In the current era of rapid advancements in medical technology, pluripotent stem cell therapy, with its tremendous potential and continuous breakthroughs, has become the core focus of the global medical field.

Recently, this cutting-edge field has witnessed a series of exciting advancements - not only have they opened up brand-new treatment paths for many intractable diseases, but they have also injected strong impetus into the development of the entire regenerative medicine, making the hope of conquering difficult and complicated diseases increasingly clear.

01 Induce human blood cells into pluripotent stem cells

On July 30th, the research group led by Deng Hongkui from the School of Basic Medical Sciences of Peking University, in collaboration with the research group led by Guan Jingyang from the School of Pharmaceutical Sciences of Peking University, published the latest research results in the international academic journal Cell Stem Cell. This research has for the first time achieved the induction of human blood cells into pluripotent stem cells by chemical methods, overcoming the key bottleneck of the initial cell source in the preparation of chemically induced pluripotent stem cells, and will further promote the accessibility of chemically induced pluripotent stem cell technology.

In this study, the team successfully overcame the key challenges of chemical reprogramming technology and established a method to reprogram human umbilical cord blood and adult peripheral blood cells into hCiPS cells. This novel chemical reprogramming system can induce the generation of hundreds of hCiPS clones in a single well within 20 days, with the fastest induction taking only 12 days. Meanwhile, this technical solution can also stably induce hCiPS cells from cryopreserved blood cells, laying the foundation for the large-scale preparation of hCiPS cell lines using large peripheral blood banks and umbilical cord blood banks.

The team also successfully established a system for reprogramming cells obtained from a small amount of fingertip blood collection into hCiPS cells, providing a simple and efficient strategy for the large-scale construction of hCiPS cell banks. The blood reprogramming method has significantly expanded the application scope of chemical reprogramming technology and for the first time achieved the efficient preparation of hCiPS cells from the most accessible and abundant tissue samples in the human body.

Chemical reprogramming induces human blood cells from different sources to become pluripotent stem cells

The blood chemical reprogramming system represents a new generation of human pluripotent stem cell preparation technology and has the potential to surpass traditional transgenic strategies. This study shows that for peripheral blood mononuclear cells from the same donor, the efficiency of the chemical reprogramming method is more than 20 times higher than that of the traditional four-factor reprogramming method. In addition, the chemical small molecule method is simple and easy to operate, does not require the delivery of any exogenous genes, is convenient for the development of ready-to-use kits, and is easy to be widely applied. This effective and convenient method for reprogramming blood cell chemistry has the potential to become a widely used platform.

02 iPSC-derived cell therapy

On July 8, 2025, BlueRock Therapeutics, a wholly-owned subsidiary of Bayer, announced that the first patient in its Phase I/IIa CLARICO trial (NCT06789445) had received OpCT-001 administration.

OpCT-001 is the world's first IPSC-derived cell therapy to enter clinical trials. It targets primary photoreceptor cell diseases such as retinitis pigmentosa and cone-rod dystrophy. By injecting functional cells into the vitreous cavity to replace degenerated photoreceptor cells, it aims to restore patients' vision. CLARICO is the first human, multi-center, two-part study, which plans to recruit approximately 54 adults. In Phase I, safety was evaluated using a 3+3 dose escalation approach. In Phase II, visual function, functional visual acuity, and anatomical implant indicators will be further assessed. This therapy was granted Fast Track designation by the FDA in February 2025.

The administration of this first patient marks an important step forward in the treatment of photoreceptor degenerative diseases using iPSCs technology, bringing new hope to patients with hereditary retinal diseases who currently lack effective treatment options.

The two breakthroughs mentioned above have pressed the "accelerator" for the clinical application and popularization of pluripotent stem cell therapy. They not only demonstrate the tremendous potential of pluripotent stem cell technology in the field of regenerative medicine, but also mark a crucial step forward for humanity in conquering difficult diseases and achieving precise treatment. In the future, they are expected to bring hope of recovery to more patients.