Hangzhou Qihan Biotech Co. Ltd. has gained IND approval by the FDA for QT-019B, a universal, dual-target CAR T-cell therapy for refractory systemic lupus erythematosus (SLE). A phase I/II trial will be conducted in the U.S.

Researchers at the University of Minnesota and collaborating institutions have developed a promising stem cell-based therapy for the treatment of muscular dystrophies. The team has successfully created a novel myogenic progenitor cell product called Myopaxon, derived from human-induced pluripotent stem cells (iPSCs).

Rhabdomyosarcoma is the most frequent cancer affecting soft tissues in children, and existing therapies often fail to stop relapse or prolong survival. Researchers in Germany and the U.K. have developed a potential new therapy based on natural killer cells expressing a chimeric antigen receptor against epidermal growth factor receptor (EGFR-CAR).

Despite the increasing sophistication of anticancer therapies, many malignancies continue to evade treatment. T cells expressing chimeric antigen receptor (CAR) can effectively attack some tumors by recognizing antigens expressed on the tumor surface, but the therapy may prove ineffective if the target antigen is not abundant enough throughout the tumor.

Chimeric antigen receptor (CAR) T-cell therapy has been a game changer in the treatment of B-cell malignancies, although their manufacturing process is complex and needs lymphodepletion, thus limiting their use. Researchers from Capstan Therapeutics Inc. have recently published data regarding an in vivo engineering approach to generate CAR T cells using targeted lipid nanoparticles (LNPs) for mRNA delivery to specific T-cell populations for the treatment of cancer and B-cell-mediated autoimmune diseases.

Newco Avidicure NV arrived on the scene with a hefty $50 million in seed funding to advance novel antibody formats the company says will surpass the best qualities of first-generation antibodies, checkpoint inhibitors, T-cell engagers and antibody-drug conjugates.

Gene and cell therapies (GCTs) can target the kidney to treat congenital, acute or chronic diseases affecting this organ. However, its complex structure poses a challenge for these technologies. To be precise and effective in the long term, new approaches should circumvent the specificities of renal tissue, with novel methods of delivery and gene transfer to offer new therapeutic options for patients who lack them.

Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.

Onward Therapeutics SA’s subsidiary Emercell SAS has received investigational medicinal product dossier (IMPD) approval from the EMA to initiate a phase I trial of OT-C001, an allogeneic natural killer (NK) cell therapy, in combination with an anti-CD20 monoclonal antibody, rituximab.

Mesenchymal stem cells (MSCs) have long been recognized for their potential in cancer therapy. However, the effectiveness of MSCs in cancer treatment has been hampered by their limited tumor-homing ability and the heterogeneity of tissue-derived MSCs. To address these challenges, researchers have focused on induced pluripotent stem cell (iPSC)-derived MSCs, which offer improved homogeneity and expansion potential.