Functionally active hepatitis B virus (HBV) DNA is open and accessible, while the action of an epigenetic editor turns it into functionally inactive DNA, which is closed and inaccessible. Chroma Medicine Inc. has presented data regarding their epigenetic editor CRMA-1001, which is delivered by lipid nanoparticles.
Nanovation Therapeutics Inc. has established a multiyear partnership with Novo Nordisk A/S to advance the development of novel genetic medicines targeting cardiometabolic and rare diseases. The partnership brings together Nanovation’s proprietary long-circulating lipid nanoparticle (lcLNP) technology for RNA delivery to cells outside of the liver, and Novo Nordisk’s expertise in cardiometabolic and rare disease R&D and clinical translation.
Korro Bio Inc. has established a collaboration with Novo Nordisk A/S to advance the discovery and development of new genetic medicines, initially to treat cardiometabolic diseases. The collaboration brings together Novo Nordisk’s deep cardiometabolic disease understanding and drug development experience with Korro’s proprietary platform to develop RNA editing product candidates for two undisclosed targets.
Voyager Therapeutics Inc. has announced the selection of a development candidate in a gene therapy program for the treatment of an undisclosed neurological disease under its collaboration with Neurocrine Biosciences Inc.
A novel gene therapy that leads to cellular rejuvenation could restore vision after non-arteritic anterior ischemic optic neuropathy (NAION) and glaucoma. The technique is based on a reprogramming process that reverses the epigenetic DNA alterations caused by aging. Preclinical studies in glaucoma mice and nonhuman primates (NHP) models for this stroke-like disorder that affects the eye, showed an improvement of vision and restoration of the damaged axons of the optic nerve.
Researchers from Chroma Medicine Inc. presented preclinical data for the novel PCSK9-targeted epigenetic editor (PCSK9-EE) being developed as cardiovascular therapy for the reduction of low-density lipoprotein cholesterol.
Multiple sulfatase deficiency (MSD) is a rare lysosomal storage disorder caused by pathogenic variants in the sulfatase modifying factor 1 gene (SUMF1). Researchers from the University of Pennsylvania described the efficacy of hematopoietic stem cell transplantation (HSCT) with ex vivo SUMF1 lentiviral gene therapy (SUMF1-GT) in a mouse model of MSD.
Vivet Therapeutics SAS has announced its gene therapy VTX-806 has been awarded European orphan drug designation for the treatment of cerebrotendinous xanthomatosis (CTX), a rare disorder of bile acid metabolism.
Skyline Therapeutics (Shanghai) Co. Ltd.’s SKG-1108, a novel one-time intravitreally delivered gene therapy, has been awarded U.S. orphan drug designation for the treatment of retinitis pigmentosa.
Metagenomi Inc. has reported data from an ongoing preclinical study designed to provide evidence supporting the potential durability and safety of the company’s hemophilia A gene editing investigational therapy, MGX-001.
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