Researchers from Janssen Research & Development LLC presented preclinical data for JNJ-75220795 (ARO-PNPLA3), a GalNAc-conjugated small interfering RNA (siRNA) therapeutic targeting the PNPLA3 gene, being developed for the treatment of metabolic dysfunction-associated steatohepatitis (MASH).
France’s Théa Open Innovation, a subsidiary of Laboratoires Théa SAS, terminated its licensing agreement with South Korea’s Olix Pharmaceuticals Inc., and returned global rights of Olix’s ocular small interfering RNA (siRNA) biologic agents, OLX-301D and OLX-301A.
France’s Théa Open Innovation, a subsidiary of Laboratoires Théa SAS, terminated its licensing agreement with South Korea’s Olix Pharmaceuticals Inc., and returned global rights of Olix’s ocular small interfering RNA (siRNA) biologic agents, OLX-301D and OLX-301A.
Shares of Avidity Biosciences Inc. hit a 52-week high on reports of promising data from the first efficacy cohort of its phase I/II Fortitude study testing delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD), including impressive biomarker results that could indicate a path for potential accelerated approval.
Previous studies have found that the effectiveness of vaccines can be increased with the knockdown of HBsAg using a small interfering RNA (siRNA). The efficacy of a bivalent mRNA vaccine in combination with the siRNA AD-66810, which targets the X gene expression in the hepatitis B virus (HBV) genome, was tested in a murine model of AAV/HBV by scientists from Clearb Therapeutics Inc.
Silence Therapeutics plc disclosed positive top-line 36-week data from the Alpacar-360 phase II study with zerlasiran, a short interfering RNA gene muter that targets lipoprotein(a), but also said Mallinckrodt plc has quit development of a separate prospect.
In a paper published on Feb. 21, 2024, in Science Translational Medicine, researchers from the Versiti Blood Research Institute described how they successfully controlled bleeding for months in hemophilia A models using a siRNA therapy that targeted plasminogen.
Medicxi is spinning six of its early stage companies into an immuno-dermatology specialist and turbocharging development with a $100 million seed round.
Researchers from Sirnaomics Inc. presented preclinical evaluation of novel intravenous polypeptide nanoparticle designed to simultaneously deliver two siRNAs, silencing TGF-β and COX-2.
N4 Pharma plc’s subsidiary Nanogenics Ltd. has signed a contract to start the formulation and sequence selection work to prepare its ECP-105 product for testing in preclinical studies.
