Researchers from City of Hope discussed preclinical data for CD33-targeted chimeric antigen receptor (CAR) T cells being developed for the treatment of acute myeloid leukemia (AML).
Friedreich’s ataxia (FA) is an autosomal recessive disorder caused by mutations in the FXN gene and characterized by cardiomyopathy, gait ataxia and sensory loss, with cardiac complications the main cause of death among patients with FA.
Homology Medicines Inc. has reported data on an adeno-associated viral (AAV) vector-based therapy, HMI-104, an AAV treatment intended to induce hepatic expression of a complement C5 monoclonal antibody, named as C5mAb, for the potential treatment of paroxysmal nocturnal hemoglobinuria (PNH) as well as other complement-driven pathologies. C5mAb is thought to bind to C5 and inhibit the C5-mediated hemolysis observed in PNH.
Arrhythmogenic cardiomyopathy (ACM) is a devastating inherited disorder characterized by massive cardiomyocyte loss, fibrofatty infiltration and ventricular arrhythmias, among others. Most known genetic causes of ACM involve the gene PKP2, which encodes plakophilin-2. An unmet medical need exists regarding therapies that correct this PKP2 deficiency.
Trigeminal neuralgia (TN) is a chronic disorder caused by the hyperactive functioning of a damaged trigeminal nerve that provokes severe facial pain coming from the trigeminal nerve.
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.
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