As I have written about previously, the IP and competitive landscape for mRNA and lipid nanoparticles is complex. Pioneers, mid-stage, and new market entrants must take careful consideration of this landscape and develop a strategy to best position their businesses in this evolving space. This post provides an analytical framework for advanced IP considerations to achieve this goal.

What you do up front matters most

The most exciting thing about cell and gene therapy, besides the science itself, is the scope of possibilities for therapeutic application to treat human disease. The mRNA space is no exception, which has a wide array of potential indications  including infectious disease, oncology, rare disease, and other indications.

A company developing a therapeutic to address a specific disease indication has key decisions to make with regard to the design of its payload and delivery system. Because pioneers and more recent innovators have flooded this space with patents and applications directed to their inventions, careful consideration must be made of both legacy and new patents and applications to confirm that the subject therapeutic does not risk infringing this IP.

Doing this early in the process is critical because using a lipid, for example, that is covered by a third-party patent would require a license and in some cases – especially if the patent is owned by another market player – obtaining such a license may not be possible. The last thing you would want as a program is to spend the time and resources with regulatory only to be forced to launch at risk. Investors do not like that and having this risk on your profile could be detrimental to your ability to obtain needed funding and launch at all.

Accordingly, companies developing therapeutics should consult with their patent counsel very early in the process to develop a strategy to address freedom to operate. This analysis should take into account (1) infringement risks; (2) timing for commercialization; (3) timing for patent expiration; (4) market locations; (5) scope of applicable pre-commercialization safe harbors; (6) licensing; and (7) home-grown innovation.

Home-grown innovation

Oftentimes in this space developers will formulate a strong lipid nanoparticle delivery system for their novel therapeutic payload only to discover that one of the lipids they are using is covered by a third-party patent. Rather than viewing this as a roadblock, it instead presents an opportunity for innovation.

This has especially been the case with regard to ionizable lipids in 4-lipid lipid nanoparticle systems, as thousands of different ionizable lipids have been disclosed and/or claimed in patent applications and thousands more remain to be invented. This has been a ripe area for innovation and the number of patent applications claiming ionizable (as well as conjugated) lipids has skyrocketed over the past several years.

Not all of these lipids are equal, however. Each performs differently in different lipid and payload systems, which is to be determined during therapeutic optimization. Accordingly, patent applications directed to new lipids must take this into account and ensure that the patent claims provide proper coverage for the most promising lipids and their substituents and that the patent specification supports those specific compounds as required by 35 U.S.C. § 112.

Ideally, your patent portfolio should protect your foundational technology but also be significantly strong with regard to your market differentiators. For example, if you have a promising ionizable lipid, you will want to make sure you have a claim to the lipid itself in a general formula as well as separate claims for substituents. Such composition of matter claims – as compared to method of treatment claims – are the most valuable claims for licensing and enforcement.

Go to market with IP leverage

If you seek to in-license the lipids or other technologies needed for a therapeutic that you plan to launch, you will want to choose your partners wisely. Consider such engagements as long-term relationships which are often intended to last at least as long as the term of the licensed patents, which generally expire 20 years from their filing date. Think through what the relationship will look like in 5 or 10 years when a product is on the market and selling well and ask yourself whether you can live with the deal under those circumstances. This is perhaps the most difficult thing to clearly imagine when you are in early development.  

Also keep in mind that you can run parallel strategies where a third-party license may be necessary for your “get-on-base” product that establishes the proof of concept but leads the way for your additional follow-on therapies. In that situation, you can secure what may be a license for (e.g., the delivery system for) your first product, and in parallel develop your own delivery technology for your follow-on therapeutic that will not require a license.

In all cases when developing your own technology, you also have the opportunity to license it. Having patent protection on this technology (e.g., to a class of lipids) is crucial because that protection establishes the rights that comprise the central asset being licensed.  

You will want to work closely with your counsel and business team to develop licensing terms that will maximize the value of your IP portfolio while giving you flexibility to, for example, work with other partners. Moreover, while carrying sometimes significant costs, you will also want to seek and obtain patent protection in the U.S. and key markets abroad which increases the size and applicability of your portfolio increasing your odds for strong investment and acquisition value as applicable.

Disclaimer: The information contained in this posting does not, and is not intended to, constitute legal advice or express any opinion to be relied upon legally, for investment purposes or otherwise. If you would like to obtain legal advice relating to the subject matter addressed in this posting, please consult with your attorney. The information in this post is also based upon publicly available information, presents opinions, and does not represent in any way whatsoever the opinions or official positions of the entities or individuals referenced herein.