IP priorities are especially important to businesses developing advanced therapeutic products. Although businesses developing cellular or gene therapy products operate on a range of different business models, these models typically involve the development of therapeutic assets. In order to produce a consistent, clinical grade therapeutic product and successfully negotiate the complex regulatory process required to bring the product to market, a small business will typically need to collaborate or license with, or be acquired by, a big pharmaceutical company or other larger entity.
Even getting to this stage is likely to be an expensive endeavour. Multiple rounds of fund raising may be needed to progress a cellular or gene therapy asset to a stage where it is sufficiently attractive for a larger entity to get involved. Once involved, the larger entity will then need to invest significant resources and money to take the asset through clinical trials and into the marketplace.
The ability to attract this level of investment relies on the parties being able to reap a sufficient reward if the asset is marketed successfully. If competitors are able generate copy-cat products as soon as the asset shows promise or becomes a commercial success, then the potential reward for the development of the asset is diminished and there may be no incentive for investors or larger entities to finance the asset or the business developing it. It is therefore crucial for advanced therapy businesses at all stages to ensure that their assets have the best possible IP protection to attract the investment and collaboration that is required for success.
Launch
Given the central role played by IP in cell and gene therapy business models, many businesses in the sector will be launched with an existing portfolio of background IP. This background IP may, for example, protect a platform technology or a biological target that the business is proposing to use.
Although at this “launch stage” the asset may not be in its final form, or indeed in any tangible form, the background IP defines the exclusive technical space in which the business intends to operate. Because early-stage businesses are unlikely to have the resource to hire in-house legal counsel, managing this background IP effectively can be a challenge. However, systems and plans need to be in place from the outset to control costs and ensure the management of the background IP is aligned with the objectives of the business.
This may also be a convenient time to carry out some due diligence to check that all the paperwork relating to the background IP is in place and there are no awkward problems that might be unearthed later by a potential investor or competitor who comes to review the IP.
Growth
After launch, advanced therapeutic businesses typically use the technology defined by the background IP to develop their asset to a stage where it can be tested in the clinic. At this “growth stage”, the business may file further patent applications to protect different aspects of the cellular or gene product as it is developed. For example, improvements in the vector or delivery vehicle of a gene product or the antigen receptors, co-expressed factors or cell genotype of a cellular product may be protected by further patent applications that strengthen the IP around the asset.
Keeping track of the expanding IP portfolio and deciding what to protect and how, whilst at the same time controlling costs, may be a considerable challenge. Patent landscaping at this stage may be helpful for an advanced therapeutic business in identifying which potential aspects of a therapeutic product are most likely to be free of competitor IP and therefore most promising to take forwards.
Expansion
Promising cellular or gene products that are identified in the initial development phase are likely to require further development to generate a potential therapeutic that is ready for clinical trials. Potential therapeutics and their methods of production are the key assets on which the value of the advanced therapeutic business is likely to be based.
In particular, the development of cellular therapeutics typically has a heavy emphasis on the development and optimisation of production processes. Modification and optimisation of the production process can have a significant effect on the cells that are produced. As they impact on the commercial prospects of the cellular therapeutic, these process modifications are often the subject of further patent applications and provide another layer of protection around the cellular therapeutic. In some cases, particularly where the cellular therapeutic is very similar to a natural cell, patents directed to production processes may be the only layer of protection around the product.
Effective targeting and stable expression in the correct cell type are important in the development of gene therapies, as well as the identification of doses, administration routes and co-treatment regimens that minimise host immune responses. The ability to produce the gene therapy consistently at scale may also be a significant factor in preparing for clinical trials. Patent applications covering developments in any of these areas provide additional protection around the gene therapeutic.
Careful planning may be needed to maximise the value of the IP portfolio protecting a cellular or gene therapeutic. Any IP that does not provide coverage for the therapeutic in its commercial form might be out licensed or dropped altogether. For example, there may be no need to maintain IP around delivery vehicles or production processes that have been superseded or dropped during optimisation.
Freedom to operate exercises will also be important in establishing that none of the features of a cellular or gene therapeutic infringe third party IP, ensuring that the business will be able to fully exploit their asset and maximise its value. Exploring and investigating the possible uses and applications of a therapeutic asset may require collaborations with academic institutions, pharmaceutical companies and/or other life sciences businesses. Legal agreements will be needed to support these collaborations. Where any barriers to the exploitation of the IP are identified, work will be required to assess the risk to the business, and work will be required to address that risk. Careful planning and well-judged legal agreements may be needed to steer the business around these barriers.
Maturity
As the therapeutic asset enters preclinical and clinical testing there may be opportunities to add further IP to the protection around the key asset. For example, new formulations, combination therapies with other drugs, production methods, administration schedules, disease indications, biomarkers, or patient groups relating to the therapeutic may be the subject of further patent applications . IP filed at this stage may extend the period of exclusivity beyond the expiry of the initial patent filings and so adds particular value to the IP portfolio, and by extension to the business also. At this mature stage, many cell or gene therapy businesses will be planning their exit. Regardless of how this exit is achieved, it is likely to require the IP portfolio to be due diligence-ready and a host of legal agreements to be put in place.
Lifecycle stage is key
The different stages of the development lifecycle of advanced therapy businesses therefore pose different IP challenges and opportunities. Given the importance of IP in extracting value in the cell and gene therapy space, successfully meeting the IP challenges of each different stage is crucial in achieving the commercial objectives of any business in this field.
Mewburn Ellis works extensively with cell and gene therapy businesses in all stages of their development, from foundation and spin-out to growth and later acquisition. We have also worked with investors and acquisitive life sciences businesses, giving us a key insight into their demands and expectations of early and developing businesses in the sector. We have unrivalled experience in guiding companies through the four stages of business development, providing pragmatic and timely advice throughout the business evolution. Working with us, you can be sure of Forward Looking IP to protect your cell and gene therapy innovations.
To learn more about how we work with cell and gene therapy businesses, visit our advanced therapeutics spotlight page. You can also download our IP Guide for SMEs.
