在 FDA 批准后，Excision 的针对 HIV 的 CRISPR 基因编辑疗法正在

StephenW

在 FDA 批准后，Excision 的针对 HIV 的 CRISPR 基因编辑疗法正在进入人体测试作者：安娜莉·阿姆斯特朗 | 2021 年 9 月 17 日上午 10:10HIV 将其遗传物质整合到宿主细胞的基因组中，这意味着可用的疗法无法将其移除。 (NIAID)分享Facebook推特领英电子邮件打印一种 CRISPR-Cas9 基因编辑技术在清除小鼠中的 HIV 方面显示出前景，即将进入人体测试。根据该公司的新闻稿，在 FDA 批准一项研究性新药申请后，Excision BioTherapeutics 将把基于 CRISPR 的疗法 EBT-101引入临床试验。EBT-101 正在开发中，作为一种潜在的 HIV 病毒清除疗法，或者用该公司的话说，“一种潜在的慢性 HIV 功能性治愈方法”。我们不喜欢在这里到处乱说“治愈”这个词。但 Excision 认为这种疗法可以取代标准护理逆转录病毒疗法，后者可以防止 HIV 复制但不会将其从体内清除。这意味着患者继续接受治疗，这会导致严重的副作用并影响生活质量。现在，随着人体测试的开始，真正开始了解这项备受赞誉的新技术能否实现这一目标。相关：使用 CRISPR 消除 HIVHIV 将其遗传物质整合到宿主细胞的基因组中，这意味着可用的疗法无法将其移除。天普大学和内布拉斯加大学医学中心的一组科学家在临床前测试期间使用 CRISPR 和抗逆转录病毒疗法的组合成功地从小鼠身上完全清除了病毒。他们还没有发现可能与该研究中的疗法相关的不良事件，该研究于 2019 年发表。“如果你只是剪一个，病毒就会在它周围发生变异。我们进行了多次削减以停用病毒基因组，”首席执行官 Daniel Dornbusch 此前在接受采访时告诉 Fierce Biotech。此后，EBT-101 已在非人类灵长类动物中进行了测试，结果表明它到达了体内每个 HIV 储存库所在的组织。切除术从大学获得许可，目的是将其转移到临床试验中。现在，FDA 已加入。相关：生物制药通过基因和细胞疗法应对 COVID-19、HIV 和其他病毒声明称，该生物技术公司计划在今年晚些时候启动 1/2 期临床试验。Excision 使用的技术获得了著名 CRISPR 先驱 Jennifer Doudna 实验室的许可。该公司还在研究其他病毒的类似治疗方法，包括疱疹和乙型肝炎。Excision 的消息是在 Intellia 宣布急性髓性白血病的基因编辑疗法已被 FDA 批准进行人体试验的同一周发布的。

StephenW

Excision's CRISPR gene editing therapy for HIV is heading into human testing after FDA clearance
by Annalee Armstrong | Sep 17, 2021 10:10am
HIV integrates its genetic material into the genome of a host cell, meaning available therapies just can’t remove it. (NIAID)
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A CRISPR-Cas9 gene editing technology that has shown promise in clearing HIV from mice is headed into human testing.

Excision BioTherapeutics will usher the CRISPR-based therapy EBT-101 into clinical trials after the FDA cleared an investigational new drug application, according to the company’s press release.

EBT-101 is under development as a potential virus-clearing treatment for patients with HIV—or, put in the company’s words, “a potential functional cure for chronic HIV.”

We don’t like to throw the word “cure” around here. But Excision thinks the therapy could replace standard-of-care retroviral therapy, which keeps HIV from replicating but does not remove it from the body. That means patients stay on the treatment, which can cause serious side effects and affect quality of life. Now with the start of human testing, the real path to see if this new and lauded tech can accomplish this really begins.

RELATED: Using CRISPR to eliminate HIV

HIV integrates its genetic material into the genome of a host cell, meaning available therapies just can’t remove it. A team of scientists at Temple University and the University of Nebraska Medical Center managed to remove the virus completely from mice during preclinical testing using a combination of CRISPR and antiretroviral therapy. They also found no adverse events that could be linked to the therapy in the study, published back in 2019.

“If you just make a single cut, the virus can mutate around it. We make multiple cuts to deactivate the viral genome,” CEO Daniel Dornbusch previously told Fierce Biotech in an interview.

EBT-101 has since been tested in nonhuman primates, which showed it reached every tissue in the body where HIV reservoirs reside.

Excision licensed the therapy from the universities with a goal of moving it into clinical trials. Now, the FDA is on board.

RELATED: Biopharma tackles COVID-19, HIV and other viruses with gene and cell therapies

The biotech plans to initiate a phase 1/2 clinical trial later this year, according to the statement.

The technology used by Excision was licensed from the lab of famed CRISPR pioneer Jennifer Doudna. The company is also working on similar treatments for other viruses, including herpes and hepatitis B.

Excision's news arrives the same week that Intellia announced a gene editing therapy for acute myeloid leukemia had been cleared for human trials by the FDA.

StephenW

https://www.fiercebiotech.com/bi ... DA&mrkid=767901