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

据估计，全球有五分之一的人终生面临永久性视力丧失或失明的风险（DeloitteAccessEconomics-2017）。英国有200万人患有严重的视力丧失，严重影响了他们的日常生活，其成本估计为281亿英镑（RNIB-2017）。青光眼是致盲的第二大原因，英国有50万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，并且在标签外使用（非预期用例）。这种治疗方法有局限性，包括靶向性差、脱靶细胞毒性增加，导致严重的副作用（失明、组织损伤、感染）。靶向性眼纤维化预防治疗的发展被认为是临床眼科最大的未满足需求之一（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，并利用超过90年的综合经验来解决影响其大规模采用的关键障碍。NanoGenics开发了LipTide-ECP105，这是一种具有竞争力的价格的创新眼纤维化预防治疗方法，适合全球市场实施，独特地提供：有效负载保护在肽纳米颗粒中被脂质层包围，以促进内体释放。*使用表面显示的特定肽序列进行靶向递送。*完全新颖的siRNA序列靶向减少青光眼术后纤维化和瘢痕形成。LipTide-ECP105将通过靶向治疗彻底改变青光眼术后治疗，减少局部治疗的毒副作用和成本，具有广泛的药物输送潜力。到2026年，青光眼治疗市场估计价值76亿美元（复合年增长率：2.9%）（transparencymarkeresearch -2018），眼科是基因治疗技术（spark - therapeutics和nightstar - therapeutics）进入市场的明确初始途径。NanoGenics旨在通过开发LipTide-ECP105来解决这一未满足的需求，LipTide-ECP105是一种具有竞争力价格的创新药物递送方法，适合全球市场实施，独特地提供：有效负载保护在肽纳米颗粒中被脂质层包围，以促进内体释放。*利用半胱氨酸环表面显示的特定肽序列进行靶向递送。*完全新颖的siRNA序列靶向减少青光眼术后纤维化和瘢痕形成。在成功的体外和体内研究的基础上，初步毒理学/疗效结果得到证实，需要一个15个月的研究计划来准备LipTide-ECP105用于人体临床试验。LipTide平台也提供了巨大的潜力，作为药物递送市场中突破性的基因治疗递送途径，与传统的腺相关病毒(AAV)/慢病毒有效载荷递送相比，提供了许多优势，可以治疗广泛的适应症（例如，神经母细胞瘤/囊性纤维化/癌症有待探索）。

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.