All Gene Therapy articles
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ArticleWhy the blood–brain barrier blocks most drugs – and how ultrasound could help
The blood–brain barrier protects the brain from harmful substances, but it also prevents many medicines from reaching their target. Researchers are investigating whether focused ultrasound could safely improve drug delivery.
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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.