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Establishing Commercial AAV Manufacturing in the UK

Establishing Commercial AAV Manufacturing in the UK
在英国建立商业 AAV 制造
批准号:
103241
负责人:
金额:
$191.09万
依托单位:
依托单位国家:
英国
项目类别:
Collaborative R&D
财政年份:
2018
资助国家:
英国
项目状态:
已结题
起止时间:
2018 至 --

项目摘要

项目成果

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中文摘要
翻译
NightstaRx(NSR)是一家生物技术公司,其使命是通过开发新的基因疗法来治疗导致失明的罕见退行性疾病,从而恢复和维持视力。NSR正在与合作伙伴Cell & Gene Therapy Catapult一起申请这笔赠款,以加快开发一种名为视网膜色素变性的遗传性失明疾病的治疗方法。CGT是一个拥有科学专业知识和认可实验室的组织,可以帮助NSR完成最先进的制造工艺的开发,以进行这种处理。该项目的结果将意味着NSR将能够治疗患者,以帮助改善他们的生活质量并保持他们的独立性。这种生产工艺的建立不仅使其他类似产品的生产更容易,而且有助于使英国成为全球一流的病毒基因疗法生产商,用于后期临床和商业供应。反过来,这也将增加熟练劳动力并创造就业机会。所有这些好处将导致每家公司增加收入,这将有助于使英国经济更加强大。
英文摘要
NightstaRx (NSR) is a biotechnology company whose mission is to restore and maintain sight by developing novel gene therapies to treat rare, degenerative diseases causing blindness. NSR are applying for this grant, in conjunction with a partner, Cell & Gene Therapy Catapult, to expedite the development of a treatment for an inherited blindness disease called Retinitis Pigmentosa. CGT are an organisation who have the scientific expertise and accredited laboratories to help NSR complete the development of a state of the art manufacturing process to make this treatment. The result of this project will mean NSR will be able to treat patients to help improve their quality of life and keep their independence. Establishment of this type of manufacturing process would not only make manufacturing of other similar products easier, but also help put the UK in a position to be a first in class global manufacturer of viral gene therapies for late stage clinical & commercial supply. In turn it would also increase the skilled workforce and create jobs. All of these benefits will result in each company increasing their revenues, which will contribute to making the UK economy stronger.
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