The transition from complex and costly ex vivo strategies to platforms that enable direct cellular intervention within the body, known as in vivo therapies, is marking a paradigm change in the field of gene and cell therapies by simplifying manufacturing, improving tissue targeting and expanding clinical access to treatments.

The pathogenesis of multiple sclerosis (MS) has been tied to ineffective immune control of Epstein-Barr virus-driven autoimmune responses. Patients with MS are deficient in protective adaptive natural killer cells (pNK cells) in contrast to healthy individuals. These pNK cells are positive for NKG2A, NKG2C and NKG2D and recognize and kill autoreactive B cells in a selective and efficient manner.

Activation of cannabinoid receptors in the peripheral nervous system may help treat inflammatory and gastrointestinal disorders as well as pain, and several full agonists have been reported but they present safety concerns. Partial agonists, such as those that activate receptors to 20%-50% of full activity, may be safer, yet so far such agonists have emerged serendipitously.

Neuromyelitis optica spectrum disorder (NMOSD) is neuroinflammatory disease characterized by optic neuritis and myelitis with presence of anti-aquaporin-4 (AQP4) antibodies. Satralizumab is an anti-IL-6 receptor (IL-6R) humanized antibody approved for preventing relapses in NMOSD (AQP4 IgG positive) in adults and children in Japan.