Essentia BioSciences Advances Stem Cell-Derived Islet Therapy for Diabetes Amid Global Industry Momentum

According to data released by the International Diabetes Federation (IDF), the global number of people with diabetes is projected to reach 629 million by 2045. Statistics show that in 2021, diabetes accounted for at least $966 billion in healthcare expenditures worldwide, representing 9% of total adult healthcare spending and marking an approximately 316% increase over the past 15 years. As the number of patients with diabetes continues to rise year by year, the diabetes drug market has firmly become the second-largest pharmaceutical market, surpassed only by the oncology drug market.

As a chronic disease requiring long-term, uninterrupted management, diabetes is currently treated primarily through oral medications, insulin injections, and islet transplantation. While the former two approaches can control blood glucose levels to some extent, they cannot cure diabetes. Although islet transplantation offers a potential cure, it is limited by the scarcity of donor islets, as well as the high surgical risks and significant side effects associated with the procedure.

With the advancement of biomedicine, the emergence of stem cell therapy holds promise for a radical cure for diabetes. By leveraging the capacity of stem cells to undergo multilineage differentiation and proliferation under specific conditions, they can be induced to differentiate into insulin-secreting beta-like cells, thereby regenerating and enhancing islet cell function to achieve a curative outcome. Furthermore, stem cells participate in immunomodulation and the induction of immune tolerance, helping to restore immune homeostasis within the pancreatic islets.

The ability of stem cells to cure diabetes has prompted numerous pharmaceutical companies to compete in this field.Currently, major multinational pharmaceutical companies such as Eli Lilly, Novo Nordisk, and Sanofi, as well as innovative biopharmaceutical companies including Vertex Pharmaceuticals, ViaCyte, Sernova, AltuCell, Semma Therapeutics, and Beta-O2, have all established a presence in this field.

Among international pharmaceutical companies focusing on the stem cell industry, Vertex has recently garnered the most attention. At the end of last month, Vertex presented the latest data from its Phase 1/2 clinical trial of the stem cell therapy VX-880 for the treatment of type 1 diabetes at the 83rd Scientific Sessions of the American Diabetes Association (ADA).

VX-880 is a stem cell-derived, fully differentiated islet cell therapy.Essentially, it consists of pancreatic beta cells derived from the in vitro induced differentiation of pluripotent stem cells (iPSCs, human induced pluripotent stem cells, and ESCs, embryonic stem cells). These cells are infused into patients via the hepatic portal vein to secrete insulin; therefore, VX-880 requires long-term immunosuppressive therapy to protect the islet cells from immune rejection.

According to disclosed data, Vertex has designed a Phase 1/2 multicenter, single-arm, open-label clinical trial for patients with impaired hypoglycemia awareness and severe hypoglycemia. The study is designed to last five years and comprises three parts (A, B, and C), planning to enroll 17 patients with severe type 1 diabetes to evaluate the safety and efficacy of VX-880.

To date, six patients with type 1 diabetes have received VX-880 in a Phase 1/2 study. Following treatment with VX-880, all six patients demonstrated restored insulin secretion for glucose regulation to varying degrees, resulting in significant improvements in glycemic control.

The positive data disclosed for VX-880 further demonstrate the clinical accessibility of pluripotent stem cell-derived islet products.

In recent years, the clinical translation of pluripotent stem cell-derived islet products has been preliminarily validated by major pharmaceutical companies.As rising stars, startups possess advantages such as greater flexibility and agility compared to large enterprises when catching up on progress and laying out new fields.

Dr. Liu Tongri, Founder and CEO of Essentia Biosciences, told VCBeat, “In terms of R&D technology, we also follow the pathway currently adopted by major global pharmaceutical companies to differentiate pluripotent stem cells into islets. Horizontal data comparisons show that our differentiation efficiency, cell yield, and the functionality of the differentiated islet cells are no lower than, and in many cases even superior to, other publicly available data.”

Essentia Biosciences’ efforts were soon recognized by the public. In October 2022, the company received a £15,000 grant from Innovate UK’s RTO/Catapult program to support the research and development of its technology for differentiating pluripotent stem cells into pancreatic islet cells.

Essentia Biosciences is a biopharmaceutical company dedicated to the research and development of pluripotent stem cell-derived islet therapies and the construction of a universal pluripotent stem cell differentiation platform. It was founded in Beijing in 2021 by Dr. Liu Tongri.

Dr. Liu Tongri is a member of the London Stem Cell Society and the UK Society for Gene and Cell Therapy in Regenerative Medicine. The founder, who hails from the School of Life Sciences at Peking University and the University of Oxford, previously served as the Chief Overseas Representative and General Manager of an iPSC company. In these roles, he was involved in corporate execution, financing, and matrix management prior to launching his venture.Possesses many years of overseas experience in iPSC stem cell research and development, regulatory affairs, corporate operations, and the biomedical industry.

He has led the technical translation and CMC (Chemistry, Manufacturing, and Controls) production of clinical trial drugs in the UK. He organized and delivered presentations at scientific advice meetings (Pre-IND) with the UK Medicines and Healthcare products Regulatory Agency (MHRA), designed Phase 1/2a clinical protocols, and organized and presented at Innovation Office Meetings (Pre-Pre-IND) with the MHRA. He possesses in-depth knowledge of the laws and regulations governing cell-based therapies and cell quality standards, as well as the design and execution of animal studies for cell therapy development and clinical trial regulatory requirements, across the NMPA, FDA, EMA, and MHRA jurisdictions.

Throughout his many years in scientific research and industrialization, what left the deepest impression on Dr. Liu Tongri was not the numerous achievements he has attained, but a lecture he attended during his doctoral studies. “It was a lecture on embryonic stem cell-based therapies. An Italian professor successfully differentiated embryonic stem cells into skin cells to treat a child’s skin disease. Through a video, we witnessed the child’s remarkable recovery—from being covered in blood with no normal skin remaining, to happily running on a lawn after receiving the cell transplant.”

“At the time, the audience consisted entirely of PhDs and postdoctoral fellows from the University of Oxford, yet these scientists were moved to tears when the chair professor played an interview with the patient’s mother, featuring her child running in the background.”“That was the first time I truly and viscerally felt how cell therapy could bring about a transformative change in the lives of patients who had once been hopeless,” remarked Dr. Liu Tongri. “This also became my original motivation for persisting in the development of cell-based drugs.”

Guided by this vision, Dr. Liu Tongri founded Essentia Biosciences. “Entrepreneurship is not my ultimate goal; rather, my aim is to accelerate the clinical translation of pluripotent stem cell-derived islet therapies through pharmaceutical partnerships. Since large pharmaceutical companies rarely incubate innovative projects from the ground up, I chose to establish a startup as a vehicle to fast-track the development of these therapeutics.”

To date, Essentia Biosciences has successfully established a core management team covering the entire process of R&D, operations, registration, and quality management. The team brings together talents from Peking University, Tsinghua University, the University of Oxford, and the University of Cambridge. Currently, Essentia Biosciences has evolved into an R&D-led, multidisciplinary team, with members possessing academic and industrial expertise spanning physiology, biophysics, oncology, biology, chemistry, immunology, as well as materials science, organizational management, and corporate finance.

Backed by a strong team and a promising market, Essentia Biosciences quickly attracted investor attention. In March 2022, the company secured favor from Yusheng Ventures, completing an angel financing round worth tens of millions of RMB. The proceeds were primarily allocated to the research and development of two product pipelines and to support operational activities in both China and the United Kingdom. In May of the same year, Essentia Biosciences officially obtained overseas direct investment (ODI) registration from the Beijing Municipal Development and Reform Commission and the Beijing Municipal Bureau of Commerce, and established its wholly-owned subsidiary, Aceso BioSciences, in Cambridge, UK.

The completion of the overseas investment filing marks a substantive advancement in Essentia Biosciences' internationalization strategy.

“The reason we chose the UK as our entry point into the international market is that in 2016, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) approved the world’s first iPSC-based drug to enter clinical trials. In the field of industrializing pluripotent stem cells, Europe has a relatively mature regulatory and industrial environment,” Dr. Liu Tongri told VCBeat.

With the support of a professional team and capital, Essentia Biosciences has rapidly completed the establishment of a GMP-compliant clinical-grade iPSC cell bank and a pluripotent stem cell (universal) platform. Its cell bank has passed audits by the European and U.S. regulatory authorities, and related technologies have been patented both domestically and internationally, possessing independent intellectual property rights. Currently, the company has completed cell differentiation, identification, and functional testing, demonstrating the feasibility and efficacy of developing pluripotent stem cell-derived islet drugs.

Currently, Essentia Biosciences has established a pluripotent stem cell-derived islet drug pipeline (ESN5800).This pipeline leverages allogeneic pluripotent stem cell-derived islet technology to achieve efficient and safe differentiation of islet cells or cell clusters, followed by clinical transplantation to realize the therapeutic goal of islet regeneration. Through active collaboration with upstream and downstream partners in the industry chain, Essentia Biosciences is progressively refining the testing of multiple pluripotent stem cell lines using various differentiation methods, thereby establishing a comprehensive differentiation pathway and methodology spanning the entire process from stem cells to endoderm, pancreatic progenitors, and finally islet cells.

Building on its cell-based therapies, Essentia Biosciences has also developed encapsulation technologies that combine biomaterials with islets or other cell-like products, establishing a drug pipeline integrating pluripotent stem cells, islets, and medical devices.Essentia Biosciences develops islet organoids derived from pluripotent stem cell differentiation and utilizes bio-3D printing technology to encapsulate these organoids with biomaterials, creating a combination drug-device product consisting of pluripotent stem cell-derived islet cells and encapsulation materials. The company is developing products similar to ViaCyte’s therapies and Vertex Pharmaceuticals’ VX-264, aiming to enhance transplantation efficacy, reduce immune rejection, and provide more options for transplantation sites.

To address critical challenges such as immune rejection associated with pluripotent stem cell therapies, Essentia Biosciences has utilized CRISPR/Cas9 gene-editing technology to engineer cells that evade the human immune response, thereby enabling the large-scale production of “off-the-shelf” therapeutics.

Dr. Liu Tongri analyzed, “In my opinion,The biggest industry pain point for cell therapies derived from pluripotent stem cells is their druggability.Issues such as tumorigenicity, immune rejection, and differentiation capacity at various stages of cell therapies derived from pluripotent stem cells can all be controlled through well-defined technical and analytical methods.However, approaches to establishing corporate production and quality control systems, as well as defining product release specifications or industry standards, vary among companies, as there is currently no unified industry standard. These systems are critical to the successful commercialization of pipeline products. Once established, these systems must ensure consistent product quality while scaling up production. Moreover, even a minor change in a single parameter after product launch may entail substantial human and financial resources.

To address this pain point, Essentia Biosciences adopted a “begin with the end in mind” approach from the outset of product development. “We first established the concept of developing a pluripotent stem cell-derived islet therapy, and then built the required technology platform, complemented by corresponding quality systems and multinational corporate frameworks, rather than pursuing mere commercialization based solely on existing technologies.”

Essentia Biosciences has consistently focused on the development of pluripotent stem cell differentiation-based therapeutics. Prior to its establishment, the company thoroughly reviewed international regulatory frameworks and has maintained long-term communication with the National Medical Products Administration (NMPA), ensuring that every operational step aligns with compliance systems. “Although this is an emerging field, our close engagement with the regulatory system from the outset of R&D has significantly facilitated the smooth advancement of our pipeline.”

To support the rapid advancement of its subsequent pipeline, Essentia Biosciences is advancing the construction of its Global Production Center in Daxing District. Upon completion, the facility’s production capacity is expected to meet the demands of Phase II/III clinical trials for islet transplantation and other CDMO services. In line with this expansion, Essentia Biosciences is actively pursuing a new round of financing.

We look forward to Essentia Biosciences launching its off-the-shelf pluripotent stem cell-derived islet cell therapy soon, bringing hope to hundreds of millions of diabetes patients worldwide.