EN
Milestone: World's First CRISPR Gene Editing Therapy Approved in the UK!
2023-11-17
Article Details
On November 16, the world's first CRISPR gene editing 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). Analysts had previously predicted that the pricing would exceed $2 million.
This is a milestone event in CRISPR gene editing therapy. MedGenCell is committed to innovative CRISPR gene editing therapies that embody scientific advancement and industry leadership. The company completed the world's first-in-human clinical trial of gene editing, which was featured in a front-page article in Nature, suggesting that it sparked a "biotech race between China and the United States." The full results of the complete Phase I clinical trial were published in Nature Medicine in 2020. Since then, multiple institutions in the U.S. and elsewhere have conducted various gene editing clinical trials, leading to the approval of the relatively straightforward single-gene disorder gene editing therapy today. This demonstrates the spirit of innovation for the benefit of humanity! While gene editing therapies hold significant potential in treating monogenic inherited diseases, they also show immense promise in combating solid tumors, which pose the greatest threat to human health due to their complex mechanisms involving multiple genes. This remains one of the primary focuses of MedGenCell.
Note：
（1）Chengdu MedGenCell, as the product developer, collaborated with West China Hospital to complete the world's first-in-human clinical trial of gene editing. This was featured on the front page of Nature, with “the father of CAR-T research,” Carl June, commenting that it triggered a “biotech race between China and the United States.”
Nature reported:http://www.nature.com/news/2016-in-news-the-science-events-that-shaped-the-year-1.21159
Sohu reported:https://www.sohu.com/a/119191833_545560；
（2）On April 28, 2020, Nature Medicine published online the results of the world's first Phase I clinical trial of gene editing: safe and effective. As the product developer, MedGenCell, along with its clinical trial partner West China Hospital, was listed as co-first author.
https://baijiahao.baidu.com/s?id=1665195158928694517&wfr=spider&for=pc
