Transgenic mouse models have become valuable tools for advancing discoveries related to the diagnosis and treatment of neurodegenerative diseases. Once an animal model of a disease exists, it is possible to begin to understand disease mechanisms and to develop and test therapeutic approaches. The first transgenic animal model for a neurodegenerative disease potentially has considerable scientific and commercial value. Whereas the other chapters of this volume discuss the scientific aspects of various transgenic models, this chapter will address topics relating to the patenting and commercialization of such models. Collectively, we have worked to protect and commercialize transgenic mouse models of neurodegenerative disease for over 18 years, and will use some examples from that experience.

The development of a transgenic animal raises intellectual and tangible property issues (1). These issues relate to an institution’s ability to make, use, and sell such an animal. The two primary issues are: how does the institution (such as a university) protect the transgenic animal and did the university have the right to make or sell the animal in the first place? We will address each of these topics in turn.

The US government grants patents on novel, nonobvious, and useful inventions. The scope of novelty, obviousness, and usefulness, from a legal perspective, evolves over time as new cases add to the understanding of these concepts. However, it is a well-settled law that a party may obtain patent rights on a novel, nonobvious, and useful transgenic animal. Provided the invention meets these criteria, a party may also patent the gene construct, cell line, or techniques used to create the animal, in addition to the methods of using the animal, e.g., to diagnose a specified disease.

A patent allows the owner to exclude others from making, using, and selling the claimed invention. This exclusion applies to all activities, unless there is an exception under the law, of which there are very few. The Hatch–Waxman law provides an exception to acts of infringement in furtherance of seeking US Federal Drug Administration (FDA) approval for a product. However, the scope of this exception as it relates to research tools, such as transgenic animals, has not been settled. The reality is that uses of transgenic animals, including in a research setting, may constitute infringement of an issued patent claim.

A party owning a patent on a gene construct may prevent both use of that gene construct to develop a new transgenic mouse and use of the mouse if it is developed. This is true even if the mouse is used primarily for research purposes, as discussed above. There is no requirement that a party grant any right under a patent to any other party. Patents are still one of the few areas that permit a legalized monopoly, in the United States and elsewhere. In the United States, the patent term is 20 years from the date of filing, with extensions available under certain circumstances. Accordingly, a patent, which is enforced, can offer the patent owner very significant protection.

Filing for and obtaining a patent is costly. This process involves drafting a patent application with the help of a patent agent or patent attorney.¹ Once the patent application is drafted, it is filed with the US Patent and Trademark Office (PTO). The PTO examines the patent application to determine whether the application meets the requirements for patentability, fails to meet the ­requirements, or whether there is some other deficiency in the patent application itself. The PTO then issues an office action, which requires a response from the party seeking to obtain the patent. This process, called prosecution, is usually conducted with the assistance of a patent agent or patent attorney. Typically, prosecution involves two to three rounds of office actions and responses prior to obtaining an issued patent. Once the patent is issued, the party owning the patent is required to pay periodic maintenance fees. This process from application to maintaining an issued patent in the United States may cost tens of thousands of dollars in attorney’s and government fees. If the application is also filed overseas, the cost can balloon to hundreds of thousands of dollars.