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基因治疗初创公司首次试验获批

Gene therapy startup emerges with green light for first-of-its-kind trial

BioPharma Dive 等信源发布 2024-09-12 23:15

可切换为仅中文


Vironexis Biotherapeutics emerged from stealth with plans to start what it claims will be the first-ever clinical trial testing a cancer immunotherapy delivered with the help of an adeno-associated virus, or AAV.

Vironexis Biotherapeutics从隐形中脱颖而出,计划开始进行首次临床试验,测试在腺相关病毒(AAV)帮助下进行的癌症免疫疗法。

The startup’s technology combines an AAV — a common gene therapy delivery tool — with an immunotherapy approach now widely used in oncology. The idea is to give patients a one-time treatment that spurs the immune system’s T cells into continuously attacking tumors over the long term.

这家初创公司的技术将AAV(一种常见的基因治疗传递工具)与目前广泛用于肿瘤学的免疫治疗方法相结合。这个想法是给患者一次性治疗,刺激免疫系统的T细胞长期持续攻击肿瘤。

Vironexis’s first prospect is known as VNX-101 and it will be tested in certain people with acute lymphoblastic leukemia in a Phase 1/2 trial, the company said Thursday. VNX-101 has received a pair of designations from the Food and Drug Administration meant to encourage and speed up development of treatments for pressing medical needs..

Vironexis公司周四表示,它的第一个前景被称为VNX-101,将在某些急性淋巴细胞白血病患者中进行1/2期试验。VNX-101已获得美国食品和药物管理局(FDA)的两项指定,旨在鼓励和加速开发满足紧迫医疗需求的治疗方法。。

The company licensed its core technology, dubbed TransJoin, from Nationwide Children’s Hospital in Columbus, Ohio, a leading hub for gene therapy research. Nationwide’s Timothy Cripes, a pediatric division chief, led work on the technology that was published in Science Advances in 2022. Cripes is one of the company’s founders, along with Brian Kaspar — the scientific founder of the company that developed Zolgensma — and CEO Samit Varma..

该公司从俄亥俄州哥伦布市全国儿童医院(National Children's Hospital)授权其核心技术TransJoin,该医院是基因治疗研究的领先中心。Nationwide的儿科部门主管蒂莫西·克里佩斯(TimothyCripes)领导了这项技术的研究,该技术于2022年发表在《科学进步》杂志上。克里佩斯是该公司的创始人之一,布莱恩·卡斯帕(BrianKaspar)是开发佐尔根斯马(Zolgensma)的公司的科学创始人,首席执行官萨米特·瓦玛(SamitVarma)。。

Vironexis is not the only company thinking about harnessing the power of gene therapy to treat cancer in a different way. Siren Biotechnology emerged from stealth last year with its own “immune-gene therapy” technology in preclinical testing.

Vironexis并不是唯一一家考虑利用基因疗法的力量以不同方式治疗癌症的公司。Siren Biotechnology去年以其自身的“免疫基因疗法”技术在临床前测试中脱颖而出。

Vironexis is starting off with $26 million in seed financing led by Drive Capital and Future Ventures. Moonshots Capital and Capital Factory also participated in the funding.

Vironexis以2600万美元的种子融资开始,由Drive Capital和Future Ventures牵头。Moonshots Capital和Capital Factory也参与了资助。

The company says its approach has the potential to produce safer, more effective, longer-lasting and more easily manufactured and administered therapies than those currently available. But AAV gene therapies have issues of their own; manufacturing is complex and the approach offers risks that are much different than pills a patient can stop taking if they experience issues..

该公司表示,其方法有可能产生比目前可用的更安全,更有效,更持久,更容易制造和管理的疗法。但是AAV基因疗法有自己的问题;制造过程很复杂,这种方法带来的风险与患者遇到问题时可以停止服用的药物大不相同。。

All told, Vironexis says it has more than 10 product candidates in the pipeline. It plans to advance VNX-101 into the clinic in the fourth quarter and is working to obtain FDA permission to start a clinical trial for VNX-202 to prevent metastatic HER2+ cancer next year. It also has a range of other products designed to treat blood-based cancers and prevent solid tumor metastases, as well as a cancer vaccine..

Vironexis表示,总共有10多种候选产品正在开发中。它计划在第四季度将VNX-101推进临床,并正在努力获得FDA的许可,以便在明年开始VNX-202的临床试验,以预防转移性HER2+癌症。它还拥有一系列其他产品,用于治疗血液癌症和预防实体瘤转移,以及癌症疫苗。。