Article source: Medical wheat customers
Regenerative medicine company Cellusion has announced that its iPSC Derived Cornea Endothelial Cell replacement therapy (CLS001) has been awarded by the FDA as an orphan drug (ODD) for the treatment of bullous keratopathy. This determination will further accelerate the global development of the CLS001.
Bullous keratopathy is a kind of cataract, eye surgery or genetic factors reduce corneal endothelial cells caused by corneal blisters and visual impairment, the progressive disease in the case of no effective treatment may lead to blindness, and the current standard therapy is through corneal transplantation, replacement of normal function corneal endothelial cells. According to public data, about 13 million people worldwide are waiting for corneal transplants, and only about 180,000 cases can be performed each year. It is predicted that the global corneal transplant market is expected to grow from $421 million in 2021 to $641 million in 2028, with an estimated compound annual growth rate of 6.2%.
CLS001 CECSi cells developed based on Cellusion proprietary technology for corneal endothelial regeneration is expected to change the current global corneal donor shortage by combining "CECSi cells made of iPS cells with excellent proliferation characteristics" and "simple injection cell delivery procedures without expertise". Unlike traditional corneal transplantation, which uses barometric adhesion, CLS001 therapy, which uses gravity adhesion and must remain face down for three hours after transplantation, creates a smaller wound and can significantly reduce complications.
Also, this candidate addresses one limitation of the current generation of corneal endothoid cells from ESC or iPSC. Previous studies have shown that the production of corneal endothelial-like cells from ESC or iPSC needs to undergo the development of neural spinal cells (NCC) as an intermediate, which may produce a variety of cells, including chondrocytes, adipocytes, and neuronal cells, which is not conducive to large-scale manufacturing. While Cellusions proprietary technology can directly differentiate iPS cells into corneal endothelioid cells without intermediates, which effectively ensures stable cell quality and facilitates rapid mass production of cells. And, CLS001 can be cryopreserved, meaning that the corneal endothelial replacement cells needed for transplant therapy can be produced and stored in advance to be transported everywhere for easy use in patient need.
Previously, Cellusion had announced that it had completed the first patient transplant for CLS001, and in an independent data monitoring committee review held in January 2023, no adverse events were observed within 3 months of the CLS001 transplant, and the patients vision, central corneal thickness, and minimal corneal thickness had tended to improve. Currently, Cellusion is planning research in several regions around the world.
Cellusion Born in 2015 in the Department of Ophthalmology, Keio University School of Medicine, with a unique differentiation induction method including cells from iPSC to CECSi cells. In January 2022, Cellusion announced the completion of 1.1 billion yen financing; in June this year, Cellusion completed a Series C financing of 2.83 billion yen. By the completion of Series C financing, Cellusion raised a total of 4.5 billion yen (about 226 million RMB) to promote phase 1 / 2 clinical trials of CLS001 in Japan and overseas.
In September 2022, Cellusion and Hangzhou star, true creature exclusive license agreement, the greater China region, including Chinese mainland, Hong Kong, Macao and Taiwan, development, manufacturing and commercialization of the exclusive rights to license out star ray true creature, and may get more than $100 million funds, including sales milestone, development milestones, and gradient royalties.
Star ray true creature was founded in July 2022, is a focus on cell therapy and regenerative medicine development of innovative drug platform company, focus on stem cell related technology research and development and clinical transformation, it has completed 45 million yuan of seed round, investors for fosun medicine, incubation fund rehabilitation capital new drug innovation fund. According to public information, The company focuses on tissue regeneration and tumor therapy, and its candidates for tissue regeneration include corneal endothelial cell therapy introduced from Cellusion; in the field of tumor therapy, it is working with top scientists in the field of tumor cell therapy in the United States to jointly develop stem cell-derived CAR-NK.
Recent progress in the domestic iPSC field
eMedClub
The iPSC is a promising track that is used in regenerative medicine, oncology, rare diseases and more. The iPSC track has made a lot of progress recently.
Sankai Biology presented relevant preclinical data on its iPSC-derived CAR-iNK products targeting GPRC5D at the 65th American Society of Hematology (ASH) Annual Meeting. Its iPSC-derived CAR-NK cells were over 99% pure, and its NK cell activation receptor (NKG 2 D, NKp 30) and costimulator (CD226, CD244) expression all exceeded 96%. In in vitro trials, single administration of CAR-iNK after resuscitation significantly reduced tumor burden and improved survival in the Xenograft transplant model. It is reported that based on this project, Sanqi Biology has further developed CIB-315, the first fourth-generation CAR-NK product for MM, combined with the advantages of its own gene editing platform, and plans to apply for IND in 2024.
In December, Ruijians "human dopaminergic precursor cell injection" was approved in clinical trials for early-onset Parkinsons disease with onset age earlier than 50 years old. So far, Ruijian Pharmaceutical has carried out a strategic layout of fully covering the life cycle and treatment cycle in the Parkinsons field.
In November, the global first pipeline clinical grade iPSC derived subtype neural precursor cell product for the treatment of ALS was awarded the title of orphan drug by the FDA, which is the worlds first iPS derived cell drug with the qualification of orphan drug used for the treatment of ALS.
In the same month, allogeneic endothelial progenitor cells (EPCs) injection solutions (ALF202) were implicitly approved in clinical trials for the treatment of severe lower limb ischemia. It is based on the injection of endothelial progenitor cells (EPCs) induced by iPSC. Dennom also has an iPSC-derived cell candidate for acute ischemic stroke, ALF201 injection, which has completed its first patient dose.
In November, The "NCR300 injection" was approved to prevent recurrence after allogeneic hematopoietic stem cell transplantation for acute myeloid leukemia. This is the second IND won by Zhongsheng Source in iNK cell therapy this year, which further expanded the clinical trial of tumor product pipeline registration of the company.
sum up
eMedClub
iPSC is progressing very fast worldwide, especially in recent years, and can be regarded as a key node in the clinical transformation of research results. And the domestic rise is very fast, many enterprises in this year will be candidate pipeline to the clinical. Despite the challenges of tumorigenicity, heterogenicity and immunogenicity in the iPSC field, according to the disclosed data and research trends, the current enterprises all pay attention to its safety, and create technologies as much as possible to reduce the proportion of undifferentiated iPSC, while improving the efficacy. In the future, as iPSC product candidates show more potential in the clinic, they will attract more players, and the industry chain will be further improved.reference material:
1. Official websites of each enterprise
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