Cellares and Seoul National University Hospital Partner to Automate Manufacturing of Gene-Modified HSPC Therapy for PNH
Cellares, an Integrated Development and Manufacturing Organization (IDMO), and Seoul National University Hospital (SNUH), a leading translational medical center in South Korea, have announced a strategic collaboration to evaluate automated manufacturing for a gene-modified hematopoietic stem and progenitor cell (HSPC) therapy being developed for paroxysmal nocturnal hemoglobinuria (PNH).
The partnership will assess whether SNUH’s existing gene-modified HSPC manufacturing process can be transferred and automated on Cellares’ Cell Shuttle platform. The collaboration will specifically examine the use of Cellares’ fully integrated, single-use electroporation module to support a two-step transfection workflow.
The companies intend to use the collaboration to establish a manufacturing approach that can support the preclinical development and investigational new drug (IND)-enabling stages of SNUH’s PNH program. The work is also intended to help establish a scalable manufacturing strategy that could support future clinical development of the therapy in the United States.
The partnership represents Cellares’ second collaboration in Asia and expands its network of relationships across the Asia-Pacific region. It also brings together SNUH’s expertise in translational medicine and gene-modified hematopoietic stem cell therapies with Cellares’ focus on automated cell therapy manufacturing.
Focus on Gene-Modified HSPC Manufacturing
Hematopoietic stem and progenitor cells are responsible for generating blood and immune cells throughout the body. Because of their ability to repopulate the hematopoietic system, HSPCs have become an important platform for the development of gene therapies targeting inherited and acquired blood disorders.
Gene modification of HSPCs can potentially enable a patient’s own stem cells to be engineered outside the body and subsequently returned to the patient. Once engrafted, the modified cells can generate blood cells carrying the desired genetic modification.
However, manufacturing gene-modified HSPC therapies can be complex. Processes may involve multiple manipulation steps, specialized equipment, highly controlled environments and significant hands-on labor. Maintaining consistency across batches is particularly important as programs move from early research into clinical development.
Cellares and SNUH are therefore evaluating whether automation can help address some of these challenges in the PNH program.
The proof-of-concept collaboration will focus on translating SNUH’s manufacturing process onto the Cell Shuttle platform. The companies will assess whether the automated system can reproduce key elements of the existing process while improving manufacturing consistency and scalability.
Addressing Paroxysmal Nocturnal Hemoglobinuria
The program being supported by the collaboration targets paroxysmal nocturnal hemoglobinuria, commonly known as PNH.
PNH is a rare acquired blood disorder associated with abnormal destruction of red blood cells, known as hemolysis. The disease results from mutations affecting hematopoietic stem cells, leading to the production of blood cells that are vulnerable to complement-mediated destruction.
Patients with PNH can experience a range of complications associated with hemolysis and abnormal blood-cell function. Symptoms and disease manifestations can vary, and the condition may require long-term medical management.
Gene-modified HSPC therapy represents an investigational approach aimed at addressing the underlying cellular source of the disease. By modifying hematopoietic stem cells, researchers are exploring whether a durable population of genetically corrected cells can be established following treatment.
For such approaches to progress through clinical development, however, manufacturing processes need to be sufficiently reliable, reproducible and scalable.
The Cellares-SNUH collaboration is intended to address this manufacturing component of the PNH program before potential future clinical development in the United States.
Translating SNUH’s Process to the Cell Shuttle
A central component of the collaboration will be the translation of SNUH’s existing gene-modified HSPC manufacturing process onto the Cell Shuttle platform.
The Cell Shuttle is designed as an end-to-end automated cell therapy manufacturing platform. Rather than requiring operators to manually perform individual manufacturing steps using separate pieces of equipment, the system is intended to integrate multiple processes into a more automated workflow.
For the SNUH program, particular attention will be given to a two-step transfection workflow.
Transfection refers to the introduction of genetic material into cells. In gene-modified cell therapy manufacturing, efficient and controlled delivery of genetic material is critical to generating the desired cellular product.
Cellares’ integrated electroporation module is designed to support this process. Electroporation uses controlled electrical pulses to temporarily increase the permeability of cell membranes, allowing genetic material or other molecular payloads to enter cells.
By incorporating electroporation directly into the Cell Shuttle, Cellares aims to reduce the need for separate equipment and manual transfers during manufacturing.
The proof-of-concept work will determine how effectively SNUH’s process can be implemented within this automated environment.
Potential Benefits of Automation
Manufacturing is one of the key challenges facing advanced cell and gene therapies. While scientific development can establish the biological rationale for a therapy, translating that research into a repeatable clinical manufacturing process can require substantial investment in facilities, equipment, personnel and quality systems.
Gene-modified HSPC manufacturing can be particularly demanding because of the sensitivity of the cells and the number of processing steps involved.
Manual processes can introduce variability and require highly trained personnel to perform repetitive operations. As clinical programs expand, increasing production capacity can also require additional manufacturing resources and facilities.
Automation offers a potential way to standardize manufacturing activities and reduce reliance on manual intervention.
For SNUH’s PNH program, the companies will evaluate whether automation can improve manufacturing reliability while creating a process that can potentially be scaled as the program advances.
The collaboration will also examine the potential economic impact of automation. Cellares believes that reducing manual labor and integrating manufacturing steps could help lower production costs.
Lower manufacturing costs could be particularly important for complex cell and gene therapies, where manufacturing can represent a substantial component of overall development and treatment expenses.
The companies emphasize that the current collaboration is a proof-of-concept evaluation. The ability to achieve these potential benefits will depend on the results of the manufacturing assessment and subsequent development work.
Supporting Future U.S. Clinical Development
Another objective of the collaboration is to establish manufacturing workflows that can support SNUH’s future clinical development plans in the United States.
Moving a cell therapy program from one country or manufacturing environment into another can require extensive process characterization, validation and regulatory preparation. Establishing a scalable and reproducible manufacturing strategy early in development may help support future clinical and regulatory activities.
Cellares will support SNUH’s preclinical development and IND-enabling manufacturing workflows for the PNH program. The work is intended to provide a foundation for potential future clinical development in the U.S.
Professor Youngil Koh, Director of the SNUH GMP Facility, said the partnership will allow the institution to evaluate how automation could support the complexity and requirements associated with gene-modified HSPC therapies.
“Advancing gene-modified HSPC therapies requires a manufacturing approach that can support both the complexity of the process and the rigor of clinical translation,” Koh said.
He added that the collaboration will evaluate whether automation can strengthen manufacturing reliability and scalability as the program moves toward clinical development.
Cellares Expands Asia-Pacific Collaboration Network
The partnership also expands Cellares’ activities in the Asia-Pacific region.
SNUH is an established translational medical center with experience collaborating with global institutions on gene-modified hematopoietic stem cell therapies. Its GMP manufacturing capabilities provide an important foundation for developing and translating advanced cell therapy programs.
For Cellares, working with SNUH provides an opportunity to apply its automated manufacturing platform to another gene-modified HSPC program while expanding its relationships with clinical and research institutions in Asia.
The collaboration is Cellares’ second partnership in Asia, according to the company, further extending its international network.
Fabian Gerlinghaus, Co-Founder and CEO of Cellares, said gene-modified HSPC therapies represent a natural application for the Cell Shuttle platform because of the manufacturing complexity involved.
“Gene-modified HSPC therapies are a natural fit for the Cell Shuttle and an area where Cellares has established manufacturing expertise,” Gerlinghaus said.
He added that automation of complex workflows, including electroporation, could provide the scalability, reliability and manufacturing economics needed to advance such therapies through clinical development.
Gerlinghaus said the partnership will extend Cellares’ capabilities to SNUH as the institution prepares its PNH program for potential future clinical development in the United States.
Building a More Scalable Manufacturing Model
The collaboration reflects a broader trend in the cell and gene therapy industry toward greater automation of manufacturing processes.
As more cell therapies progress into clinical development and potential commercialization, developers face the challenge of producing increasingly complex therapies consistently and at a cost that can support broader patient access.
Traditional manufacturing models can require extensive manual processing and highly specialized personnel. While these approaches have enabled the development of numerous cell therapies, they can become difficult to scale as production volumes increase.
Automated manufacturing platforms are being developed to address these challenges by integrating multiple process steps, standardizing operations and reducing manual intervention.
Cellares’ Cell Shuttle is designed around this model, with the goal of creating a more integrated manufacturing environment for cell therapies.
The SNUH collaboration will provide an opportunity to assess how this approach can be applied specifically to gene-modified HSPC manufacturing and a two-step transfection process.
Next Steps for the Collaboration
The immediate focus will be the proof-of-concept evaluation of SNUH’s manufacturing process on the Cell Shuttle platform. The companies will assess the feasibility of transferring the process to the automated system and evaluate the resulting manufacturing workflow.
The work will contribute to SNUH’s preclinical development and IND-enabling activities for its PNH program. If successful, the collaboration could establish a manufacturing strategy capable of supporting later-stage development and potential U.S. clinical trials.
The companies will also assess whether automation can improve process reliability, reduce manufacturing costs and provide greater scalability as the program advances.
For SNUH, the partnership provides access to an automated manufacturing platform as it develops its gene-modified HSPC therapy for PNH. For Cellares, it represents another application of its Cell Shuttle technology and expands its collaboration footprint across Asia.
Ultimately, the partnership is focused on one of the practical challenges facing gene-modified cell therapies: developing a manufacturing process that can move from specialized research environments toward reproducible, scalable clinical production.
As the PNH program progresses, results from the proof-of-concept work will help determine the role that automated manufacturing can play in supporting its future development. The collaboration also illustrates the growing importance of manufacturing technology in advancing complex gene-modified cell therapies from translational research toward potential clinical use.
About Cellares
Cellares is the first Integrated Development and Manufacturing Organization (IDMO), providing global cell therapy development and manufacturing services through an Industry 4.0 approach to the mass manufacture of the living drugs of the 21st century. The company enables drug sponsors to develop, scale, and commercialize cell therapies with the capacity, reliability, and economics required to meet total patient demand.
Cellares’ fully automated platforms — Cell Shuttle® for end-to-end cell therapy manufacturing and Cell Q™ for automated in-process and release quality control — are deployed across its network of IDMO Smart Factories worldwide. These technologies deliver industry-leading manufacturing economics, higher process success rates, and the ability to produce up to 10× more cell therapy batches than conventional CDMOs with comparable footprint and headcount, resulting in the lowest cost of manufacturing in the industry.
The Cell Shuttle is the first cell therapy manufacturing platform to receive the FDA’s Advanced Manufacturing Technology (AMT) designation, and has demonstrated a 100% automation success rate across more than a dozen automated processes. Cellares was also accepted into the FDA’s inaugural Manufacturing PreCheck cohort, the only cell therapy platform among seven companies nationwide, giving Cellares early and ongoing engagement with the FDA during facility development and application submission periods, compressing regulatory timelines and de-risking the path to commercial-scale production for the drug sponsors building on Cellares’ platform.
Cellares has achieved key clinical validation milestones, including a successful IND Amendment enabling active clinical manufacturing on the Cell Shuttle platform, and the successful dosing of first patients in a partner clinical trial — marking the platform’s transition from development-stage technology to clinically validated manufacturing infrastructure. These milestones span multiple therapeutic areas and cell therapy modalities, including both oncology and autoimmune indications.
Headquartered in South San Francisco, California, Cellares operates its first commercial-scale IDMO Smart Factory in Bridgewater, New Jersey, with additional facilities under construction in Europe and Japan. Through its global manufacturing network, Cellares is purpose-built to support both clinical and commercial programs and to expand access to life-saving cell therapies worldwide. For more information, visit www.cellares.com and follow Cellares on LinkedIn.
About Seoul National University Hospital
Seoul National University Hospital (SNUH) is a medical center in South Korea dedicated to advancing patient care through the integration of education, research and clinical practice. SNUH works to advance state-of-the-art medical care and expand knowledge through medical research, while fostering the next generation of global healthcare leaders. As a translational medical center, SNUH also collaborates with leading institutions worldwide on the development of innovative therapies, including gene-modified hematopoietic stem cell-based treatments. Its institutional mission emphasizes excellence across medical care, research and education.