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

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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.