What are iPSCs? And what is their potential?

What are iPSCs? And what is their potential?

What are iPSCs? And what is their potential?

The concept of reverting a cell to its most primitive and adaptable state is no longer a mere figment of imagination. This is the reality of induced pluripotent stem cells (iPSCs), which have the potential to morph into nearly any cell type in the human body. But how can this be harnessed for the betterment of patient care?

Stem Cell Spectrum

Stem cells are foundational cells with the unique ability to develop into various cell types in the body. They are primarily divided into two main categories: embryonic stem cells and adult stem cells.

Embryonic stem cells are extracted from the inner cell mass of embryos. These cells are pluripotent, which means they can transform into more than 200 different cell types in the body. Their versatility makes them a kind of “blank slate.” This inherent plasticity, self-renewal and differentiation potentials has made them a significant focus of scientific research.

What are induced Pluripotent Stem Cells (iPSCs)?

Introducing induced pluripotent stem cells (iPSCs) added a new dimension to the stem cell spectrum. iPSCs are generated from somatic cells, like skin cells, that have undergone genetic reprogramming to revert to a pluripotent state akin to embryonic stem cells. Due to their adult somatic origin, iPSCs have sidestepped many of the ethical concerns tied to embryonic stem cell research.

In summary, iPSCs emerged as a game-changer in stem cell research. By harnessing the potential of pluripotent cells and merging it with the patient-specific origin, they offer immense promise for therapeutic applications, disease modelling, and further scientific exploration.

Cellular Time Machines

The term “cellular time machines” aptly describes induced pluripotent stem cells (iPSCs). At their core, iPSCs are adult cells that have undergone a transformative process, allowing them to revert to an embryonic-like state. This reprogramming grants them the flexibility and adaptability characteristic of embryonic cells.

To understand this analogy, consider the life of a cell. As cells mature, they differentiate and become specialized, losing their original pluripotency. However, with iPSCs, scientists have found a way to reverse this process. It’s akin to turning back the hands of time on a cell’s life, restoring its youthful potential and versatility. This remarkable ability to revert mature cells to their primitive state is why iPSCs are often likened to “time machines” in the cellular world.

Yamanaka Factors: The Cellular Catalysts

In 2006, a pivotal shift occurred in stem cell research. Shinya Yamanaka and his team introduced the world to induced pluripotent stem cells (iPSCs). This discovery was not just another scientific finding; it represented a monumental leap, bridging the gap between the theoretical potential of stem cells and their tangible applications in medical science.

Central to creating iPSCs are the Yamanaka factors: Oct4, Sox2, Klf4, and c-Myc. These factors play a crucial role in the reprogramming process. When introduced into adult cells, they reactivate the dormant pluripotency genes, effectively rejuvenating the cell and reverting it to an embryonic-like state. It’s as if these factors hold the “keys” to unlocking the latent potential within cells, enabling them to regain their youthful flexibility and adaptability. This process has opened up new avenues in stem cell research, offering a more ethical and accessible way to harness the power of pluripotent cells.

Transformative Role of iPSCs in Regenerative Medicine

Induced pluripotent stem cells (iPSCs) are at the forefront of advancements in regenerative medicine, heralding a new era of therapeutic possibilities. Their inherent versatility is showcased in multiple ways.

For instance, they can potentially repair damaged tissues, such as cardiac tissues after a heart attack or neural tissues following spinal cord injuries*. This ability to regenerate and restore function to injured areas offers hope to many patients with limited treatment options.

Furthermore, the potential of iPSCs extends beyond tissue repair. Scientists are exploring their capacity to grow entire organs for transplantation*. Imagine a future where patients no longer have to wait on extensive donor lists for a life-saving organ. Instead, they could receive a custom-grown organ derived from their cells, diminishing the risks of organ rejection.

The adaptability of iPSCs is genuinely unparalleled. For instance, in neurodegenerative diseases like Parkinson’s, researchers are investigating how iPSCs can be used to replace damaged dopamine-producing neurons*. Similarly, in the field of diabetes research, there’s an ongoing exploration into how iPSCs can be transformed into insulin-producing beta cells, potentially offering a more definitive solution for diabetes patients*.

As the landscape of regenerative therapies continues to evolve, iPSCs stand out as a cornerstone. Their diverse applications, from tissue repair to organ generation, make them invaluable. With each research breakthrough, the promise of iPSCs broadens, bringing us closer to medical solutions that once seemed beyond our reach. As we delve deeper into the capabilities of these cells, the future of medicine looks brighter, offering hope for transformative treatments and cures.

Advancing Disease Modelling

Beyond their role in regenerative medicine, induced pluripotent stem cells (iPSCs) are pivotal in disease modelling and drug discovery. Generating patient-specific cells allows researchers to gain a more profound understanding of various diseases at a cellular level. This personalized approach provides a unique window into the intricacies of disease mechanisms, facilitating the identification of potential treatment pathways. As a result, iPSCs are accelerating the discovery of effective treatments and paving the way for potentially revolutionary cures in the medical landscape.

Ethical Advantages

Stem cell research, particularly involving embryonic stem cells, has long been at the centre of ethical debates. However, induced pluripotent stem cells (iPSCs) offer a distinct advantage in this contentious arena. Derived from adult cells, iPSCs eliminate the need to use embryos, sidestepping the primary ethical concerns that have overshadowed embryonic stem cell research. As a result, iPSCs present a more universally accepted approach, providing the scientific community with a powerful tool that aligns more harmoniously with ethical considerations.

The Future of iPSCs

Looking forward, the prospects for induced pluripotent stem cells (iPSCs) are incredibly promising. These cells hold the potential to drive unprecedented medical advancements, reshaping the landscape of healthcare. As research continues to delve deeper into the capabilities of iPSCs, we are on the cusp of transformative breakthroughs that could herald a new era in medical treatments and interventions. The journey of exploring iPSCs leads us towards a profound evolution in healthcare, with possibilities that were once deemed the stuff of science fiction now within reach.

Webinar: How to develop your Cell Therapy manufacturing process

Webinar: How to develop your Cell Therapy manufacturing process

Webinar: How to develop your Cell Therapy manufacturing process

Webinar: How to develop your Cell Therapy manufacturing process

How do you translate your Cell Therapy processes to GMP? What are the challenges of bringing an iPSC-based therapy to the patient? How to set up a CAR-T manufacturing process?

On Wednesday 14 December we will explore the challenges one may face in establishing a Cell Therapy process for application in clinical trials. During this webinar, Melissa van Pel, head of Cell Therapy at NecstGen, will discuss two exemplary cell therapy types:

1) Induced pluripotent stem cell therapy (iPSC), a relatively new type of Cell Therapy that is regarded as one of the most promising solutions in regenerative medicine.

2) Chimeric antigen receptor T-cell therapy (CAR-T), a well established type of Cell Therapy that has faced commercial and clinical success, as well as logistical challenges as it matures.

We look forward to welcoming you to this online seminar.


  • 16:00hr – Welcome – Tristan Pritchard-Meaker (Head of Business Development, NecstGen)
  • 16:05hr – How to set up your Cell Therapy process – Melissa van Pel (Head of Cell Therapy, NecstGen)
  • 16:40hr – Interactive Q&A session

Date and time

  • Wednesday, December 14, 2022, 4:00 PM – 4:45 PM CET


  • Online 
European Cell and Gene Therapy companies

European Cell and Gene Therapy companies

European Cell & Gene Therapy Companies

European Landscape For Cell & Gene Therapy companies

At NecstGen, we aim to support industry partnerships and growth. And to enable the next generation of therapies, it is pivotal to have oversight of the gene and therapy landscape to start from, which is why we created this helpful tool. Feel free to use and share it, so together, we challenge today’s possibilities and enable the unthinkable.


Did we miss your organisation? 

The biotechnology business is booming around the globe. Increased private equity investments in biotech, global cross continent acquisition deals, and IPOs have risen to record levels. This boom in development will enable the global biotech industry to surpass its older sibling, the pharmaceutical industry, in interest. One area of biotech that holds a particularly great promise to meet patients’ unmet needs is Cell and Gene Therapy. Such therapies defined as ATMPs (Advanced Therapy Medicinal Products) have a substantial therapeutic potential to treat the patients that current treatments may fail. Although their development can be complex, this is not holding back drug developers, innovation continues in leaps and bounds. But where are most Cell and Gene Therapy developers located? This article and map share insights into the ATMP landscape in Europe and the existence of several hubs

Cell & Gene Therapy Companies in the United Kingdom

With three hotspots in the UK, the Britons lead as Europe’s biotech hub for breakthrough life-science start-ups. McKinsey wrote a fascinating report covering how this hub has matured relative to its peers and what lies ahead on its road to playing a leading role in the Cell and Gene Therapy sector globally.

The report based its results on a Biotech Innovation Index, which assessed the biotech sector on discovery, translation, growth capital, and various impact indicators. Although they do not directly target ATMPs in the study, the large hubs you can see on the map here definitely confirm their findings.

In the UK, most Cell and Gene Therapy companies are located in one of these three clusters:

Cell & Gene Therapy Companies in the Netherlands

The Netherlands has several biotech hubs, all connected through a strong infrastructure network despite its size. Its central location in Europe makes it no surprise that EMA relocated its headquarters to Amsterdam following Brexit in 2020. The Netherlands is also home to several knowledge centres with extensive capacity in Cell and Gene Therapies

Investments into the Leiden University Medical Center (LUMC) have accelerated patient access to innovative Cell & Gene Therapies, including stem-cell therapies. NecstGen is a fruit of those investments and supports organizations worldwide to develop novel therapies for patients. In addition, Utrecht is home to the Utrecht Cell Therapy Facility: a hub for ATMP development specialised in Cell and Tissue based therapies.

In the Netherland, most Cell and Gene Therapy companies are located in these clusters:

Cell & Gene Therapy Companies in Switzerland

Switzerland is known for being the home country of pharmaceutical giants Roche and Novartis and many other leading biotech companies. Together, the combined sector contributes over 40% of Swiss exports. Over 300 biotech start-ups are located in the country and specialise in diverse fields such as oncology, antibodies, and orthopaedics. It’s not a surprise that multiple Swiss companies have already worked on advancing their presence in Cell & Gene Therapy.

Novartis opened its Cell and Gene Therapy facility back in 2019, for manufacturing Kymriah. Last month, Cytiva opened its new manufacturing facility in Grens (Link) and served as the base of operations regarding their Cell and Gene Therapy-related operations and a training centre for European customers.

Swiss Biotech companies are located around the following three clusters:

Cell & Gene Therapy Companies in Italy

The Italian biotechnological sector is concentrated around the northern parts of the country. The North-Western part of Italy is where companies specialising in drug development and new therapeutic approaches appear to be located, including for Cell & Gene Therapies.

Italy’s ATMP-specific service provider market ranks third  in Europe. Therefore, it may not also surprise that some of the earliest advanced therapies and four out of seventeen authorised for the European market originate from Italian academic research. Nature published an article on the challenging yet promising future of ATMP development in Italy.

In Italy, most biotech companies specialised in Cell and Gene Therapy are located in Milan and surroundings.

Cell & Gene Therapy Companies in Germany

Even though there is no clear hotspot in which Cell and Gene Therapy-focussed companies are settled in Germany, the sector has seen immense growth. This builds on the underlying strength of Germany in R&D and manufacturing  in Pharma. The country is home to 660 biotech companies with a total of 50,000 employees. Of those 660 companies, many are focused on Cell & Gene Therapy.

In 2021, more than 29 active clinical trials evaluating CAR-modified immune cells took place, most of which involved CAR-T cells. In Germany, over 50 clinical studies have been conducted in Gene Therapy, making Germany a country heavily involved in the development of Cell & Gene Therapies. Nature published an interesting report on the past and future of Gene Therapy in Germany.

The German Cell and Gene Therapy companies are not clustered, but are located all throughout the country, demonstrating the broad strength of innovation locally.

Cell & Gene Therapy Companies in France

Named as one of the best three biotech centres in Europe by McKinsey, France is home to 720 biotech companies with a combined workforce of over 50,000 employees.France has a thriving academic environment, which can be seen from the number of biotech firms backed by academic research, which was forty-six percent iin 2017.

It may come as no surprise that France is one of the leading countries in Cell and Gene Therapy research. The French government and biotech companies invested in Gene Therapy development early on, and it paid off. More than 10-world leading biotech companies and research groups operate from France, such as Genethon. While most companies and institutes involved in Cell and Gene Therapy development, such as Institut Imagine or Institut Vision are located in Paris and its surroundings, there are several other biotech hubs in France.

Cell & Gene Therapy Companies in Sweden

Sweden definitely has the ambition to rank globally as a Cell and Gene Therapies player. They share the aspiration of the countries mentioned above to provide patients with innovative treatments and have even started a program specifically tailored to ATMPs, Vinnova. By following this programme, Sweden aims to be a leader in advanced therapies by 2030!

The complete program involves partners, including research companies, universities, the Swedish Medical Products Agency, patient representatives, and the pharmaceutical industry’s trade association. Therefore, we can consider their combined perspectives and goals the driving force behind the accelerated, developing landscape ATMPs.

We can find a hotspot of ATMP companies located around the Ideon Science Park in Lund

Cell & Gene Therapy Companies in Belgium

Finally, we mention Belgium on our list of hotspots for Cell and Gene Therapy developers. The Belgium biotech industry is thriving, with more than 140 operating biotechs despite its small size. Known for its vivid biotech landscape, Belgium is also developing their share in ATMP development.

Compared to surrounding countries, large companies always favoured Belgium as a country for their manufacturing operations. Shortly, UCB plans to open up a new gene therapy facility in Braine-l’Alleud, and we can expect it to be operational in 2024.


Related Questions

Which Cell Therapies are approved?

In these figures, we gathered and visualized overviews of approved ATMPs over the past years for you.

What does the Cell Therapy Development process look like?

From idea to treatment, you’ll face changing requirement and development challenges. View the figure to see how knowledge of the process inversely relates to freedom to make changes to your process.  

Our experts are only a message away to help you understand the impact of any of these aspects and make informed decisions on outsourcing.

We’d be happy to discuss and help you bring cell therapies to patients.

Outsourcing Cell & Gene Therapy Manufacturing

Outsourcing Cell & Gene Therapy Manufacturing

Outsourcing Cell Therapy Manufacturing

Questions to ask yourself

Cell and Gene therapies include extracting cells or genetic material (DNA) from a patient, altering that material to develop a highly personalized therapy and subsequently injecting it back into the patient. Therapies like these are based on state-of-the-art technology and can require specific, hard-to-find expertise to manufacture. While the entire manufacturing costs are crucial, you also want to ensure a rapid path to market and sufficient flexibility if your plans change.

If you need to produce a Cell or Gene Therapy, there is an ever-growing pool of contract manufacturers offering a range of services and expertise. Due to a scarcity of adequate manufacturing facilities and expert employees, the Cell Therapy sector’s reliance on outsourcing is a constant source of concern.

By outsourcing, you get access to a specialized company’s internal resources, including both its equipment and in-house experts. And, by working with a professional CDMO, you can usually scale up faster.

Here are the questions we recommend asking yourself when deciding if you should outsource your Cell Therapy manufacturing.

Where do you need help?

Outsourcing has become increasingly popular among biopharmaceutical businesses, while others have decided to build in-house production facilities instead. So how did others decide?

It is best to think carefully about what you will outsource. As the need for Cell and Gene development services grows, CDMOs constantly examine and evolve their portfolios and what they can provide to their new Cell Therapy customers. Drug developers are increasingly evaluating the different advantages of outsourcing, including increases in productivity, efficiency gains, quality improvements, and many other aspects that will help them shorten their time-to-market.

Therefore, your analysis should be elaborate and well-thought-out, so you make the right decisions in weighing outsourcing against manufacturing your product in-house. Aspects to consider include:


  • Process and analytical development
  • CMC
  • Quality Control
  • Quality Assurance
  • Manufacturing
  • Training
  • Cleanroom rental
  • De-risking capital investment

What is in your pipeline?

A first step toward determining if you want to outsource your Cell Therapy manufacturing is to take a step back and evaluate the number of products in your pipeline. Simply said, that means that smaller biotechs working on a limited number of single Cell Therapy products might be wise to choose a different route than large, established Cell Therapy developers with many candidates in their pipeline. 

Additionally, you will have to consider the kind of therapy you are developing. For example, is your product Allogeneic or Autologous? Fresh or frozen? And what about the administration route? Does your product incorporate a medical device? The characteristics that define your therapy cannot be left out of the equation. They represent an important aspect in deciding if you want to outsource your Cell Therapy production and to which CDMO.

Key here is your product classified as an Advanced Therapy Medicinal Product (ATMP)?

Technology: Two-Sided Sword

Although the lack of certain services or technologies could drive Cell Therapy developers towards an in-house manufacturing facility, there is another option. Some CDMOs offer you the option to invest in the required equipment but can be accommodated at the manufacturing site and subsequently used for your therapy.

To no one’s surprise, Cell Therapy manufacturers should offer modern facilities, including state of the art technology. However, technology is a two-sided sword when choosing to insource or outsource your Cell Therapy production.

Making use of existing technology for Cell Therapy manufacturing

The main benefit of outsourcing is that there is no need for a significant upfront capital investment, and you can access expertise immediately, if no queue exists. Although the waiting time to access contract manufacturing services can be significant, the bandwidth and expertise of a Cell Therapy CMO might mean accelerated delivery times instead of building, commissioning and then manufacturing in your facility. In addition, outsourcing also allows you to manage your cash flow more steadily this way.

Adoption of new Cell Therapy Manufacturing technology

You might want to employ novel technologies to ensure a state-of-the-art manufacturing process. Sometimes it is easier and faster to integrate these yourself in-house than expect a CDMO to prioritize adopting new technology. It is a misconception that because of pressure on CDMOs who currently manage the competing demands, they can’t cater to tailored needs for cell therapy production. 

If swift adoption of your technologies is essential to your plans, you can choose to do so through in-house capabilities, but you can quickly check whether it’s possible at your CDMO too.

The path in between

Although the lack of certain services or technologies could drive Cell Therapy developers towards an in-house manufacturing facility, there is another option. Some CDMOs offer you the option to invest in the required equipment but can be accommodated at the manufacturing site and subsequently used for your therapy. Or rent cleanrooms?


What are the costs?

Comparing the costs of building in-house capacity versus sourcing capacity externally for the long term is essential. It would be even better to consider multiple scenarios that take success rates for the processes you are developing into account. And in every budget estimation, you should include unexpected expenditures during the project and throughout its lifetime.

If you’re interested in building an in-house facility, these will be major costs to examine:

  • Maintenance
  • Utilities
  • Labour
  • Consumable costs

If you plan on outsourcing your cell manufacturing, these are major costs to examine:


  • Operational
  • Pass-through
  • Tech transfer
  • Consumable costs.

    What kind of expertise do you need?

    Core team for your Cell Therapy manufacturing program

    Sometimes, onboarding experts might be strategically advantageous, mainly if you are operating in a highly competitive part of the market. It could allow you to build in-house expertise for your core strengths and expand your team from there on if needed; based on the complexity of your product. Some CDMO, like NecstGen, can also assist you in-house and can train your team. 

    Especially Quality Assurance/Control and GMP manufacturing processionals are essential, and if budget restrictions prevent you from hiring a complete team, consultants might be worthwhile to investigate.

    Multidisciplinary Cell Therapy development

    Suppose you require various competencies and resources, which is likely in a highly innovative environment such as the Cell and Gene Therapy field. Then, outsourcing is an excellent plan. It allows you to take advantage of your partner’s expertise on short notice, which can be an essential success factor in bringing cell therapies to patients.

    Keep in mind that with a CDMO, you’re engaging with an entire organization specialized in manufacturing cell therapies.

    Quality in Cell Therapy Manufacturing

    Most Cell Therapies are initially developed in academic institutions. While this environment excels at producing new leads for future therapies, the lack of experience with GMP infers a rather substantial challenge for a scale-up. Therefore, it is essential to invest early in manufacturing and supply capabilities, including Quality Lifecycle Management and Quality By Design principles.

    You can decrease the notable risks of modifying your manufacturing process in the subsequent development phase by taking these principles into account. The same holds for integrating sound CMC processes early on in your clinical operations. That way, you ensure rapid, uninterrupted product development.

    It is clear that not living up GMP-compliant process puts everything at risk, both the stakeholders and the development of your therapy itself. If you cannot gather an in-house team of seasoned (ATMP) quality experts around you, it might be wise to consider contacting a CDMO.

    Availability & Capacity

    In the longer term, a key consideration is whether a CDMO has the flexibility and capacity to increase (or decrease) manufacturing volumes on demand. So, besides the question, if a CDMO can accommodate your manufacturing at all, limited capacity or flexibility would mean you have to plan and book far in advance.

    You need to ensure that your CDMO can adapt to the maximum required volumes, even though that might seem far away from an early-stage perspective. 

    Of course, it’s not always black and white. For example, as a developer, you can’t always establish a complete set of requirements for an in-house facility if your final scalability needs aren’t precise yet. As a result, many companies go about this decision opportunistic and use outsourcing as an in-between-manufacturing-sites until they fully understand the complete picture.

    How do you ensure Tech Transfer?

    Your technology will transfer from academics to commercialization from smaller labs to larger facilities along with comprehensive process knowledge to reproduce your Cell Therapy at the required quality and scale and in line with regulatory requirements. Therefore, you should ensure a detailed and robust process description, including critical quality attributes (CQAs) and critical process parameters (CPPs).

    If you’re optimistic about keeping manufacturing in-house, there is no need to worry about tech transfer, licensing fees, or concerns about intellectual property rights. Everything is kept in-house.

    Keep in mind that, per definition, most Cell Therapy products are highly specialized and require tailored utilities and experienced, well-trained staff. Not all CDMOs are thoroughly prepped for this particular challenge. As mentioned before, a CDMO can experience difficulties accommodating novel technologies resulting in delayed tech transfer. Our recommendation would be to go over entire the tech transfer process with your CDMO candidates to ensure you’re making the right choice if you want to outsource.

    Are the operators of the CDMO undertaking the development or manufacturing involved in the tech transfer? This can streamline and improve overall success.

    When is the right time to start a conversation?

    When would be an excellent time to speak to a possible candidate for outsourcing your Cell Therapy manufacturing? We recommend when you are transitioning from the fundamental to the pre-clinical stage. CDMOs are also used to make preclinical material, and this will likely be a better outcome.

    Many Cell Therapy biotechs choose to outsource, as it is crucial for your product development.  Although it is best not to rush into commitments, the earlier you start a conversation the better. As long as you thoroughly inventory your needs and analyze potential partners’ capabilities and in-house quality and expertise.

    So, to sum up, all the items you should investigate if outsourcing is a good fit for you, you should look into your needs regarding:


    • Type of Cell Therapy
    • Budget estimations
    • Technology requirements
    • Regulatory and CMC expertise to upscale 
    • Quality / GMP expertise to mitigate risk early on
    • Availability of established process development 
    • Flexibility and scalability of manufacturing
    • Understanding of technology transfer

      Related Questions

      Which Cell Therapies are approved?

      In these figures, we gathered and visualized overviews of approved ATMPs over the past years for you.

      What does the Cell Therapy Development process look like?

      From idea to treatment, you’ll face changing requirement and development challenges. View the figure to see how knowledge of the process inversely relates to freedom to make changes to your process.  

      Our experts are only a message away to help you understand the impact of any of these aspects and make informed decisions on outsourcing.

      We’d be happy to discuss and help you bring cell therapies to patients.

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