bamosiran (SYL040012) / PharmaMar - LARVOL DELTA

Emerging drugs for the treatment of glaucoma: A review of phase II & III trials. (PubMed, Expert Opin Emerg Drugs) - "However, the availability of generic prostaglandin analogs may limit adoption of these novel compounds as first line agents, except for certain subgroups of glaucoma patients. Use as adjuvant or second line therapy appears more likely for the majority of glaucoma patients."

Journal • P2 data • Review • Glaucoma • Ocular Inflammation • Ophthalmology • Optic Neuritis • Pain

RNA therapeutics in ophthalmology - translation to clinical trials. (PubMed, Exp Eye Res) - "This review provides a detailed insight into the recent developments and clinical trials that have been conducted for several gene-silencing therapies, including ISTH0036, SYL040012, SYL1001, PF-04523655, Sirna-027, QR-110, QR-1123, QR-421a and IONIS-FB-L in glaucoma, dry eye disease, age-related macular degeneration, diabetic macular oedema and various inherited retinal diseases. Our aim is to explore the potential of these drugs whilst evaluating their associated advantages and disadvantages, and to discuss the future translation of RNA therapeutics in ophthalmology."

Clinical • Journal • Review • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Diabetic Macular Edema • Dry Age-related Macular Degeneration • Dry Eye Disease • Glaucoma • Macular Degeneration • Macular Edema • Ocular Infections • Ophthalmology • Retinal Disorders

The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market. (PubMed) - Pharm Res - "...The siRNAs in focus are PF-04523655, TKM-080301, Atu027, SYL040012, SYL1001, siG12D-LODER (phase 2), QPI-1002, QPI-1007, and patisiran (phase 3)...Miravirsen is an AntimiR-122 for hepatitis C virus infection. The flexibility of RNAi technology is easily understood taking into account: (i) the different drug targets (i.e. p53, caspase 2, PKN3, β2-adrenergic receptor, mutated KRAS, microRNAs); (ii) therapeutic conditions, including ophthalmic diseases, kidney injury, amyloidosis, pancreatic cancer, viral hepatitis; and (iii) routes of administration (ocular, intravenous, subcutaneous, intratumoral). Although some issues are still matters of concern (delivery, toxicity, cost, and biological barriers), RNAi definitively opens a wide avenue for drug development."

Journal • Review • Biosimilar • Gastrointestinal Cancer • Hepatitis C Virus • Immunology • Oncology • Ophthalmology • Pancreatic Cancer • Renal Disease

SYL040012, Treatment for Open Angle Glaucoma (clinicaltrials.gov) - P2; N=180; Recruiting; Sponsor: Sylentis, S.A.; Not yet recruiting -> Recruiting

Enrollment open • Biosimilar • Hematological Malignancies

Progress on Ocular siRNA Gene Silencing Therapy and Drug Delivery Systems. (PubMed, Fundam Clin Pharmacol) - "RNA interference (RNAi) is being increasingly used in the treatment of these disorders with siRNA drugs, bevasiranib, AGN211745 and PF-04523655 for AMD, and SYL040012 and QPI-1007 for glaucoma. Compared with the non-viral vectors, viral vectors have limited payload capacity and potential immunogenicity. This review summarizes the progress of the ocular siRNA gene silencing therapy by focusing on siRNA drugs for AMD and glaucoma already used in clinical research, the main routes of drug delivery, and the non-viral vectors for siRNA drugs."

Journal • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Gene Therapies • Genetic Disorders • Glaucoma • Macular Degeneration • Ophthalmology • Rare Diseases • Retinal Disorders