For researchers, universities and startups, determining when data is sufficient for a patent application can be challenging. Patent laws continue to evolve, particularly in the life sciences space, and the standards differ significantly from those required for scientific publications.
Understanding these distinctions is crucial for securing strong IP protection. In this article, we explore the types of data needed for different patent claim types, highlighting key differences in how data is assessed in patents versus scientific journals.
Case study: Gene therapy
The field of gene therapy is rapidly expanding and is providing a new strategy for treating diseases such as cancer. Generally, cancer gene therapies involve delivering genetic cargo to a specific tissue where expression of a gene of interest is then either up- or down-regulated, where the ultimate purpose is to provide a therapeutic intervention for the treatment of the cancer. Several approaches have been developed for cancer gene therapy, including naked nucleic acid therapy, microRNA therapy, oncolytic virotherapy, suicide gene based therapy, cell mediated gene therapy, and CRISPR/Cas based therapy.
Unsurprisingly, this field produces a large number of exploitable and commercially valuable patents.
There are often a number of aspects of a cancer gene therapy which may be patentable, including:
- the specific therapeutic compound
- methods of treating the cancer using the compound
- the delivery system which has efficacy in delivering the compound to a tissue area
- methods of producing the compound, and
- pharmaceutical compositions comprising the compound.
Unlike patent applications, scientific data is typically generated without commercial applications in mind. Instead, there is a focus on substantiating the scientific validity and robustness of the data. Whilst there is some crossover, having data sufficient for a patentable cancer gene therapy will generally be viewed quite differently during the patenting process.
Putting aside the requirements for any claimed invention to be both novel and inventive, which are discussed at length elsewhere (for example, please see the following article regarding inventive step), there are important legal grounds that play a significant role in determining the ultimate scope of the claims and by extension, the commercial value of the patent to stakeholders. The relevant legal grounds are called Sufficiency and Support, and require a comparison of the disclosure and data in a patent application with the scope of the claims that define the invention.
Sufficiency and Support
In Australia, for a claim to be enabled and supported, the following two respective requirements must be met.
- The specification must disclose the invention in a manner which is clear enough and complete enough for the invention to be performed by a person skilled in the relevant art,[1] and
- a patent claim must be supported by the specification.[2]
In relation to the first requirement, this assessment involves considering whether it is plausible that the invention can be worked across the full scope of a claim and whether there would be an undue burden for a skilled person to perform the full scope of the claimed invention.[3] As for the second ‘support’ requirement, a relevant question is whether the scope of the claims extends beyond the technical contribution made by the inventors, as described in the specification.[4]
This can be further explained using the following hypothetical claims.
Hypothetical claim 1
A method of treating cancer, the method comprising administering to a subject a therapeutically effective amount of a composition capable of modulating expression of gene X.
This claim is directed towards treatment of any cancer and, accordingly, the patent application will need to include data which demonstrates that treatment of all cancers would be plausible using the composition. For this reason, we recommend that the research investigate multiple different forms of cancer (e.g., colorectal, breast, melanoma, etc) in the course of testing a therapeutic candidate, in order to provide as broad of protection as possible in a patent application. Typically, this could be achieved by testing efficacy in various cancer cell lines and does not necessarily require in vivo data. Alternatively, if the therapeutic target is known to be dysregulated in a number of different cancers, this may support the plausibility of a claim to the treatment of any cancer.
Another issue with the breadth of this claim is that it encompasses any composition capable of modulating expression of gene X including CRISPR-Cas components, miRNA, siRNA, lincRNA, etc. However, it is typical for a patent applicant to have only exemplified one of these in the application (e.g., CRISPR/Cas based modulation). Furthermore, an exemplification of one or two sgRNA sequences of a given CRIPSR-based technology will almost always require the claim to be limited to this scope.
In our experience, if a broader claim scope is desirable, an applicant will need to experimentally verify that a number of different modalities – sgRNA, miRNA, and siRNA, for example – have utility in targeting the expression of gene X. Moreover, obtaining a claim to a given class of modulator will require a general technical principle, such as a common sequence or structural relationship, that imparts the required function.
Hypothetical claim 2
A pharmaceutical composition for treating cancer, the composition comprising a nucleic acid sequence having at least 70% sequence identity to SEQ ID NO: 1.
This claim defines a composition by reference to a sequence that is disclosed in the specification but allows variation in the nucleotides in the sequence. This variation is desirable to a patentee as it will capture potential infringers that may intend on changing a small number of nucleotides for the purpose of escaping infringement.
Obtaining allowance to a claim with a nucleic acid with 100% sequence identity can therefore be considered to be of limited commercial value. However, in order to obtain allowance for 70% sequence variation recited above, the specification will need to provide examples of sequences that have varying levels of sequence identity to SEQ ID NO: 1 (i.e., with as little as 70% identity) that still show efficacy in treating cancer.
Without experimental validation, it is often necessary to recite a degree of sequence identity that aligns with the sequences used in the disclosed experiments. In many cases, only claims to the exact identity will be allowed.
In addition to the above considerations for claim 2, it is also worth highlighting that the composition “comprises” the recited nucleic acid sequence. This term is construed in many jurisdictions (including Australia and New Zealand) as including of the recited nucleic acid sequence but does not limit the addition of further nucleotides.
This is often a strategic decision employed when drafting where SEQ ID NO: 1 does not recite the full nucleic acid sequence which is used in the examples section. For example, for a naked mRNA therapy, SEQ ID NO: 1 may define the coding sequence of the mRNA sequence without the 5’ or 3’ UTR sequences. However, if the UTR sequences are particularly important for the efficacy of the mRNA therapy, a lack of support objection may be raised unless the specification demonstrates that different UTR sequences can be used with SEQ ID NO: 1 to provide an efficacious cancer therapy.
In other words, features of the nucleic acid that are essential for the functional effect will need to be defined in the claims.
Hypothetical claim 3
A viral vector comprising a nucleic acid sequence represented by SEQ ID NO: 1.
This claim is directed to any viral vector comprising the nucleic acid gene therapy. To obtain allowance of a claim of this scope, the specification will need to provide different examples of viral vectors (e.g., adenovirus, adeno-associated virus, and lentivirus) that effectively deliver the genetic cargo in the treatment of cancer.
As above, testing different viral vectors may be accomplished using a suitable in vitro cell model without needing to conduct in vivo animal experiments.It is also worth considering that the specific viral serotype may be important in treating a specific cancer subtype. It may have a particular efficacy in targeting a cell type (i.e., tissue tropism) that is important to effectively treat the cancer, for instance.
In this case, unless the specification discloses data that demonstrates the range of viral vectors outlined above and that other serotypes retain the same functional effect, such as tissue tropism, the claim is likely to be limited to a particular viral vector having a particular serotype.
Engage assistance early
Maximising the commercial potential of your research starts with careful consideration of patent support and sufficiency requirements. Engaging with a patent attorney early in the process ensures your experimental plan aligns with patent laws, giving you the best chance of securing strong, broad claims that enhance the commercial viability of your technology.
If you’re navigating these decisions, our team can provide tailored guidance to help you protect and maximise the value of your innovation. Reach out to us to discuss your research and IP strategy.
[1] Patents Act 1990 (Cth) s 40(2)(a).
[2] Patents Act 1990 (Cth) s 40(3).
[3] Explanatory Memorandum, Intellectual Property Laws Amendment (Raising the Bar) Bill 2011 (Cth) 46–7; ToolGen Incorporated v Fisher (No 2) [2023] FCA 794 [400].
[4] Boehringer Ingelheim Animal Health USA Inc v Zoetis Services LLC [2023] FCA 1119 [535].