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This Week’s CGT News: Capsida Pauses Study After Patient Death

Our latest news roundup reports on biosensors for CAR T cell manufacturing, a new medium for gene therapy production, exosomes from stem cells, and more.

The Medicine Maker
Death in gene therapy trialThe first patient to participate in Capsida’s SYNRGY trial for gene therapy CAP-002 has died. The AAV vector therapy was being investigated in children with syntaxin-binding protein 1 (STXBP1) encephalopathy – which causes neurodevelopment issues and seizures. The trial reportedly began in July.A letter from Capsida said, “We have voluntarily paused the CAP-002 SYNRGY study while we determine the root cause of the patient’s passing. As we continue to work closely with our partners and relevant experts, we have alerted the FDA and will be providing them with a full report in compliance with regulations.”Biosensor for CAR TsSwRI and the University of Texas at San Antonio are collaborating to develop novel biosensor coatings that enable continuous, closed-loop monitoring and characterization of cells during CAR T cell manufacturing. The aim is to reduce reliance on manual, discrete measurements (such as flow cytometry), minimize human error, automate parts of the production process, and cut costs associated with immunotherapy manufacture.The coating technology builds on Quartz Crystal Microbalance principles, previously adapted for virus detection during the COVID-19 pandemic. The teams plan to integrate the biosensor coatings into existing bioreactor platforms used for propagating T-cells.Promising interim results for Canavan disease therapyMyrtelle has published interim results from a phase I/II trial of its gene therapy for Canavan disease – a fatal childhood brain disease. Preliminary results indicate the treatment is well tolerated with early evidence of therapeutic effect.rAAV-Olig001-ASPA (MYR-101) is a proprietary recombinant AAV vector that delivers a functional ASPA gene directly to oligodendrocytes, the brain cells responsible for producing myelin. In Canavan disease, these cells fail to produce the enzyme aspartoacylase, leading to disrupted myelination. As the disease progresses, children can experience seizures, spasticity, difficulties in swallowing, and overall muscle deterioration. By age 10, most patients have life-threatening complications.The investigational therapy aims to restore enzyme activity, enable proper N-acetylaspartate metabolism, and facilitate myelin repair.SourceNew medium for gene therapy production from FujifilmFujifilm Biosciences has launched BalanCD HEK293 Perfusion A, a cell culture medium developed to support gene therapy production using HEK293 suspension cells in perfusion processes. The medium is intended for use in the manufacture of viral vectors such as AAV and lentivirus, as well as in applications including transient protein expression and recombinant protein production. It has been formulated to sustain high cell density and intensified processes, and it is compatible with a range of cell retention devices and transfection methods. According to Fujifilm, the medium is designed to facilitate scalability and consistency in production while addressing the technical and economic demands of virus manufacturing.Tackling bladder cancer with CAR Ts and diabetes drugsResearchers at the University of California, San Francisco have combined a CAR T cell therapy with a drug originally designed for diabetes with the goal of developing a new approach to treat bladder cancer.Their work centered on NECTIN4, a surface protein already targeted by the antibody-drug conjugate enfortumab vedotin (EV). While EV has shown clinical benefit, many patients eventually relapse. The researchers engineered CAR T cells to target NECTIN4, but the potency was dependent on how much NECTIN4 the tumors expressed. To raise those levels, the team used rosiglitazone, a PPARγ agonist previously approved for diabetes. The drug primed tumors to express more NECTIN4, making them more susceptible to CAR T attack in lab experiments and mouse models.SourceExosomes from stem cellsIn a correspondence article, researchers have highlighted the potential role of stem cell-derived exosomes as an alternative to whole-cell therapy for heart failure. Existing pharmacological and mechanical interventions often failing to restore damaged cardiac tissue. Research over the past two decades has explored stem cell transplantation as a strategy for cardiac repair, but the approach can be associated with oncological risks and inconsistent therapeutic outcomes. The authors suggests that exosomes could offer a safer and more targeted route to cardiac regeneration.Mesenchymal stem cells have been a focus of regenerative medicine research, but the paper highlight growing evidence that much of the cardioprotective effect associated with MSCs is not because of direct differentiation but rather to the extracellular vesicles they release. These vesicles, typically 30–150 nanometers in size, are capable of transporting proteins, lipids, and nucleic acids to recipient cells, influencing both physiological and pathological processes.Source