Gene therapies can correct diseases at their genetic core, but their success hinges on viral vector manufacturing. As gene therapy technology becomes more advanced, producing viral vectors becomes more challenging, requiring a manufacturing partner that is not only versed in viral vector production but also agile enough to adapt to the shifting tides of innovation.

基因疗法可以从基因核心纠正疾病，但其成功取决于病毒载体的制造。随着基因疗法技术的进步，生产病毒载体变得更加具有挑战性，需要一个不仅精通病毒载体生产，而且足够灵活以适应不断变化的创新潮流的制造合作伙伴。

A closer look at the experiences of two companies offers insight into what sponsors at different stages of the development and manufacturing cycle need in a contract development and manufacturing organization (CDMO) partner.NysnoBio: A pioneer in early-onset Parkinson's therapy Jennifer Johnston, PhD, has dedicated more than two decades to researching Parkinson’s disease.

仔细观察两家公司的经验，可以深入了解处于开发和制造周期不同阶段的赞助商在合同开发和制造组织（CDMO）合作伙伴中需要什么。尼斯诺比奥（NysnoBio）：早发性帕金森病治疗的先驱詹妮弗·约翰斯顿（JenniferJohnston）博士致力于帕金森病的研究20多年。

In recent years, she has focused on the Parkin (PRKN) gene, which is implicated in Parkin-PD, an early onset form of the chronic disease. “Parkin-PD is the most potent known genetic form of Parkinson’s, with almost 100% penetrance,” Johnston explained at Thermo Fisher Scientific’s viral vector manufacturing site.

近年来，她专注于帕金（PRKN）基因，该基因与帕金PD（一种慢性病的早期发作形式）有关。约翰斯顿在Thermo Fisher Scientific的病毒载体制造厂解释说：“帕金PD是帕金森氏症最有效的已知遗传形式，几乎具有100%的外显率。”。

“Patients affected are typically younger than 35 years old, experiencing significant disability in their prime years.” Having pioneered the basic biochemistry and therapeutic potential for the Parkin gene, Johnston founded NysnoBio in 2019 to take aim at this target. With a goal of developing a Parkin gene replacement therapy, NysnoBio is in the early stages of its journey and their challenges are manifold: navigating the gene therapy landscape, ensuring their work's reproducibility, and scaling production without compromising quality.

“受影响的患者通常不到35岁，在青春年少时会出现严重残疾。”约翰斯顿开创了帕金基因的基本生物化学和治疗潜力，并于2019年成立了NysnoBio，以实现这一目标。NysnoBio的目标是开发一种帕金基因替代疗法，目前正处于其旅程的早期阶段，他们面临的挑战是多方面的：导航基因疗法领域，确保其工作的可重复性，以及在不影响质量的情况下扩大生产规模。

Their efforts got a significant boost in July of 2023, when the company was awarded a research grant from the Michael J. Fox Foundation for Parkinson’s Research. Like many emerging companies, NysnoBio.

2023年7月，该公司获得了迈克尔·J·福克斯帕金森病研究基金会的研究资助，这大大推动了他们的努力。像许多新兴公司一样，NysnoBio。