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MedGenCell's PD-1 Gene-Edited T Cell Injection Approved for Clinical Trials
2024-02-01
Article Details
January 24, 2024
eMedClub News
According to the CDE official website, MedGenCell (Beijing) Co., Ltd(hereinafter referred to as "MedGenCell") has received approval for its Investigational New Drug (IND) application for the clinical trial of its PD-1 gene-edited T cell injection. The product is an autologous T cell preparation with PD-1 gene knockout, intended for use in patients aged 18-75 with advanced non-small cell lung cancer.
MedGenCell is a low-key "powerhouse." Many people still associate the company with the headline news in Nature magazine in 2016, which reported on China's first clinical trial using CRISPR-edited T cell products to treat recurrent tumors. This was commented on by "the father of CAR-T research," Carl June, who believed it triggered a biotech competition between China and the United States.
As the "first to take the plunge," MedGenCell did not overly capitalize on its title as the "world's first clinical GMP-grade gene-edited T cell medicine." The company also did not let itself be disrupted by the U.S. side's immediate reporting in Science after completing three cases. Instead, MedGenCell adhered to GCP new drug standards to complete a standard Phase I clinical trial, including the number of cases required. Together with West China Hospital of Sichuan University, it completed the world's first Phase I clinical trial of gene-edited cell anti-tumor therapy. In April 2020, the complete Phase I clinical trial results were prominently published in Nature Medicine. This report also represents the largest number of cases completed to date in a Phase I clinical trial of gene-edited cell therapy for tumors globally.
After the publication, MedGenCell once again focused intently on returning to scientific research, and this period of “quietness” lasted for two years. Although the scientific validity and safety of the gene-edited cell products were proven in its Phase I investigator-initiated trial (IIT), MedGencell did not rush into immediately filing for IND approval. Instead, the company dedicated itself to optimizing its processes and refining its work to develop a more efficient, less toxic, and stable editing and cell engineering amplification system.It is reported that the editing efficiency has been improved from 20-30% with the initial generation process to a consistently high rate of over 90%. Additionally, the success rate of expanding edited cells has reached over 90%, making the pharmaceutical process more stable and controllable. Moreover, changes in the epigenetic landscape of T cells have been observed, with a significant increase in TCR repertoire diversity and a proportion of stem-like T cells exceeding 30%. These enhancements lead to a more persistent and effective anti-tumor response upon infusion, significantly improving the drugability of the treatment, as reported in Nature Medicine.
The industry-leading new media platform eMedClub reported on MedGenCell, noting that their evaluation of the "Sino-U.S. biotech competition" seems prescient today. Although MedGenCell has not extensively disclosed its progress or engaged in commercial promotion, its pioneering approach has ignited a trend among domestic enterprises, much like a spark that has now grown into a prairie fire. The long-term iterative development of the PD-1 gene-edited T cell injection fully embodies the company’s philosophy: science for the benefit of humanity and craftsmanship to build a brand. It is believed that with years of technological accumulation, this approval will mark the beginning of MedGenCell’s emergence, leading to remarkable achievements.
Drug Development Process
The PD-1 gene-edited T cell injection, previously developed jointly by MedGenCell and West China Hospital of Sichuan University, was used in the world's first-in-human clinical trial of gene-edited cells. A commentary in the top headline of Nature magazine noted that this "triggered a biotech race between China and the United States"; Goldman Sachs Group rated it as a "milestone event in China's biotechnology journey in 2018"; and the complete Phase I investigator-initiated trial (IIT) results were published in the prestigious international journal Nature Medicine (April 2020).
Despite having already demonstrated the scientific validity and safety of the gene-edited cell product in the Phase I IIT, the company did not rush to file for IND approval. Instead, it dedicated itself to the iterative optimization of its processes and meticulous refinement, resulting in a more efficient, less toxic, and stable editing and cell engineering amplification system. The editing efficiency has been improved from an initial 20-30% to over 90%, and the expansion capability of the cells post-editing has also significantly increased, making the pharmaceutical process more stable and controllable. The long-term iterative development of this product fully embodies the company’s philosophy: science for the benefit of humanity and craftsmanship to build a brand.
Milestone Events in Gene Editing Therapy
On November 16, 2023, the world's first CRISPR gene-edited stem cell therapy (Exa-cel, CASGEVY™) received conditional approval from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). On December 8 of the same year, the U.S. Food and Drug Administration (FDA) announced its approval of the product for the treatment of sickle cell disease (SCD). Analysis predicts that the price will be over one million dollars, marking a milestone event in CRISPR gene-edited cell therapy.
Innovative Technology, Industry Leadership
MedGenCell is a high-tech enterprise primarily engaged in research and product development in the fields of cell medicines, gene editing, and epigenetics. The company is committed to innovative CRISPR gene editing therapies that embody technological innovation and industry leadership. Gene editing therapies show great potential not only in monogenic inherited diseases but also in the treatment of solid tumors, which pose the greatest threat to human health due to their complex mechanisms involving multiple genes. This is one of the main focuses of MedGenCell.
The "PD-1 Gene-Edited T Cell Injection," which has now received implicit approval for clinical trials, was developed and produced by Chengdu MedGenCell Co., Ltdand submitted to the National Medical Products Administration through its wholly-owned subsidiary, MedGenCell (Beijing) Co., Ltd. This is also the first IND-approved cell-based new drug in Sichuan Province. With the implicit approval of the IND, the company is accelerating the industrialization process related to this product.
