Research
REGENXBIO reports pivotal Duchenne data for AAV gene therapy
REGENXBIO has reported positive topline results from the pivotal phase III portion of AFFINITY DUCHENNE for RGX-202, an investigational one-time AAV microdystrophin gene therapy for Duchenne muscular dystrophy. The trial met its primary endpoint, with 93 percent of evaluable participants achieving at least 10 percent microdystrophin expression at week 12. Editing was sustained for at least three months after the last dose, with no serious adverse events or clinically meaningful liver test elevations reported. Source
Candel reports extended benefit for adenoviral prostate cancer immunotherapy
Candel Therapeutics has reported extended follow-up data from its phase 3 trial of aglatimagene besadenovec, also known as CAN-2409, an off-the-shelf, replication-defective adenoviral immunotherapy, in intermediate- to high-risk localized prostate cancer. After a median follow-up of 58 months, the therapy showed a statistically significant 39 percent improvement in prostate cancer-specific disease-free survival compared with placebo in patients receiving radiotherapy. The therapy is designed to deliver the HSV-tk gene directly into the tumor, where subsequent prodrug treatment triggers immunogenic cell death and stimulates an individualized anti-tumor immune response. Candel says it plans to submit a BLA to the FDA in Q4 2026. Source
Cabaletta reports preconditioning-free CAR-T data in pemphigus vulgaris
Cabaletta Bio has reported early clinical data for rese-cel, an investigational autologous CD19 CAR-T cell therapy, given without preconditioning in patients with refractory pemphigus vulgaris. In the lowest-dose cohort of RESET-PV, a single infusion produced drug-free clinical responses lasting six months in two of four patients, with all four showing initial improvement in disease activity scores. Source
JCR presents CNS-targeting AAV platform data at ASGCT
JCR Pharmaceuticals has presented preclinical data for JUST-AAV, its modified AAV vector platform designed to improve gene therapy delivery to the central nervous system and muscle while reducing liver tropism. The platform uses capsids engineered with miniaturized receptor-binding antibodies, including transferrin receptor-targeting approaches, to enhance tissue-specific delivery and potentially improve the safety and efficacy of AAV-based therapies. Across several preclinical studies, JCR and collaborators reported improved CNS uptake compared with conventional AAV9. Source
Scribe reports preclinical CRISPR platform data for cardiometabolic programs
Scribe Therapeutics has presented preclinical data for its engineered CRISPR technologies, including ELXR, a CRISPR-based epigenetic silencing modality, and XE, a CasX-derived genome-editing platform. The data support Scribe’s lead cardiometabolic program, STX-1150, a liver-targeted epigenetic silencing therapy designed to repress PCSK9 and lower LDL-C without making permanent DNA changes.
In preclinical studies, allosteric ELXR molecules improved on-target gene repression and reduced off-target transcriptional effects compared with non-allosteric designs, including sustained in vivo knockdown of PCSK9. Scribe also reported XE data showing potent liver editing across multiple target loci in non-human primates, supported by DeepXE, an AI model designed to predict potent guide RNAs and accelerate therapeutic design. Source
Armored CAR-T strategy takes aim at glioblastoma
A cytokine-armored CAR-T cell therapy developed at UCLA Health has shown preclinical promise against glioblastoma and other recurrent high-grade gliomas. The autologous cell therapy approach engineers CAR-T cells to recognize IL-13Rα2, a tumor antigen commonly found on glioblastoma cells, while secreting the immune-stimulating cytokines IL-12 and decoy-resistant IL-18 to recruit broader anti-tumor immune activity.
In mouse models, the IL-12/DR-18-armored CAR-T cells improved tumor control, including against heterogeneous tumors containing cancer cells that lacked the CAR-T target antigen. UCLA researchers are now completing preclinical work and seeking funding to support a planned phase 1 trial in patients with recurrent high-grade gliomas. Source
Mezigdomide gives exhausted T cells a second wind in myeloma
Mezigdomide could help restore the cancer-fighting activity of exhausted T cells in relapsed multiple myeloma, according to preclinical and translational studies from Mount Sinai, Bristol Myers Squibb, and the University of Oxford. The cereblon E3 ligase modulator and targeted protein degrader was shown to reduce exhausted T cell populations and enhance the tumor-killing activity of both CAR-T cells and bispecific T cell engagers across two companion studies in Blood.
The drug works by degrading the transcription factors IKZF1 and IKZF3, also known as Ikaros and Aiolos, which help maintain dysfunctional T cell states. Early-phase trials are now evaluating combinations of mezigdomide with T cell-based therapies, with the aim of improving response depth and durability in patients whose disease has returned after multiple lines of treatment. Source
DNA-guided CRISPR opens new route to RNA targeting
University of Florida researchers have developed a new CRISPR-based RNA-targeting technology that uses DNA guides instead of RNA guides to direct CRISPR enzymes to their targets. The preclinical platform is designed to act on RNA transcripts rather than permanently altering genomic DNA, offering a potentially more stable, precise, and lower-cost approach to RNA editing and molecular diagnostics.
The system was shown to reduce unintended effects compared with existing RNA-guided approaches, improving precision by orders of magnitude in some cases. Because DNA guides are more stable and easier to manufacture than RNA guides, the researchers say the technology could support applications ranging from early viral diagnostics to ex vivo therapeutic strategies, including potential use in cells, tissues, or donor organs before transplantation. Source
Business and Regulation
UK framework aims to strengthen advanced therapy trial delivery
Skills for Health has launched the Advanced Therapy Clinical Trials Capability Framework, a national workforce framework designed to support NHS delivery of advanced therapy clinical trials in the UK. The framework was developed for the Advanced Therapy Treatment Centre network as part of a wider initiative with NIHR, Innovate UK, Cell and Gene Therapy Catapult, and ATTC partners to strengthen the UK’s advanced therapy research environment. The framework defines the capabilities needed by clinical and non-clinical staff involved in advanced therapy trials, with the aim of standardizing training, helping trial sites work more effectively with sponsors, and improving patient access to cell, gene, and other advanced therapy trials across the UK. Source
FUJIFILM opens new iPSC manufacturing facility in Madison
FUJIFILM Cellular Dynamics has opened a new headquarters and iPSC development and manufacturing facility in Madison, Wisconsin, expanding capacity for induced pluripotent stem cell-based research products and development services. The 175,000-square-foot site is part of Fujifilm’s $200 million US investment in research products and cell therapy manufacturing, and is expected to quadruple the company’s iPSC-based manufacturing footprint. The facility includes cell culture manufacturing laboratories, process development laboratories, and a gene editing center of excellence, supporting research-grade iPSC products, iPSC-derived iCell product lines, and partner programs developing next-generation cell therapies. Source
CREATE raises $122 million to advance in vivo CAR pipeline
CREATE Medicines has closed a $122 million Series B financing to advance its in vivo immune programming pipeline across autoimmune disease and oncology. The company’s lead autoimmune program, CRT-402, is a repeat-dose-capable CD19-targeted in vivo CAR-T therapy delivered using an mRNA-LNP platform that engineers immune cells directly inside the body. Source
Children’s Mercy expands pediatric cell and gene therapy delivery
Children’s Mercy is expanding its pediatric cell and gene therapy program through a collaboration with Basepath Health, an AI-native operating platform for advanced therapies. The initiative is designed to help the health system deliver more in vivo gene therapies, ex vivo gene therapies, and CAR-T cell therapies to children and young adults with rare genetic disorders, aggressive cancers, and other complex diseases. Source
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