One of the challenges in designing genetic and cellular strategies is getting the therapy to the right place. This is even more complicated when it comes to the nervous system. The brain is a complex organ that contains the most differentiated and inaccessible cells in human biology. It is an impassable safe, protected by the blood-brain barrier.

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).

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).

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.