Post-Clinical Trial Commercialization for a Gene Therapy

Over the years, the rate of applications for investigational new drugs (INDs) for gene therapies has been increasing. As of Q1 2025, there were 33 gene therapies approved for clinical use for diseases¹ ranging from diseases such as cancer to hemoglobinopathies and genetic eye diseases.¹

Through the challenges and failures, gene therapy development has evolved and is becoming a potential treatment option for several diseases, such as hemoglobinopathies, genetic eye diseases, HIV, cancers, and more.²

We chatted with Samira Shore, who served as the Director of Program Design at Thermo Fisher Scientific, about the path to commercialization of a gene therapy following successful clinical trials. Read on for the full interview.

Hi Samira! Thank you for chatting with us. Please introduce yourself and your role at Thermo Fisher Scientific.

As the Director of the Technical Program Design team, I have the privilege of leading an exceptional team while supporting our customers in their search for a CDMO partner to develop and manufacture their cell and gene therapy products. Our customers follow a specific process and criteria when evaluating potential CDMO partners, and it is our responsibility to provide them with comprehensive knowledge of Thermo Fisher Scientific’s development and manufacturing capabilities.

We respond to requests for proposals, offer technical insights based on our extensive experience and expertise, inform customers about our offerings across our network, and consult on their specific needs. This role is incredibly rewarding, as it allows me to build strong relationships with both internal and external teams, leveraging my expertise and our robust capabilities to assist customers in delivering critical therapies to patients.

In your words, what is gene therapy and why is it important for the future of science and healthcare?
Gene therapy can be used to treat a variety of diseases, including genetic disorders, some types of cancer, and certain viral infections. The therapy can be delivered using different methods such as viral vectors—which are engineered viruses that can deliver the therapeutic gene to the patient’s cells—or non-viral methods like direct injection of nucleic acids.

The ultimate goal of gene therapy is to correct the underlying genetic problem to provide a long-term and potentially curative solution.

Gene therapy has changed a lot in recent years. How has the COVID vaccine and therapeutics development influenced the way we develop and commercialize drugs?
The most significant advancement in gene therapy in recent years, whether driven by the pandemic or other factors, has been the heightened emphasis on accelerating the delivery of therapies to patients while ensuring product quality and patient safety remain paramount. Substantial progress has been made in refining the processes used to manufacture gene therapies, enhancing productivity, scalability, and potency. Regulatory agencies have also made considerable efforts to provide more guidance to innovators, facilitating faster approvals.

I am particularly excited about the remarkable advancements in analytics and the innovations supporting these critical technologies, which enhance our understanding of product quality, efficacy, and safety. Additionally, there has been an inevitable shift in our customers’ business strategies, likely influenced by new scientific knowledge and changes in financial behaviors driven by industry trends and external factors.

What are some benefits and limitations of gene therapy?
The evident advantages of gene therapies lie in their potential to deliver transformative outcomes for patients suffering from rare conditions that currently lack effective treatments or require interventions that significantly impair their quality of life. The advancements in gene therapy have shifted our focus from merely managing disease symptoms to pursuing actual cures.

However, a major limitation of gene therapy is the substantial cost associated with its development and production. Despite ongoing progress, significant efforts are still required to make these therapies more affordable for both innovators and patients. As most gene therapies are still in the early stages of research and clinical trials, there is a continuous learning process to translate promising R&D results into safe and effective patient treatments. This evolution is supported by leveraging existing knowledge from other industries to mitigate risks in both manufacturing and clinical applications.

Once a company has received approval for their therapy, how should they prepare for commercialization and production?
It is crucial to maintain focus on the ultimate goal throughout the product lifecycle. This demands incorporating commercialization strategies early, even during the initial discovery phases. While adopting a phase-appropriate approach is both acceptable and necessary, implementing tools such as Quality by Design from the beginning ensures that all aspects of the product lifecycle are aligned with the appropriate objectives, thereby guaranteeing the product’s readiness for commercialization when required.

Commercial readiness includes the critical raw materials, manufacturing process, analytics, final product logistics, and a strong regulatory strategy. All of these require the right level of expertise and capabilities to support successful product commercialization.

How do you support gene therapy developers through clinical manufacturing?
Our services encompass comprehensive support for both early and late-phase drug substance and drug product clinical manufacturing. Recognizing the distinct strategies required for products transitioning from early discovery to first-in-human stages, we offer tailored solutions to meet these specific objectives. Our support includes candidate screening, research-grade feasibility studies, process and analytical development, toxicology material generation, product characterization, critical starting material evaluation, product stability, and early-phase cGMP drug substance and product manufacturing and release.

In addition to customized solutions, we provide accelerated options by leveraging our extensive expertise, tested GMP-ready processes, and advanced analytics to ensure cost-effective and expedited timelines to the clinic. For later-phase clinical stages, we extend our services to include process characterization, assay validation, scale-up, regulatory support, and comprehensive drug product clinical services.

What are some of the common roadblocks that gene therapy researchers encounter during commercialization?
From a technical standpoint, these challenges encompass a lack of process robustness, which is intrinsically linked to the development strategy employed. Therefore, the application of Quality by Design (QbD) from the beginning is crucial. QbD facilitates a meticulously planned approach throughout the entire product lifecycle, encompassing all aspects of manufacturing a high-quality product.

This comprehensive strategy not only ensures the successful development of the concept but also supports scale-up, commercialization, regulatory approval, and ultimately, successful disease treatment.

Some common examples include high variability in manufacturing process, lack of regulatory experience or strategy, complex procurement approach (vendor management), and robust supply chain.

What type of solutions help overcome these challenges?
There are several solutions to help overcome these challenges. The majority of challenges can be mitigated with key planning, including:

Leveraging standardized and tested platforms to enhance manufacturing reproducibility
Creating a strong development plan with a focus on commercialization from the beginning (scale up, clinical strategy, regulatory, etc.) that includes:
- An experienced CMC team and manufacturing partner
- Current and innovative technologies
- A strong foundational knowledge of target product quality profile
- Continuing to re-visit and adjust
Establishing a regulatory strategy that allows for flexibility without sacrificing quality and risk to commercialization, including:
- Phase-appropriate requirements, like critical starting and raw materials
- Early and frequent engagement with regulatory agencies
- A good understanding of global regulatory requirements (clinical trials in multiple countries, etc.)
- A plan in mind for potential change
Building strong partnerships with vendors that have a broad range of capabilities from starting material through clinical/commercial delivery to strengthen your plan and prep you for scalability and supply chain management, including:
- Products
- CDMO/CRO services
- Drug product storage and shipping logistics
- Regulatory inspection history
- Clinical support and more

How would you help a customer decide which aspects of gene therapy production to outsource, and what factors should they consider when choosing a CDMO?
While outsourcing multiple aspects of gene therapy production can help support a smooth commercialization phase, it’s helpful to identify key aspects that would be most beneficial. I would urge customers to consider a variety of general factors, including your business strategy and resources (different for a small biotech vs large pharma), short- and long-term needs, process complexity, and product phase.

Key considerations when choosing a CDMO include:

Flexibility and cost
- Is the cost of investing in a CDMO more beneficial than leveraging existing infrastructure and expertise?
- Does the CDMO have broad capabilities, and can they react to process changes?
Product lifecycle
- Are you in the early phase with a focus on clinical development and trials vs. manufacturing and leveraging tested processes?
- Are you in the commercial phase and want to have more control on manufacturing timeline and process?
Expertise
- Does leveraging CDMO expertise vs. investing in building a team with required expertise support your goals?
- Is the cost of investing in building a team with set expertise worth it when you can leverage the capabilities of an experienced CDMO?
Timeline and capacity
- Are you able to react faster with internal capabilities, and can you manage capacity?

What new opportunities do you see emerging in the gene therapy field when it comes to commercialization and production, and how is Thermo Fisher Scientific preparing to leverage them?
There is a need for better characterization of these complex gene therapy products. The best way to do this is by leveraging next generation analytical tools to not only better understand product characteristics, but also establish a better regulatory strategy.

This will ultimately ensure the utmost patient safety, especially when moving products from early clinical stages to commercialization. While there are higher requirements during the early clinical stages, this is when urgency is paramount. It’s important at this stage to establish extensive product knowledge and use multiple analytical methods for evaluation of viral vector attributes such as safety, purity, identity, potency, and stability.

New opportunities that I can foresee include:

Even more importance on having experienced analysts: This includes working with experienced providers and keeping analytics on the forefront.
New delivery methods: Another trend in improving the efficiency of these viral vectors involves the development of custom engineered capsids with the aim to improve delivery of the therapeutic product to the target tissues.

Thermo Fisher Scientific is involved in these emerging trends through the development of analytical instruments that drive these enhanced viral vector characterization efforts as well as by providing the appropriate analytical capabilities through our CRO services to support the development and manufacturing of viral vectors.

What are you most looking forward to with this technology?
I am very excited about the recent advancements in the gene therapy space, particularly in the areas of manufacturing optimization and scalability. The development of new critical reagents has significantly enhanced the productivity and quality of viral vectors, leading to more cost-efficient manufacturing processes.

This progress expands the potential application of gene therapy to treat diseases affecting larger patient populations. Furthermore, the emergence and continuous evolution of advanced analytical technologies are improving our ability to learn more and better understand key product quality attributes, ultimately increasing success of clinical trials and potential for commercialization.

The increased engagement and support from regulatory agencies for drug sponsors and manufacturers is a strong indicator of progress in the gene therapy space. This collaboration is expediting drug approvals, especially for rare diseases and conditions with no available alternative treatments.

What does gene therapy development and the solutions that utilize it mean for the future of health?
To put in most simple terms, gene therapy is changing lives. The idea of using gene therapy to provide curative treatments for these complex diseases is fascinating and exciting.

We have a lot more work to do from the manufacturing standpoint as we need to find ways to make these therapies safer, more effective, and affordable, but I am confident that with the continued focus on the scientific innovation, we can achieve that.

There is a lot of potential to further expand the market of gene therapy as we continue to learn more about how this technology can be applied to more common diseases.

Anything else you’d like to touch on?
Ultimately, the driving force behind the passion within the gene therapy community is its life-saving potential. We are deeply committed to making a meaningful impact on the world, and advancing gene therapy is a powerful way to achieve that goal.

We are excited to offer you our comprehensive eBook, packed with valuable insights and solutions your peers are utilizing to drive gene therapy from discovery to commercialization.

Download your copy of the gene therapy eBook.