Development of a breakthrough anti-fibrotic gene therapy to improve surgical outcomes and reduce re-admission rates for patients with severe glaucoma.

永久性失明或失明的终生风险估计占全球人口的五分之一(DeloitteAccessEconomics-2017)。英国有200万人患有严重的失明，对他们的日常生活产生了巨大的影响，估计成本为281亿英镑(RNIB-2017)。青光眼是第二大致盲原因，有50万英国居民患有开角型青光眼。目前预防眼纤维化的治疗方法是丝裂霉素-C和5-氟尿嘧啶，这两种药物最初都是用于化疗的，但都在标签外(非预期用例)使用。此类治疗具有局限性，包括靶向性差，靶外细胞毒性增加，导致严重的副作用(失明、组织损伤、感染)。开发一种有针对性的眼纤维化预防疗法被认为是临床眼科中最大的未得到满足的需求之一(Expert.Rev.Opthamol.10：65-76)。NanoGenics是一家专门从事药物输送技术开发的中小企业，利用90多年的联合经验来解决影响其广泛采用的关键障碍。NanoGenics公司已经开发出一种价格具有竞争力的创新的眼纤维化预防疗法LipTide-ECP105，它适合在全球市场上实施，独特地提供：\*由脂质层包裹的多肽纳米颗粒内的有效载荷保护，以促进内体释放。\*利用表面显示的特定多肽序列进行靶向输送。\*全新的siRNA序列，旨在减少青光眼手术后的纤维化和疤痕。LipTide-ECP105将使用靶向疗法来革命性地治疗手术后青光眼，这些疗法减少了与局部治疗相关的毒副作用和成本，具有广泛的药物输送潜力。据估计，青光眼治疗药物市场价值很高；2026(CAGR:2.9%)(TransparencyMarketResearch-2018)，投资76亿美元，眼科是基因治疗技术进入市场的明确初始途径(Spark-Therapeutics&Nightstar-Therapuetics).NanoGenics旨在通过开发LipTide-ECP105来解决这一未得到满足的需求，这是一种价格具有竞争力的创新药物输送方法。适合全球市场实施，独一无二地提供：*脂层包围的多肽纳米颗粒内的有效载荷保护，以促进内体释放。\*使用半胱氨酸环显示的表面显示的特定多肽序列进行靶向输送。\*全新的siRNA序列，旨在减少青光眼手术后的纤维化和疤痕。在成功的体外和体内研究的基础上，初步毒理学/有效性结果得到证实，需要15个月的研究计划来准备用于人体临床试验的LipTide-ECP105。LipTide平台还提供了作为药物输送市场内突破性基因治疗输送途径的巨大潜力，与传统腺相关病毒(AAV)/慢病毒有效载荷输送相比，在治疗多种适应症(例如神经母细胞瘤/囊性纤维化/癌症--有待探索)方面具有许多优势。

Lifetime risk of permanent sight loss or blindness was estimated to be 1/5 of people globally (DeloitteAccessEconomics-2017). \>2M people(UK) live with sight loss significant enough to have a drastic impact upon their daily lives, with the cost estimated at £28.1Bn (RNIB-2017). Glaucoma is the second leading cause of blindness, with 500,000 UK residents affected by open-angle Glaucoma.Current treatments for Ocular Fibrosis prevention are Mitomycin-C and 5- Fluorouracil, both of which were originally intended for use in chemotherapy and are used off-label (not intended use-case). Such treatments have limitations including poor targeting, increased off-target cytotoxicity leading to drastic side effects (blindness, tissue-damage, infection). The development of a targeted ocular fibrosis prevention treatment is considered one the greatest unmet needs in clinical ophthalmology (Expert.Rev.Opthamol.10:65-76).NanoGenics are a SME specialising in the development of drug delivery technologies and have used over 90 years of combined experience to address key barriers effecting their wide-scale adoption. NanoGenics have developed LipTide-ECP105, an innovative Ocular Fibrosis prevention treatment at a competitive price which is suitable for global market implementation that uniquely offers:\*Payload protection within peptide nanoparticle surround by a lipid layer to facilitate endosomal release.\*Targeted delivery using specific peptide sequences displayed on the surface.\*Completely novel siRNA sequence targeted at reduce fibrosis and scarring in post-glaucoma surgery.LipTide-ECP105 will revolutionise post-surgical Glaucoma treatment using targeted therapeutics that reduce toxic side-effects and cost associated with topical treatments, with broad drug delivery potential. Glaucoma therapeutics market is estimated to be worth \>$7.6Bn by 2026(CAGR:2.9%)(TransparencyMarketResearch-2018), with ophthalmology being a clear initial route to market for gene therapy technologies (Spark-Therapeutics&Nightstar-Therapuetics).NanoGenics aim to address this unmet need through the development of LipTide-ECP105, an innovative drug delivery method at a competitive price, suitable for global market implementation that uniquely offers:\*Payload protection within peptide nanoparticle surround by a lipid layer to facilitate endosomal release.\*Targeted delivery using specific peptide sequences displayed on the surface displayed in the cysteine loop.\*Completely novel siRNA sequence targeted at reduce fibrosis and scarring in post-glaucoma surgery.Building on successful _in vitro_ and _in vivo_ studies with initial toxicology/efficacy results proven, a 15-month programme of research is required to prepare LipTide-ECP105 for human clinical trials. LipTide platform also offers vast potential as a breakthrough gene therapy delivery route within the drug delivery market, offering many advantages to treat a broad range of indications (e.g.neuroblastoma/cystic fibrosis/cancer-to be explored) over traditional Adeno-Associated Virus (AAV)/lentivirus payload delivery.