Democratisation of cell and gene therapies using NanoCAR technology

使用 NanoCAR 技术实现细胞和基因疗法的民主化

• 批准号: 10053947
• 金额: $ 6.37万
• 依托单位: IMMTUNE THERAPIES LIMITED
• 依托单位国家: 英国
• 项目类别: Grant for R&D
• 财政年份: 2023
• 资助国家: 英国
• 起止时间: 2023 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=10053947
• 关键词: Democratisation; cell; gene; therapies; using

The immune system contains many different cells with different properties, and recently, technologies have been developed that empower immune cells against specific diseases. An example where immune cells are modified to make them effectively kill cancer cells is CAR-T therapy. These therapies hold the promise of selectively destroying cancer cells by finding these cells throughout the body (unlike surgery/radiotherapy) and have been successful against previously incurable blood cancers. However, these life-changing treatments are **prohibitively expensive** to the NHS (~250 patients per year allowed within the NHS cancer budget, and 5000 UK patients diagnosed annually) because of their current external manufacturing process. The production can take 60 days, requiring expert labour, and expensive facilities/materials. **Costs exceed £280,000/dose, and only available at specialist centres.**It is clear that these therapies **cannot become mainstream medicines in current form**. This delays their transformative potential in many common diseases which could benefit millions.ImmTune has developed a **nanoparticle-based technology** (similar to the one used in Pfizer's COVID vaccine) for the purpose of making these therapies **inside patients**. Our product will be a simple injection allowing rapid dosing to all the patients in need in a way that's faster, affordable and more effective. **Our technology would democratise these powerful therapies, and remove barriers of inequality in accessing these treatments**.We have demonstrated the potential of our technology for a subset of immune cells, T cells by creating CAR-T cells using our technology, and are now expanding the reach of our platform to a wider set of patients by testing it in a different immune cell subtype; natural killer (NK) cells. These cells have shown to be more potent at addressing more blood cancer, and solid tumour conditions. A successful project outcome would lead to us catering to a wider patient population.Dr Bakul Gupta is one of the **founders/CEO** and has years of experience in working with such nanoparticle-based technologies. Her knowledge and domain e**xpertise has laid the foundation** for the technology being built by the company. She is a **young first-time scientist turned entrepreneur who aspires to create affordable therapies for all patients using her skill set and the most cutting-edge scientific developments, and also be a trailblazer for women founders in the biotech sphere.** She has been leading the company for 3 years, and wants to continue doing this effectively by tapping into different expertise via IUK and KTN networks if awarded this grant.

免疫系统包含许多具有不同特性的不同细胞，最近，已经开发出使免疫细胞能够对抗特定疾病的技术。一个例子是，免疫细胞被修饰，使它们有效地杀死癌细胞是CAR-T疗法。这些疗法有望通过在全身找到癌细胞来选择性地摧毁癌细胞（与手术/放疗不同），并且已经成功地对抗了以前无法治愈的血癌。然而，这些改变生活的治疗方法对NHS来说非常昂贵（NHS癌症预算内每年允许约250名患者，每年诊断5000名英国患者），因为它们目前的外部制造过程。生产可能需要60天，需要专业劳动力和昂贵的设施/材料。** 费用超过£ 280，000/剂，仅在专科中心提供。很明显，这些疗法 ** 不能成为目前形式的主流药物 **。ImmTune开发了一种基于纳米颗粒的技术（类似于辉瑞公司的COVID疫苗中使用的技术），目的是在患者体内进行这些治疗。我们的产品将是一种简单的注射剂，允许以更快，更实惠和更有效的方式快速给所有有需要的患者用药。** 我们的技术将使这些强大的疗法民主化，并消除获得这些治疗的不平等障碍 **。我们已经证明了我们的技术对免疫细胞亚群的潜力，通过使用我们的技术创造CAR-T细胞，T细胞，现在正在通过在不同的免疫细胞亚型中测试我们的平台，将其扩展到更广泛的患者群体;自然杀伤（NK）细胞。这些细胞已被证明在解决更多的血液癌症和实体瘤疾病方面更有效。一个成功的项目结果将使我们迎合更广泛的患者群体。Bakul Gupta博士是 ** 创始人/首席执行官 ** 之一，在使用这种基于纳米颗粒的技术方面拥有多年的经验。她的知识和领域经验为公司正在建立的技术奠定了基础。她是一个年轻的第一次科学家变成企业家谁渴望创造负担得起的疗法为所有患者使用她的技能和最前沿的科学发展，也是一个开拓者的女性创始人在生物技术领域。她已经领导公司3年了，如果获得这笔赠款，她希望通过IUK和KTN网络利用不同的专业知识继续有效地做到这一点。