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.
Bio Japan 2025 was abuzz with the news that Japan has once again snagged the Nobel Prize in Physiology or Medicine, with Shimon Sakaguchi winning for his discovery of a subtype of CD4-expressing T cells that affect the immune response. Earlier today, Susumu Kitagawa snatched the second Nobel Prize for Japan, this time in chemistry. Both Nobel Prize winners were professors at Kyoto University.
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.
As the many challenges facing cell therapies are being addressed, the CAR T field continues to evolve beyond its original design of T cells engineered to target hematological malignancies. During the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, several studies showed how this technology is being redefined as programmable and adaptable immune cells with expanded functional versatility.
Chinese researchers have published preclinical data regarding the potential of never in mitosis A (NIMA)-related kinase 2 (NEK2) as a therapeutic target in multiple sclerosis (MS), an inflammatory demyelinating disease of the central nervous system (CNS) in which B cells play a key role.
Researchers from Onco3r Therapeutics BV presented preclinical data on O3R-5671, a novel salt-inducible kinase 3 (SIK3) inhibitor developed for the treatment of inflammatory bowel disease and other autoimmune diseases.
Recent evidence in atopic dermatitis (AD) points to the involvement of additional pro-inflammatory pathways besides core Th2 responses. Current therapeutic approaches that work target mostly the IL-4/IL-13 pathway, but the duration of response and depth could be improved.
At the recent European Respiratory Society meeting, researchers from 35Pharma Inc. presented data on HS-235, an activin receptor inhibitor designed to neutralize activins and growth differentiation factors (GDFs). Disorders such as heart failure (HF) and pulmonary hypertension (PH) are associated with dysregulated activin and GDFs without neutralizing bone morphogenetic proteins such as BMP-9 and BMP-10, which play key roles in lymphatic and vascular homeostasis.
Idiopathic pulmonary fibrosis (IPF) is a rapidly progressing lung disease with high unmet needs and characterized by an excess in matrix deposition that leads to severe decline in lung functioning, where arginase expression and arginine metabolism seem to be involved and could be a promising target. Astrazeneca has presented data regarding their arginase inhibitor, AZD-8965, for the potential treatment of IPF.
RSS
