IP Considerations For Early-Stage mRNA Therapeutics Development

By Anna Rose Welch, Editorial & Community Director, Advancing RNA

In my previous life in the biosimilars sector, intellectual property was a (very) common topic of conversation. I couldn’t read or write a story that didn’t reference the infamous “patent dance” between newly commercialized biosimilar companies and innovator companies.

That’s not to say that IP isn’t/hasn’t been a central pillar in the world of advanced therapies; on the innovator side of the industry, we regularly emphasize how important it is that our scientific and manufacturing innovations be valued and protected. Regardless of its importance, it’s been somewhat rare — at least in my circle — to stumble upon prominent news stories about litigation in the CGT space. The mRNA sector of the biologics/ATMP industry, however? Well, that’s another story. Thanks to the commercialization of the COVID vaccines, the high stakes litigation landscape is alive and well on the commercial market. Likewise, I’ve been encountering conversations about how IP figures into early-stage mRNA therapeutics development much more frequently as a result.

My conversation with Rothwell Figg Partner Dan Shores started first with a high-level overview of the current litigation landscape, as well as how he sees IP encouraging — rather than limiting — scientific innovations in the RNA field.

However, we also can’t sugar coat reality: RNA companies innovating today still have much to contend with when navigating the patent landscape to ensure efficient development. Fortunately, as Shores reassured, navigating this landscape is hardly a “doomsday” scenario. But it is a challenge biotechs need to tackle early so they don’t end up too far down the path toward the clinic or in the clinic mired with delays and expensive litigation.

Here, Shores shares several strategies companies may choose to employ when facing a legal gray area to protect their products from patent litigation upon commercialization.

Proactive IP Management: A Key to mRNA Success

In the CMC world, we often talk about the importance of making big changes early in development to avoid the development/regulatory challenges and costs of later-stage comparability. In the legal world, it turns out, we need to abide by a similar mantra. During our conversation, Shores emphasized the importance of evaluating the patent landscape as early as possible because any potential infringement concerns can be addressed more efficiently and cost-effectively.  

“Even if there may never end up being a lawsuit once you commercialize your product, you need to address any potential infringements upfront because you never want to launch a product at risk,” Shores clarified. Launching at risk could ultimately result in a lawsuit. Should the courts rule in favor of the other party, a young biotech could find itself owing a hefty chunk of change in royalties.   

Naturally, my next two questions for Shores were, firstly, if and how — at a high level — we can differentiate a potential infringement from an actual infringement early in development; and how companies can course correct in the face of potential infringement.

He offered the following scenario in which a company, upon investigation, determines that an ionizable cationic lipid in their LNP formulation is owned by another party.

There are a handful of ways companies may choose to handle or “course correct” upon this discovery — a path that may in part be dictated by the amount of life remaining for the patent in question and a product’s commercialization timeline.

On the one hand, companies may find they can rely on safe harbor laws to use an invention in clinical trials without issue.

“Say, for example, someone else owns the cationic lipid you’re using in your LNP formulation,” Shores outlined. “Thanks to U.S. safe harbor law, very generally speaking, you have some leeway to use that patented invention so long as the use is reasonably relating to regulatory approval. You just cannot use the invention after approval or in commercialization. However, say the patent in question expires in two years, and your product won’t be commercialized for three years. In turn, assuming the safe harbor properly applies, you could choose to run the clock and take advantage of that safe harbor throughout development and launch following the patent’s expiration.” (Please note, however, that each nation does have different safe harbor laws.)  

In cases where the timeframes for expiration and commercialization are not so neat and tidy, companies face an alternative three options:

• The biotech can license the innovation from the patent holder. However, it’s important to note that the patent holder is not obligated to license their invention to anyone, especially if they consider the biotech seeking the license to be a competitor.
• In some cases, a lipid may be owned by a third-party company that seeks to license its lipids, in which case the biotech may find a suitable partner that could potentially also offer access to additional lipid options and formulation development assistance.
• Finally, though Shores acknowledged it’s the most resource-intensive of the three options, a company can also choose to develop a novel lipid — a move that can add to a company’s own IP portfolio. However, as he also indicated, the space for innovation as it relates to singular lipids is growing increasingly crowded; accordingly, looking to create a novel formulation — or combination of lipids — may provide a company with a much more fruitful source of IP to protect.

Shores and I will return in one more installment outlining several of the biggest outstanding litigation questions facing the mRNA-LNP space to-date and how these could go on to impact the RNA field in general. Stay tuned!