There is no universal method for the synthesis of peptide–oligonucleotide conjugates and, in most cases, the choice of method and the conditions for synthesis are on a case-by-case basis. However, the increasing potential of oligonucleotide therapeutics and the need to enhance their targeted ...
Timothy syndrome (TS) is a severe, multisystem disorder characterized by autism, epilepsy, long-QT syndrome and other neuropsychiatric conditions1. TS type 1 (TS1) is caused by a gain-of-function variant in the alternatively spliced and developmentall
Over 16 oligonucleotide therapeutics have been approved, and hundreds more are in clinical trials. But biopharma manufacturing doesn’t happen in a vacuum. The processes used for oligonucleotide synthesis create challenges for sustainability. Large volumes of hazardous solvents or reagents may...
Oligonucleotide-based therapeutics are an emerging class of small drugs completely synthetic in origin, which do not integrate into the host genome, and have a defined duration of therapeutic activity after which effects recover to baseline. In addition, they offer a high degree of specificity for ...
Ashok K. Prasad, in Carbohydrates in Drug Discovery and Development, 2020 Abstract Oligonucleotide-based therapeutics is a highly promising drug development platform as this can also target candidates which were once considered ‘undruggable’. Despite its promise, specific targeting and delivery of ...
optimization of oligonucleotide therapies, as well as their delivery mechanisms. Currently, a total of 25 approved oligonucleotide therapeutics have been approved by regulatory authorities worldwide, and numerous others are in pre-clinical and clinical studies w...
Moumnéet al. Oligonucleotide Therapeutics: From Discovery and Development to Patentability.Pharmaceutics2022,14(2), 260;https://doi.org/10.3390/pharmaceutics14020260 Alzheimer’s Association. Tau Topic Sheet.https://www.alz.org/media/Documents/alzheimers-dementia-tau-ts.pdf. Accessed Oct...
In this 12-month, multicenter, open-label, multiple-dose, dose-escalation, phase 1b/2 trial (PQ-110-001; ClinicalTrials.gov identifier NCT03140969), patients received sepofarsen (QR-110; ProQR Therapeutics) into the worse-seeing eye (treated eye) by intravitreal injection following standard pr...
Interest in and investment into the use of antisense oligonucleotides for therapeutics continue to grow. As of 2023, there are 18 FDA-approved nucleic acid based treatments, including inclisiran, a treatment for high cholesterol, and nusinersen, a therapeutic for spinal muscular atrophy - with a ...
2025, Brain, Behavior, and Immunity Show abstract Applications and Developments of Gene Therapy Drug Delivery Systems for Neurological Disorders 2024, Advanced Therapeutics Novel Therapeutic Horizons: SNCA Targeting in Parkinson’s Disease 2024, BiomoleculesPublished by Elsevier Inc. ...