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Gene and cell therapies have come a long way, but what is happening next according to stakeholders?

By M. Rebeca Gil Garzón (PhD Student at Royal Holloway University of London, BSGCT Board member as an ECR).

The journey so far…

For any new technology there is always an adoption curve as the field gathers momentum. In the case of gene and cell therapies the journey has been spectacular.

It is an exciting time to be involved in this industry; after years of research many of these technologies are reaching the final stages of clinical trials and, for some, the market. However, there is much that needs to be addressed.

As the industry grew there were different hurdles to overcome at various stages. Firstly, the development of core gene transfer technologies themselves and their subsequent assessments in the context of safety and efficacy. Then, their ethical implications and evaluating if the “potential new treatment” would be better than what was already available to treat a particular disease. Later, production became the next hurdle as clinical grade batches were needed, followed by the need to scale up.

Now we´re at this stage where gene and cell therapy manufacturing sectors are expanding and the industry is facing new obstacles, such as making these treatments cost effective and accessible for patients while dealing with regulations and navigating a complex reimbursement process in key markets, which in Europe requires working with 27 different countries to negotiate a price.

These issues are complex and affect many parties. It is only when stakeholders collaborate that we can tackle them effectively. The field is, once again, at an inflexion point.

Who are the stakeholders?

First, it is important to define who the main stakeholders are in this field and understand how they interact with each other:

· Academia.

In the context of gene and cell therapies we tend to refer to academic research groups working in this field. These groups have been the developers of the therapies and often the core manufacturing technologies. They also train the new generations of gene and cell therapy scientists who are needed to address the skills gap in the industry.

· Government and regulators.

Defining how these therapies should be tested and marketed.

· The NHS and clinicians.

They would potentially deliver these sorts of treatments to the patients.

· Patients and patient advocacy groups.

One of the most important stakeholders in this field, especially when it comes to rare diseases. They are the ones directly affected by the availability of these treatments. Their input on the developmental phase of a treatment and during the clinical trials design is extremely important.

· Payers.

The people who would pay for these therapies varies from country to country. In the UK, the NHS and occasionally private healthcare would cover the costs. In the United Stated, it would be mainly insurance companies and in other countries there would be combinations of private and socialized healthcare.

· Industry.

Industry in this field can be segmented into different groups. There are big pharma companies acquiring and developing these technologies, start-ups focusing on translating these treatments to the market, consultancies with specialist knowledge of cell and gene therapies, manufacturing related companies such as CDMOs (Contract Development & Manufacturing Organizations), CROs (Contract Research Organizations) and technology companies, supporting innovation through capabilities such as automation.

· Investors.

Private investment is critical to provide the liquidity needed to run proof of concept studies and early clinical trials, and often beyond.

· Charities and National Societies.

Such as the British Society for Gene and Cell therapy (disclaimer: the author is a member of this organization). Which is the national society, focused on supporting progress and translation of gene and cell therapies by engaging with all stakeholders and seeking to become the “editorial voice” on the discussions within the field.

It is worth mentioning the importance of building public knowledge and understanding about these therapies. The public can play a key role to advance this sector. At the end of the day, it is the needs of patients within society that is key to pressuring governments and the industry to prioritize certain issues.

What are the stakeholders saying?

As an ever-changing field, it is important to attend gene and cell therapy related events to keep up to date with the industry. On the 31st August and 1st September, Phacilitate ( hosted “Advanced Therapies Europe 2022” in London, an event designed to foster networking and encourage collaborations for the commercial success of gene and cell therapies in Europe.

It was a great opportunity to hear updates from different sectors within gene and cell therapies, such as commercialization, manufacturing & supply chain, patient engagement, clinical trials and investors. The case studies provided valuable insight into new technologies being developed and reaching the market. The panel conversations and Q&A sessions were an important insight into the mindset and views of the different stakeholders.

An issue which raised its head time and time again was the skills gap in gene and cell therapy: the difficulty finding skilled staff to support the booming sector, both in manufacturing, regulatory affairs, and R&D.

It was encouraging to see the number of gene and cell therapy start-ups joining the conversation. The buzz word of the event was “zebra companies” which is an ad hoc term for most gene and cell therapy companies that, unlike the classic “unicorn companies”, tend to not only strive for profitability, but also work to improve society, foster a more collaborative atmosphere, and succeed by working together. This does not mean that there will be no competition, but the difference will be made by the execution and collaborations facilitating translation of gene and cell therapies.

One of the hurdles highlighted for the field was the price of gene and cell therapies reaching the market and how patients are supposed to afford them. Companies will need to come up with creative financing models. We are already seeing a few examples of alternative payment models for approved gene and cell therapies (Figure 1) but there´s still work to be done around this.

Figure 1. Alternative payment models and examples of gene and cell therapies currently being offered in the market on outcome-based payment models. (Adapted from AuWerter et al, 2019 & Loche et al, 2021).

Another highlight was the discussion between venture capital partners regarding the market and potential opportunities for gene and cell therapy start-ups. The main message was to encourage these start-ups to approach funders and to be prepared by knowing their potential market in detail, having a strong technology with a defined differentiator, and having a clear strategy to reach the market. The importance of networking in this field was also highlighted.

So, what happens next?

We will continue to see new gene and cell therapies reach the market. It is unknown how long it will take for industry and regulators to solve some of the current hurdles, but we can hope it will be within the next 10 years.

It is only fair to end this update by saying that there will be ups and downs. Gene and cell therapies are still the future, and importantly, they are now the present of medicine, too.


AuWerter, T., Smith, J., Sternberg, J., & The, L. (2019, August). Unlocking market access for gene therapies in the United States. McKinsey & Company. Retrieved September 15, 2022, from

Loche, A., Paolucci, N., Peters, N., & Veken, L. V. der. (2021, January). A call to action: Opportunities and challenges for cgts in Europe. McKinsey & Company. Retrieved September 10, 2022, from


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