Researchers at Opus Genetics Inc. reported the efficacy of OPGx-BEST1, an AVV-based gene therapy developed to deliver a functional BEST1 transgene to retinal pigment epithelium (RPE) cells to re-establish normal BEST1 expression and activity.
At the European Congress of Endocrinology in Prague, researchers from Juvena Therapeutics Inc. presented the effects of JUV-161, a fusion protein consisting of human insulin-like growth factor 2 linked to human serum albumin, in preclinical models of myotonic dystrophy type 1 (DM1) and sarcopenia.
Fractyl Health Inc. has received clinical trial application authorization in the Netherlands to initiate a first-in-human phase I/II study of RJVA-001, the first clinical candidate from the company’s Rejuva Smart GLP-1 gene therapy platform.
A new mRNA and lipid nanoparticle (mRNA-LNP) platform could selectively reprogram in vivo cytotoxic effector T cells (Teff), the cells responsible for eliminating infected or tumor cells. To achieve this, scientists at the University of Pennsylvania conjugated LNPs with fractalkine, a molecule that binds to the CX3CR1 receptor, which is a marker of Teff cells. Using this strategy, the researchers delivered an mRNA encoding new proteins such as IL‑2 or human CD62 L‑selectin, opening the door to temporarily reprogramming these cells within the body, both in the blood and in lymphoid tissue, where they reside and become activated.
The University of Hong Kong has patented new transcription antitermination protein NusB/N-utilization substance E (NusE) interaction inhibitors reported to be useful for the treatment of gram-positive or gram-negative bacterial infections and fungal infections.
Boyuan Pharmaceutical (Suzhou) Co. Ltd. has disclosed new herpes simplex virus (HSV) inhibitors potentially useful for the treatment of HSV infection and dementia.
Work at Shanghai Jeyou Pharmaceutical Co. Ltd. has led to the identification of new thioether-substituted galactopyranoside derivatives acting as galectin-3 (LGALS3) inhibitors.
Researchers from Sunshine Biopharma Inc. and the University of Arizona reported the discovery and preclinical characterization of MR-1-114, a noncovalent inhibitor of the SARS-CoV-2 papain-like protease (PLpro).
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