDA Guidance on Platform Technology Designation Program for Drug Development. vernal.bio/news-insights/blog-fda-guidance-on-platform-technologies, Aug. 1, 2024.