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 iPSC Banking & Derivation

Webinar iPSC Banking & Derivation

iPSC Banking & Derivation

Ever wondered how to transition from bench to GMP suitable processes in the iPSC-derived therapies field? Yes? Watch the webinar replay on iPSC Derivation & Banking.

Key areas of focus include:

  • Transitioning from Bench to GMP Suitable Processes
  • Identifying Technologies for Scale-Up and Future Manufacturing
  • New GMP iPSC Generation
  • Master and Working Cell Bank Creation
  • Quality Control Strategies for Safe Products

Our experts can provide insights and answer your queries to advance your therapy development. This webinar is designed to help you explore and navigate the complexities of iPSC therapy development, but if you have additional questions, we invite you to ask us your questions.

NecstGen and ProteoNic Report Development of Improved Viral Vectors Through the Application of Premium 2G UNicTM Technology

NecstGen and ProteoNic Report Development of Improved Viral Vectors Through the Application of Premium 2G UNicTM Technology

NecstGen and ProteoNic Report Development of Improved Viral Vectors Through the Application of Premium 2G UNicTM Technology

Leiden, Netherlands, September 19, 2023 – Necstgen, a CDMO and centre of excellence for Cell and Gene Therapy, and ProteoNic, a leading provider of premium vector technology and services for efficient production of biologics, announce the successful development of improved lentiviral (LV) vectors, driving higher titers of functional viral particles based on the application of ProteoNic’s 2G UNic™ premium vector technology.

Both companies, located on the Leiden Bio Science Park, recognise the need to increase viral titres and optimise production processes. Cost-efficiency and manufacturing yields play a critical role in the feasibility of vector-based therapies. Through their combined efforts ProteoNic and NecstGen have achieved up to 5-fold higher functional LV titers, with significant potential impact on overall process efficiency.

Frank Pieper, CEO of ProteoNic commented “We are excited to see our collaboration with Necstgen achieving this important milestone. We will continue our efforts aimed at improving AAV and LV vectors, catering to both transient production systems and  stable viral producer cell lines”.

Paul Bilars CEO, NecstGen said “These significant results show the strength and importance of our local ecosystem, and highlight how collectively we can address the challenges of Cell and Gene Therapy development and their translation to solutions for patients and society”.

Through their combined efforts ProteoNic and NecstGen aim to improve and advance AAV and LV viral vector manufacturing technology and increase production capacity as well as payload expression in target tissues, to the ultimate benefit of patients in need of Cell and Gene Therapies.

The companies aim to make the improved technology broadly accessible. ProteoNic will offer licensing and co-development opportunities, while NecstGen plans to apply the technology in its CDMO business.

About ProteoNic

ProteoNic is a privately held company with offices in Leiden, the Netherlands and in the Boston area, USA. The company offers technology and services for the generation of cell lines and viral vectors with greatly improved production characteristics. The company commercializes its proprietary 2G UNic™ technology through licensing and partnership arrangements. For more information, see www.proteonic.nl.

 

About NecstGen

NecstGen is a new centre of excellence for Cell and Gene Therapy, located in a purpose-built GMP facility on the largest bio-cluster in the Netherlands, Leiden Bio Science Park. Here, NecstGen provides critical contract development, manufacturing and rental services to academic and small/large industrial therapy developers to deliver a new generation of therapies to patients. For more information visit www.necstgen.com.

For more information please contact:

 

ProteoNic

Mark Posno, PhD

Vice President Business Development

T: +1 617 480 8016

E: posno@proteonic.nl

 

NecstGen

Tristan Pritchard-Meaker, PhD

Head of Business Development

E: tristan@necstgen.com

Pan Cancer T and NecstGen Collaborate to Accelerate Novel TCR-T Therapies into Clinical Development

Pan Cancer T and NecstGen Collaborate to Accelerate Novel TCR-T Therapies into Clinical Development

Pan Cancer T and NecstGen Collaborate to Accelerate Novel TCR-T Therapies into Clinical Development

Rotterdam and Leiden, the Netherlands, November 23, 2022 – Pan Cancer T B.V., a biotech spin-off from Erasmus MC dedicated to the discovery and development of novel second generation TCR-T therapies against solid tumors, and NecstGen B.V., The Netherlands Center for the Clinical Advancement of Stem Cells and Gene Therapy, today announced a partnership to advance Pan Cancer T’s innovative therapies into the clinic. Under the agreement, NecstGen will provide process and analytical development capabilities.

 “We are delighted about this important partnership with NecstGen, which enables us to set up top-tier GMP manufacturing processes for our novel TCR-T therapies,” said Katrien Reynders-Frederix, CEO of Pan Cancer T. “This is a key prerequisite for reaching our next major milestone, i.e., progressing our first therapeutic candidate into clinical development.”

Both Pan Cancer T and NecstGen recognize the need to implement from the start appropriate production processes required for cutting-edge GMP manufacturing to ensure safe and effective cell and gene therapies. NecstGen’s Cell Therapy Development team, led by Melissa van Pel is excited to be working with Pan Cancer T on this project.

Paul Bilars, CEO of NecstGen, said: “I very much see this project as a demonstration of NecstGen’s mission to enable therapy developers to reach their goals. By doing so, we at NecstGen play our part in the successful delivery of cell and gene therapies. For NecstGen to be chosen by Pan Cancer T as a partner on their journey is indeed an honor.”

The NecstGen team has built a state-of-the-art development and manufacturing facility and will collaboratively work with Pan Cancer T’s experts. Each party will bring its unique knowledge and ability to this strategically important partnership.

About Pan Cancer T

Pan Cancer T was founded in late 2020 and as a spin-off from Erasmus MC (Rotterdam, the Netherlands) to advance novel, next generation TCR T-cell therapies for hard-to-treat solid tumors. The Company’s approach includes three differentiating elements. Firstly, our discovery platform uncovered 30 novel tumor targets that are selectively and robustly expressed across 10 major solid tumors. Secondly, we develop engineering technology that enhances the durability of the T cells, making them resistant to the local immune suppression of the tumor. The Company has ongoing R&D programs to develop safe and effective adoptive T cell therapies amenable to large cohorts of patients with triple negative breast cancer as well as cancers of the bladder, ovarium, colorectum, prostate, skin, esophagus, lung, or brain. For more information, please visit: www.pancancer-t.com

About NecstGen

NecstGen is a new centre of excellence for Cell and Gene Therapy development and manufacturing, located in a purpose-built GMP facility at the Leiden Bio Science Park. NecstGen has a focused expert team and bespoke facility to offer the support and capacity needed to deliver Cell and Gene Therapies to patients. Our +4,000 m2 facility encompasses development labs and GMP suites to accommodate the manufacturing of therapies in different operational modes. NecstGen’s dedicated Viral Vector suites offer segregated inoculum, upstream and downstream processing capabilities to maximize batch throughput. The multi-use Grade B and C cleanrooms are available for manufacturing therapies, including Cell Therapies, Ex vivo Gene Therapies, and Gene Therapies. NecstGen offers complete contract manufacturing for Cell Therapy and Viral Vector and cleanroom rental for any mammalian-based therapy type. 

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.

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