Your Role in Cell & Gene Therapy Ecosystem

The cell and gene therapy (CGT) ecosystem is the result of its components working together to provide CGT service in a specific area.

Developing an ecosystem of any technology is a complex task because it involves multiple sectors and entities. The fact that CGT technology is emerging in the gulf region makes the task of developing a CGT ecosystem both harder and easier at the same time. It makes this task harder because several components that are essential to the CGT ecosystem are either missing or under developed, which means more work to do. But being an emerging technology makes the task of developing a CGT ecosystem easier because (i) there is no established norm yet that need to be reviewed/adjusted with all involved resistance and retraining, and (ii) the missing or under developed ecosystem components can be built or developed in a way that serves their intended purpose.

For the CGT to bear fruit, the CGT ecosystem needs to be fully operational. Here is one approach to ensure that.

CGT ecosystem is composed of multiple components that all need to be fully developed. This is critical because having a suboptimal performing component of the system would lead to a broken system that may not support its functions. Developing each component is a time-consuming process, and need to be carefully planned and timed; I’ll talk about defining and developing CGT ecosystem components separately.

The components of the CGT ecosystem are not independent ones, rather, they are parts of a wider network that impact and complement each other. The CGT ecosystem needs to be structured in a way that makes its components operate as one unit. The goal here is to align the CGT components and make them operate in a cohesive manner. To accomplish this goal, we need the following:

1. Effective communication during the component development phase to define the (what) is required for each objective, and (who) is responsible for each requirement. The benefits of this are to remove redundancies and to bridge gaps within the system.
2. Logical flow of the process. To get the highest efficiency of the ecosystem, the process flow should be designed with all stakeholders in mind. This means the flow should be aligned with timing and with responsibility of each stakeholder. Though there are no front and back ends of the process, rather, it is a perpetual flow with a self-improvement capability.
3. Efficiency-centric. The CGT ecosystem is complex and involve many stakeholders. For this ecosystem to be efficient, it needs to be built around the interests of the involved stakeholders. The main stakeholders of the CGT ecosystem are (i) regulators, (ii) researchers, (iii) industry, and (iv) patients. Considering the needs, the requirements, and the responsibilities of each stakeholder while structuring the ecosystem makes the system efficient. 
4. Bottom-up approach. To correctly build the CGT ecosystem, and to identify its components and its stakeholders, it is best to start with the intended outcomes then move upward to identify what created them. Once the components and stakeholders are identified, we then define how each fits the system (role), and how it serves the intended outcomes (responsibility).

As the example to the right illustrate, as an outcome, Safe Product is the result of many factors, mapping them out upward clarifies the involved components and how they impact each other. By doing this simple exercise, we quickly identified couple components:

• Quality Material and Qualified Vendors, they both point to the Supply Chain component.

• Material Specifications and Investor Requirements, they both point to the Regulations component.

You can still do the same exercise with the Trained Personnel and Approved Process and see what components they lead to.

Different stakeholders take part in the system at different stages, and interact with a set of components that are specific to that stakeholder. For instance, researcher take part in the system fairly early, and would interact with Regulations and Ethics. While patients would take part in the system at later stages, and would interact with the Clinic and the Collection.

Component-component and component-stakeholder relationship is almost always a two-way relationship to provide the feedback loop. Some components may appear to have no relationship with each other, but when carefully examined, they would present an indirect relationship like in the case of patient-regulators.

It is important to mention that ecosystems affect each other as they develop and evolve. For this matter, CGT ecosystem as it develops in the region is expected to impact the related ecosystems such as the healthcare ecosystem and the research & development ecosystem. This ecosystem-ecosystem impact cycle would continue, and these related ecosystems would reach only partial maturation because of their ever-evolving and innovative nature. 

This article is intended to be a thought provoking or a conversation starter (effective communication) towards a comprehensive CGT ecosystem, and is not by any means a proposal of a specific structure. CGT ecosystem development in the gulf region requires a collaborative teamwork. It would be a little while before a comprehensive CGT is realized, but it is a critical goal that is worth working towards.

Let us know if we missed anything, or if you would like a specific subject to be discussed, write to us. 

About the author

Naseem Almezel, earned his MSc degree in Cellular Therapies in 2010, since then his career focus is to support Bone Marrow Transplant and Oncology programs. Naseem likes to work in the lab doing translational research, or in the cleanroom doing GMP production. When he is not working, Naseem likes to read and to spend time outdoors. Find more about Naseem here