At the recent ASGCT meeting, researchers from Exegenesis Bio Inc. presented preclinical data for EXG-102-031, a novel recombinant adeno-associated virus (rAAV)-gene therapy being developed for the treatment of neovascular age-related macular degeneration (AMD), also called wet AMD (wAMD).
Heart disease caused by damage to blood vessels is the leading cause of death worldwide. Arteries become clogged with fats and cholesterol when certain proteins in the body, known as lipoproteins, combine with and transport fats in the blood to cells. Scientists have long believed that the LDL receptor molecule was responsible for the intracellular transport of LDL. But given that some individuals lacking the LDL receptor still have high levels of LDL, questions remain about the mechanism.
Gene therapy technology makes it possible to select diseased or mutated cells from a patient, modify them in the laboratory and reintroduce them to the body to treat different disorders. This is known as ex vivo autologous gene therapy. The difference with allogeneic cell techniques is whether the donor is oneself (autologous) or a compatible person (allogeneic), which would provide healthy cells that do not need genetic modification.
Sania Therapeutics Inc. is setting out its stall at the American Society of Gene & Cell Therapy (ASGCT) conference in Los Angeles this week, after generating proof of concept for its chemogenetics approach to treating motor disorders. The company has engineered adeno-associated viral vectors that can be targeted to specific cell types. It will use these to deliver well-characterized ion channels to dysfunctional motor neurons.
New and updated preclinical data presented at the American Society of Gene & Cell Therapy Congress in Los Angeles, by: Adicet Bio, Adverum Biotechnologies, Bloomsbury Genetic Therapies, Canbridge Pharmaceuticals, Chroma Medicine, Mink Therapeutics, Orchard Therapeutics, Orna Therapeutics, Outpace Bio, Poseida Therapeutics, Voyager Therapeutics.
Sironax has described NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, Parkinson’s disease, multiple sclerosis, traumatic brain injury, diabetic neuropathy and chemotherapy-induced peripheral neuropathy.
Jiangsu Chia Tai Tianqing Pharmaceutical Group Co. Ltd. has divulged exportin-1 (CRM1; XPO1) receptor antagonists reported to be useful for the treatment of lymphoma and leukemia.
Acerand Therapeutics (Hong Kong) Ltd. has identified tetrahydrofuran-containing polycyclic derivatives and their pharmaceutically acceptable salts reported to be useful for the treatment of ovarian cancer.
Bolt Biotherapeutics Inc. has synthesized antibody-drug conjugates (ADCs) comprising cysteine-mutant antibodies targeting programmed cell death 1 ligand 1 (CD274; PD-L1) covalently bound to one or more Toll-like receptor 7 (TLR7) and/or TLR8 agonists through a linker reported to be useful for the treatment of cancer.
Merck Sharp & Dohme Corp. has disclosed receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis and inflammation.
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