South America and Mexico’s Cell and Gene Therapy Landscape: Accelerating Growth and Persistent Challenges

FDA-regulated research: cellular therapy considerations

Cancer is one of the world's most concerning health problems and poses many challenges in the range of approaches associated with the treatment of cancer. Current understanding of this disease brings to the fore a number of novel therapies that can be useful in the treatment of cancer. Among them, gene and cell therapies have emerged as novel and effective approaches. One of the most important challenges for cancer gene and cell therapies is correct monitoring of the modified genes and cells. In fact, visual tracking of therapeutic cells, immune cells, stem cells and genetic vectors that contain therapeutic genes and the various drugs is important in cancer therapy. Similarly, molecular imaging, such as nanosystems, fluorescence, bioluminescence, positron emission tomography, single photon-emission computed tomography and magnetic resonance imaging, have also been found to be powerful tools in monitoring cancer patients who have received therapeutic cell and gene therapies or drug therapies. In this review, we focus on these therapies and their molecular imaging techniques in treating and monitoring the progress of the therapies on various types of cancer.

Optogenetics: Controlling Cardiac Depolarization and Hyperpolarization Using Combined Cell and Gene Therapy and Light-Sensitive Proteins

Introducing a New ASGCT Forum for Publication of Methodology and Clinical Development in Gene, Cell, and Oligonucleotide Therapies

We read with great interest the meta-analysis by Mudgal et al. (2024) regarding the effectiveness and safety assessment of stem cell therapy for diabetic foot ulcers. Indeed, The management of chronic wounds requires innovative approaches to avoid unsuccessful outcomes, and stromal cell therapies have emerged as a potential solution for soft tissue repair. A critical aspect of this therapeutic strategy is the role of mast cells in stimulating delayed inflammation through their interactions with various cells as well as the extracellular matrix. Mast cells are critical in orchestrating the inflammatory response and their activation can influence macrophage behavior and secondary healing efficacy. The use of mesenchymal stromal cells in regenerative medicine for diabetic foot ulcers treatment is often limited by their time-limited anti-inflammatory responses. However, these time-limited effects could not achieve the prolonged effects and impact of cell therapy efficacy and the potential enhancement of cell function by biologically active factors such as growth factors or gene therapeutics for prolonged release. The integration of cell and prolonged release gene therapeutics is a promising approach that goes beyond regenerative medicine by preventing secondary inflammatory complications. While mesenchymal stromal cells have shown promising results in experimental and clinical studies, there are limitations to their efficacy in regenerative medicine for diabetic foot ulcers. These limitations include the heterogeneity of cell populations used in the studies, the difficulty in determining the contribution of cells when used in combination with materials, the lack of data on optimal cell numbers for tissue repair, the effect of culture conditions on cell therapy efficacy, and the potential enhancement of cell efficacy by the use of additional biologics such as growth factors or gene therapeutics. The combination of cell and gene therapy is seen as a promising approach that goes beyond regenerative medicine into the field of molecular surgery of chronic wounds.

Gene and Cell Therapy Funding Opportunities in Horizon 2020: An Overview for 2014–2015

Expanded Opportunities for Methods and Clinical Development

Knowing thyself: an audit of cell therapy manufacturing in Australia and New Zealand

After an injury, axons in the central nervous system do not regenerate over large distances and permanently lose their connections to the brain. Two promising approaches to correct this condition are cell and gene therapies. In the present work, we evaluated the neuroprotective and neuroregenerative potential of pigment epithelium-derived factor (PEDF) gene therapy alone and combined with human mesenchymal stem cell (hMSC) therapy after optic nerve injury by analysis of retinal ganglion cell survival and axonal outgrowth. Overexpression of PEDF by intravitreal delivery of AAV2 vector significantly increased Tuj1-positive cells survival and modulated FGF-2, IL-1ß, Iba-1, and GFAP immunostaining in the ganglion cell layer (GCL) at 4 weeks after optic nerve crush, although it could not promote axonal outgrowth. The combination of AAV2.PEDF and hMSC therapy showed a higher number of Tuj1-positive cells and a pronounced axonal outgrowth than unimodal therapy after optic nerve crush. In summary, our results highlight a synergistic effect of combined gene and cell therapy relevant for future therapeutic interventions regarding optic nerve injury.

BEST1: the Best Target for Gene and Cell Therapies

Viral vector systems are crucial in delivering therapeutic genetic material for gene and cell therapy in clinical settings. With new progress in viral vector-based gene therapies, healthcare professionals and pharmacists must stay informed and apply appropriate biosafety precautions when handling these products. Comprehensive guidelines covering biosafety measures for every viral vector-based gene therapy, including storage, preparation, administration, and disposal, remain scarce. As viral vector-based gene therapy advances rapidly, it is critical to equip healthcare professionals with the expertise to safely manage these agents in a clinical setting. As gene therapies become more integrated into mainstream medicine, with 43 cell and gene therapy products approved as of 2025, pharmacists, nurses, and caregivers face new safety challenges when handling these therapies. While guidelines such as United States Pharmacopeia (USP) 797 and USP 800 provide a foundation for handling viral vector-based gene therapies, no single resource consolidates all biosafety considerations for healthcare professionals. This review provides an overview of the viral vector-based gene therapy landscape. It compiles best practices from various sources to establish a potential standardized approach to guide safe handling procedures for viral vector systems in a healthcare setting. By adopting the biosafety strategies outlined in this article, healthcare staff and pharmacists can safely manage viral vectors while developing standard operating procedures tailored to their specific clinical environments. Implementing these measures will support the continued advancement of gene therapy while ensuring the safety of healthcare providers, patients, and the environment.

Despite significant advances in myocardial revascularization and reperfusion, coronary artery disease and subsequently myocardial infarction are the leading cause of morbidity and mortality in the United States. Thus, one of the main goals in the treatment of myocardial ischemia is the development of effective therapy for angiogenesis. The first evidence we found is the demonstration of alleviation of myocardial ischemia and increased number of collateral blood vessels in the early 1990s following intracoronary administration of basic fibroblast growth factor protein in dog. Multiple animal studies, including ours, and a small number of human studies have confirmed the concept of stimulation of collateral development by pharmacological and molecular means. This includes direct delivery of growth factors into the ischemic target tissues, or of genes that encode for synthesis of growth factors by target tissues. Both cell therapy and gene therapy have proven to be effective in promoting neovascularization in various animal models. Although cell therapy alone is proven to be beneficial, the combination of cell and gene therapy may enhance therapeutic neovascularization. Thus, clinically relevant, combined strategy could be an excellent strategy for treating patients with myocardial infarction.

Development of a Web Course on Gene Therapy by the International Consortium of Gene Therapy

Cell therapy clinical trials pose a variety of complex challenges. Logistics with cell harvesting, manufacturing, shipments back to sites, patient safety, changing standard-of-care treatments, and patient enrolment due to competing trials can all impact study timelines. In this episode, Vito Romita and Jai Balkissoon outline key obstacles for developing cell therapies in oncology, and provide their insights on overcoming them in order to increase patient access and design safer trials.Abi Pinchbeck, Assistant Editor, BioInsights, speaks to (pictured above from left to right) Jai Balkissoon, MD, FACS, Vice President, Medical and Scientific Lead, Immuno-Oncology, Cell and Gene Therapy, Head of Immuno-Oncology, Cell and Gene Therapy Center of Excellence, Clinical Research Group, part of Thermo Fisher Scientific and Vito Romita, Senior Director, Global Project Management Project Delivery – Hematology Oncology Therapeutic Unit, Clinical Research, PPD, part of Thermo Fisher Scientific

David McCall, Editor, Cell & Gene Therapy Insights, talks to Aditi Singh, Sony BiotechnologyDr Aditi Singh is currently working as Global Senior Product Manager at Sony Biotechnology leading the launch strategy and management of instruments utilized in cell and gene therapy manufacturing workflow. Her goal through this role is to improve patient care by making personalized medicine (cell therapy) more accessible to patients. Aditi has a PhD degree in Cell and Molecular Biology from the University of Heidelberg, Germany and post-doctoral experience from the University of California, San Diego where she worked in the fields of infectious diseases and mRNA biology.