Expert Analytics Drive Successful mRNA Therapeutic Development Programs

Messenger RNA has redefined what rapid looks like in drug development. Yet the very flexibility that makes mRNA attractive also makes it unforgiving—minute variations in purity, structure, or LNP encapsulation can eliminate potency or introduce safety risks.

It’s never too soon to start designing a characterization and quality strategy because analytics cannot be an afterthought. Instead, they are the scaffolding that keeps the entire mRNA development program on solid ground.

Below is a closer look at the value Vernal’s analytical services provide to mRNA therapy developers and what sets our Vermont‑based CDMO operations apart.

To understand the data generated, whether it is a simple cell biology screen, a preclinical study in animals, or even in the clinic, you must understand the consistency and quality of the test material you are working with. Analytical services lay the groundwork for that understanding. For successful development and quality management, we have to understand factors such as:

• mRNA integrity & capping efficiency
• Purity and dsRNA content
• LNP particle size, polydispersity, and encapsulation efficiency
• Residual solvents, endotoxin, and bioburden levels

With an early comprehensive understanding of CQAs, developers can make the best decisions, be more efficient, and ultimately increase speed to clinic. Developers can also avoid costly mistakes like dismissing a successful construct; insufficient analytical understanding might lead a developer to conclude that a sequence is ineffective when the problem is a hidden impurity causing a lack of potency.

The technologies required to evaluate the quality and purity of mRNA and LNPs from discovery to commercial manufacturing are extensive and a massive investment for an innovator or mRNA service provider. Therefore, it is rare for one organization to have the ability to support all phases of development. However, Vernal decided to build capabilities to do exactly that— offering innovators comprehensive capabilities from discovery biology through the clinic and ultimately to the market.

The upside of this approach to developers is two‑fold:

• One Data Thread Across Development Phases: Vernal can release research use only (RUO) batches for discovery, scale them for IND‑enabling studies, and produce GMP clinical lots without the analytical reset that often happens when work is split between vendors. Vernal’s phase‑appropriate approach facilitates the maturing of methods in step with the program instead of reinventing methods at each milestone.
• Standardized Yet Tailorable Assays: Vernal has platformed core mRNA analytical methods, validated them, and, where needed, pushed beyond current USP or even FDA expectations. For sequence‑ or lipid‑specific assays, Vernal dramatically shortens timelines by layering custom method development onto the company’s platform framework, eliminating duplicate validations while meeting molecule‑specific requirements.

Regulatory agencies increasingly expect mRNA sponsors to justify every surrogate they use to claim quality or potency, so Vernal’s team works backward from those expectations. The Vernal team defines the decision-enabling assays at each stage and facilitates collaboration with regulators.

Generally, analytical objectives at key development inflection points include:

• Discovery & Lead Optimization – Rapid fit‑for‑purpose screens that rank candidates without over‑engineering.
• Pre‑IND – Stability understandings, LNP characterization, and identity tests suitable for IND-enabling studies, including a well-defined CMC program.
• Clinical & GMP – Full ICH‑aligned release and stability panels, method validation, and reference standards ready for tech transfer or BLA filing.

The result is a data package that grows in depth, not redundancy—speeding IND, setting up late-phase success, and minimizing rework.

A successful mRNA development program begins with a well-constructed analytical strategy, and given the lack of an established mRNA regulatory framework, partnering with mRNA experts is essential. Despite extremely rapid COVID-19 vaccine emergency approvals, the regulatory pathway for approving the many mRNA therapeutics currently in various phases of development is far from established.

The mRNA regulatory landscape is evolving and will solidify, but advancing today’s mRNA programs requires expertise, experience, and open communications with regulators. Vernal’s exclusive dedication to and leadership within the mRNA therapeutics manufacturing space allow us to support our clients’ programs throughout the development process, even though guidances and other regulatory framework are not established.

The promise of mRNA lies in its speed and adaptability. The risk lies in its sensitivity. Vernal Biosciences bridges this gap with analytical capabilities purpose‑built for mRNA and LNP medicines—standardized where possible, customized where necessary, and integrated from preclinical to GMP. By aligning assays with regulatory endpoints, collaborating openly on risk‑based strategies, and housing manufacturing and testing under one roof, Vernal gives developers a clearer, faster line of sight to the clinic—and, by extension, to patients in need of therapeutic options.

For innovators mapping the next generation of mRNA therapeutics, the message is clear: analytics are not an afterthought; they are the roadmap. The Vernal Bio team works with innovators to chart the course.

Interested in seeing how Vernal’s analytical platform can support your next milestone? Contact us to start the conversation.