[Preview of the Weekly] Gene Editing Therapies Move from Lab to Clinic: Assessing Risks and Prospects (AI Translation)
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- The UK's Medicines and Healthcare products Regulatory Agency granted conditional marketing authorization for the CRISPR/Cas9 gene-editing therapy Exa-cel (brand name: Casgevy) on November 16, 2023, for treating sickle cell anemia and transfusion-dependent beta-thalassemia. This marks the first approved gene-editing treatment aimed at curing diseases by knocking out genes, with clinical trial effectiveness data over 95%.
- The launch of this therapy is seen as a significant boost to the field of gene editing, but it also brings mixed feelings among Chinese pharmaceutical entrepreneurs who believe China could have been at the forefront. The He Jiankui scandal in 2018, involving the creation of genetically edited babies, led to stringent regulations and halted many ongoing projects in China.
- While the shadow of He Jiankui's case is fading and more Chinese companies are entering clinical trials for gene-editing therapies, challenges remain such as patent barriers set by foreign competitors and strict domestic policies on human genetic resources. Additionally, there are concerns about ethical boundaries regarding inheritable human gene editing and debates over how to balance innovation with safety and affordability.
文｜财新周刊 蒋模婷、崔笑天，钟腾达（实习）
By Caixin Weekly's Jiang Moting, Cui Xiaotian, Zhong Tengda (Intern)
2023年11月16日，CRISPR/Cas9基因编辑疗法Exa-cel（商品名：Casgevy）获得英国药品和保健产品监管局的有条件上市许可，用于治疗镰状细胞贫血（下称“镰贫”）和输血依赖型β-地中海贫血（下称“地贫”）。更早的两周前，为讨论该疗法在美上市事宜，美国食品药品监督管理局（FDA）咨询委员会召开会议，最终专家全票支持。
On November 16, 2023, the CRISPR/Cas9 gene-editing therapy Exa-cel (commercial name: Casgevy) received conditional marketing authorization from the UK's Medicines and Healthcare products Regulatory Agency for the treatment of sickle cell anemia (referred to as "sickle cell") and transfusion-dependent β-thalassemia (referred to as "thalassemia"). Two weeks earlier, a meeting was convened by the advisory committee of the U.S. Food and Drug Administration (FDA) to discuss the marketing of this therapy in the United States, which ultimately received unanimous support from the experts.
这是基因编辑新时代的开端——全球迎来第一款利用基因敲除治愈疾病的上市疗法。更令人振奋的是，它的临床试验有效性数据高达95%以上，堪称华丽。
This marks the dawn of a new era in gene editing—the world welcomes the first marketed therapy that cures diseases through gene knockout. Even more exhilarating is its clinical trial efficacy data, which stands at over 95%, a figure that can be described as splendid.
疗法背后的CRISPR/Cas9技术，至今仍是最主流的基因编辑工具之一，被誉为“基因魔剪”“上帝手术刀”，并在2020年获得诺贝尔奖。“我认为，技术已经准备好了。”加州大学伯克利分校分子与细胞生物学教授Fyodor Urnov欣慰而自信地说，“经过近30年的发展，它将迎来自己在医学上的黄金时代（prime time）。”
The CRISPR/Cas9 technology behind the therapy remains one of the most mainstream gene-editing tools to date, hailed as "genetic scissors" and "the hand of God in surgery," and was awarded the Nobel Prize in 2020. "I believe the technology is ready," says Fyodor Urnov, a professor of molecular and cell biology at the University of California, Berkeley, with a mix of relief and confidence. "After nearly 30 years of development, it is about to enter its prime time in medicine."
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