Democratisation of cell and gene therapies using NanoCAR technology

免疫系统包含许多具有不同性质的不同细胞，最近，已经开发出使免疫细胞能够抵御特定疾病的技术。免疫细胞被修饰以有效地杀死癌细胞的一个例子是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这些细胞(与外科手术/放射治疗不同)，并且已经成功地治疗了以前无法治愈的血癌。然而，由于目前的外部制造工艺，这些改变生命的治疗对NHS来说是**令人望而却步的昂贵**(NHS癌症预算内每年允许约250名患者，每年有5000名英国患者被诊断)。生产可能需要60天，需要专业的劳动力和昂贵的设施/材料。**费用超过28万GB/剂，仅在专科中心提供。**显然，这些疗法**不能成为当前形式的主流药物**。这推迟了它们在许多常见疾病中的转化潜力，这些疾病可能会使数百万人受益。ImmTune开发了一种**基于纳米颗粒的技术**(类似于辉瑞COVID疫苗中使用的技术)，目的是在**患者体内**进行这些疗法。我们的产品将是一种简单的注射，允许以更快、更实惠和更有效的方式快速给所有需要帮助的患者服用。**我们的技术将使这些强大的疗法大众化，并消除获得这些治疗的不平等障碍**。我们已经通过使用我们的技术创造CAR-T细胞来展示我们的技术对免疫细胞子集T细胞的潜力，现在正在通过在不同的免疫细胞亚型自然杀伤(NK)细胞中进行测试，将我们平台的覆盖范围扩大到更广泛的患者。这些细胞已经被证明在处理更多的血癌和实体肿瘤方面更有效。成功的项目成果将使我们能够迎合更广泛的患者群体。巴库尔·古普塔博士是**创始人/首席执行官**之一，在此类基于纳米颗粒的技术方面拥有多年的工作经验。她的知识和领域经验为该公司正在开发的技术奠定了基础。她是一位**年轻的科学家出身的企业家，她渴望利用自己的技能和最尖端的科学发展为所有患者创造出负担得起的疗法，同时也是生物技术领域女性创始人的先驱。**她已经领导公司三年了，如果获得这笔拨款，她希望通过IUK和KTN网络利用不同的专业知识来继续有效地开展这项工作。

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.