All Gene Therapy articles
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NewsNon-endocytic delivery bypasses endosomal barriers for gene therapy
A new review examines how physical, bioengineered and biomimetic delivery platforms that bypass conventional endocytic uptake pathways could transform the clinical development of gene therapies, mRNA treatments and protein-based medicines.
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NewsGene therapy restores key features in fragile X syndrome mouse model
Researchers at Cincinnati Children’s have demonstrated that AAV-mediated delivery of the human FMR1 gene can restore FMRP expression and reverse multiple disease-related features in a fragile X syndrome mouse model, offering a promising preclinical foundation for future clinical trials.
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NewsGene therapy shows durable correction of methylmalonic acidemia in preclinical study
Preclinical findings from Genespire and SR-TIGET suggest a single administration of a liver-directed lentiviral gene therapy could provide lifelong correction of methylmalonic acidemia, a rare inherited metabolic disorder with no approved disease-targeted treatments.
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ArticleFrom R&D to QC: building a single analytical strategy for cell and gene therapies
From early research to quality control, maintaining analytical continuity is no easy task. Could a single sequencing workflow help simplify analytical assessment?
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NewsBlocking cathepsin B protein enhances CAR T-cell therapy effectiveness
University of Maryland researchers have discovered that blocking cathepsin B protein prevents CAR T-cells from losing effectiveness, potentially improving long-term outcomes for blood cancer patients. The preclinical findings reveal that engineered immune cells inadvertently weaken themselves by acquiring tumour fragments, a process that can be prevented through targeted protein inhibition.
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NewsAI-designed proteins self-assemble into virus-like drug delivery structures
An international collaboration has developed AI-designed protein structures that self-assemble into virus-like nanocages using quasisymmetry principles, offering new possibilities for targeted drug delivery, vaccine development and genetic material transport without relying on modified viral proteins.
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NewsPERM1 protein linked to heart recovery in LVAD patients
A newly identified protein may explain why some failing hearts recover function following mechanical support while others do not.
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NewsCircio’s circular RNA platform shows 40-fold gene expression boost
Circio Holding has presented preclinical data at ASGCT demonstrating that its circVec circular RNA platform achieved up to 40-fold greater gene expression in cardiac tissue and 50-fold in ocular tissue compared with conventional AAV gene therapy approaches, potentially enabling significant dose reductions and improved safety profiles.
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NewsAI-designed viral vectors achieve 50-fold brain enrichment over AAV9
WhiteLab Genomics has presented preclinical data showing that viral vectors designed using artificial intelligence achieved approximately 50-fold higher DNA enrichment in the brain compared to AAV9, with no detectable liver signal following intravenous administration in mice.
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NewsASGCT 2026 Annual Meeting opens in Boston this week
The American Society of Gene & Cell Therapy’s 2026 Annual Meeting opens in Boston on 11 May, bringing together thousands of researchers, clinicians and industry leaders for five days of scientific programming.
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NewsCircio and Acuitas partner on circular RNA CAR T therapy
Norway-based biotechnology company Circio Holding ASA has partnered with Acuitas Therapeutics to evaluate its circVec circular RNA platform for in vivo CAR T cell therapy. The collaboration will combine Circio’s technology, which reportedly delivers gene expression lasting up to six months in lymphocytes, with Acuitas’ lipid nanoparticle delivery system targeting specific T cell populations for potential applications in oncology and autoimmune diseases.
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ArticleComputational design drives new generation of synthetic promoters
Designing gene control from scratch is becoming possible. SynGenSys is using computational design to create synthetic promoters for advanced therapies.
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NewsSimple amino acid mix dramatically improves gene therapy delivery
Scientists have discovered that adding a simple cocktail of three common amino acids to lipid nanoparticle injections can dramatically improve the delivery of mRNA and CRISPR therapies.
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ArticlePreclinical takeaways from the World ADC Conference London
Experts from the World ADC Conference in London highlight how patient-centric, predictive preclinical tools and innovative ADC designs are improving safety, efficacy and clinical translation.
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NewsCircular RNA drives 50-fold boost in eye gene therapy
New in vivo data show a circular RNA platform can deliver up to a 50-fold increase in gene expression in the eye compared with conventional mRNA-based AAV approaches.
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NewsNew SHANK3 conditional knockout mouse model launched for PMS research
A new SHANK3 conditional knockout mouse model from InnoSer, CureSHANK and Ozgene has been launched to advance research into Phelan-McDermid syndrome, other neurodevelopmental disorders and the development of new therapies.
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NewsELRIG announces keynote speakers for Cell and Gene Therapy 2026 in Cambridge
ELRIG (European Laboratory Research & Innovation Group) has announced the keynote speakers for its inaugural Cell and Gene Therapy 2026 conference, taking place at Hinxton Hall in Cambridge from 9–10 March.
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ArticleThe mother of invention: from steam engines to AI-designed drugs
Every great leap in history started with a single, urgent need. Now AI is emerging as the next great engine of invention, transforming the future of medicine faster than ever imagined.
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NewsNew stealth CRISPR method reduces immune interference in tumours
Researchers have developed a novel CRISPR method that evades the immune system in mice, allowing scientists to study tumour growth and metastasis more accurately.
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NewsCRISPR disruption of NRF2 re-sensitises tumours to chemo
Researchers have shown that using CRISPR to disable the NRF2 gene can restore chemotherapy sensitivity and slow tumour growth.