https://www.bioworld.com/rss en-us Apertura Gene Therapy LLC has announced its participation in a consortium selected to receive funding from the Advanced Research Projects Agency for Health (ARPA-H) THRIVE program to develop gene therapies for rare childhood diseases. http://www.bioworld.com/articles/732900 Fri, 24 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732900-apertura-gene-therapy-joins-thrive-consortium Brain and DNA The field of cell and gene therapy is moving toward a new phase of scalability and clinical durability, with in vivo chimeric antigen receptor (CAR) T-cell therapeutics emerging as the dark horse in what speakers at BIO Asia-Taiwan 2026 described as CAR T’s second revolution. http://www.bioworld.com/articles/732840 Tue, 21 Jul 2026 11:00:00 -0400 https://www.bioworld.com/articles/732840-to-viral-or-not-to-viral-in-vivo-car-t-debates-delivery-tech Red CAR T cell on blue blackground The field of cell and gene therapy is moving toward a new phase of scalability and clinical durability, with in vivo chimeric antigen receptor (CAR) T-cell therapeutics emerging as the dark horse in what speakers at BIO Asia-Taiwan 2026 described as CAR T’s second revolution. http://www.bioworld.com/articles/732839 Tue, 21 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732839-to-viral-or-not-to-viral-in-vivo-car-t-debates-delivery-tech Red CAR T cell on blue blackground The field of cell and gene therapy is moving toward a new phase of scalability and clinical durability, with in vivo chimeric antigen receptor (CAR) T-cell therapeutics emerging as the dark horse in what speakers at BIO Asia-Taiwan 2026 described as CAR T’s second revolution. http://www.bioworld.com/articles/732736 Mon, 20 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732736-to-viral-or-not-to-viral-in-vivo-car-t-debates-delivery-tech Red CAR T cell on blue blackground Researchers from the Universities of Bologna and Torino recently presented their hematopoietic stem cell gene therapy (HSC-GT) strategy based on microglia-mediated delivery using a lentiviral vector encoding a secretable, cell-penetrating CDKL5 protein (Igκ-TATk-CDKL5). http://www.bioworld.com/articles/732786 Mon, 20 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732786-cdkl5-gene-therapy-rescues-behavioral-cognitive-and-synaptic-deficits-in-cdd-mice Pediatric brain illustration At the Alzheimer’s Association International Conference, researchers from Voyager Therapeutics Inc. presented preclinical efficacy data for VY-1706, a blood-brain barrier-penetrant AAV9 gene therapy designed to reduce tau levels in models of Alzheimer’s disease (AD). http://www.bioworld.com/articles/732646 Wed, 15 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732646-voyagers-gene-therapy-reduces-tau-levels-in-ad-models Illustration of human brain and dna Apertura Gene Therapy has entered into a cooperative research and development agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Human Genome Research Institute. http://www.bioworld.com/articles/732549 Fri, 10 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732549-apertura-gene-therapy-enters-crada-for-niemann-pick-disease Atsena Therapeutics Inc. has selected a lead clinical candidate for ATSN-401, a gene therapy for Stargardt disease. ATSN-401 is now advancing in IND-enabling studies. http://www.bioworld.com/articles/732545 Fri, 10 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732545-atsena-selects-atsn-401-clinical-candidate-for-stargardt-disease Art concept for gene therapy The Advanced Research Projects Agency for Health (ARPA-H), an agency within the U.S. Department of Health and Human Services, has announced the teams for the THRIVE (Treating Hereditary Rare diseases with In Vivo prEcision genetic medicines) program. With a commitment of up to $160 million over 5 years, THRIVE aims to accelerate solutions for rare genetic pediatric diseases across multiple technological approaches, clinical trial designs and deployment models. http://www.bioworld.com/articles/732544 Fri, 10 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732544-arpa-h-program-supports-advances-for-rare-genetic-diseases Opus Genetics Inc. has secured U.S. FDA alignment on an eight-patient phase III trial of its lead gene therapy, OPGx-LCA5, for an ultra-rare form of inherited childhood blindness. http://www.bioworld.com/articles/732420 Tue, 07 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732420-opus-aligns-with-fda-on-rare-eye-disease-gene-therapy-trial Eye and DNA illustration The gene therapy specialist formerly known as EG427 has a new name and fresh momentum, after closing a €33 million (US$37.7 million) series C that will further advance clinical development of the lead program in chronic neuro-urology indications. http://www.bioworld.com/articles/732419 Tue, 07 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732419-eg427-raises-377m-series-c-for-hsv-gene-therapy Bladder Metachromatic leukodystrophy (MLD) is a rare inherited lysosomal storage disorder characterized by progressive neurodegeneration resulting from loss of arylsulfatase A (ARSA) activity. Researchers at Kazan Federal University reported preclinical efficacy data for a gene therapy candidate in a porcine model of MLD. http://www.bioworld.com/articles/732453 Tue, 07 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732453-aavolig001-arsa-shows-promise-in-preclinical-mld-study Art concept for gene therapy research With more and more approved products, cell and gene therapies (CGTs) are moving from the initial stronghold in academic medical centers, and these complex biologics are now available across a wider network of treatment centers. http://www.bioworld.com/articles/732272 Thu, 02 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732272-cell-and-gene-therapy-access-is-up-but-capacity-constraints-may-be-looming Stem cells and DNA MED-EL Elektromedizinische Geräte GmbH has acquired two gene therapy programs from Rescue Hearing Inc. for genetic hearing and balance disorders. http://www.bioworld.com/articles/732337 Wed, 01 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732337-med-el-acquires-gene-therapy-programs-from-rescue-hearing Hand cupping ear to illustration hearing loss Spinal cord traumatic injury can lead to loss of motor function and progressive development of muscle spasticity and rigidity. Researchers from the University of California San Diego and collaborating institutions investigated a novel gene-delivery-based antispasticity strategy. http://www.bioworld.com/articles/732312 Tue, 30 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732312-preclinical-study-of-subpial-aav-gad65-vgat-delivery-for-spinal-injury-induced-spasticity Digital spine concept art Neurodegenerative disorders such as Alzheimer’s disease (AD) and frontotemporal dementia are characterized by the accumulation of hyperphosphorylated tau protein, forming neurofibrillary tangles, ultimately leading to synaptic dysfunction and cognitive decline. http://www.bioworld.com/articles/732209 Mon, 29 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732209-ext1-targeted-aav-gene-therapy-inhibits-tau-propagation-in-model-of-tauopathy Silhouette of head and brain with DNA double helixes Credit: Jonathan Bailey, NHGR Merck KGaA has agreed to buy Bio-Techne Corp. for an enterprise value of approximately $11.3 billion, in a bid to strengthen its life sciences business. The company is offering $73 per share in cash, a 36% premium to Bio-Techne's one-month volume-weighted average trading price, and a 24% premium to the company’s previous close on June 24. The deal will expand Merck’s position in multi-omics, spatial biology, cell and gene therapy, precision diagnostics and advanced research tools. http://www.bioworld.com/articles/732157 Thu, 25 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732157-merck-to-buy-bio-techne-for-113b-to-boost-life-sciences-portfolio Lab glassware and scientist Eli Lilly and Co. posted $19.8 billion in first-quarter 2026 revenue, driven by tirzepatide, marketed as Mounjaro for type 2 diabetes and Zepbound for obesity. The Indianapolis-based drugmaker is channeling cash flow from these blockbuster GLP-1 drugs into an aggressive dealmaking campaign — about $25.1 billion across 10 announced acquisitions so far this year and more than $26 billion in other closed deals. http://www.bioworld.com/articles/732076 Wed, 24 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732076-lillys-glp-1-windfall-fuels-m-and-a-land-grab-across-10-deals-25b Business handshake and holographic dollar sign Deficiencies of the enzyme β-N-acetylhexosaminidase (Hex) cause rare, autosomal recessive, fatal, neurodegenerative lysosomal storage disorders called GM2 gangliosidoses, including Tay-Sachs disease (TSD) and Sandhoff disease. Hex enzyme is a heterodimer encoded by HEXA (α subunit) and HEXB (β subunit), whose mutations result in TSD and Sandhoff disease, respectively. N-acetylhexosaminidase (Hex) cause rare, autosomal recessive, fatal, neurodegenerative lysosomal storage disorders called GM2 gangliosidoses, including Tay-Sachs disease (TSD) and Sandhoff disease. Hex enzyme is a heterodimer encoded by HEXA (α subunit) and HEXB (β subunit), whose mutations result in TSD and Sandhoff disease, respectively.]]> http://www.bioworld.com/articles/732109 Tue, 23 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732109-csf-delivered-aav-based-gene-therapy-rescues-ocular-symptoms-in-model-of-tay-sachs-disease 3D rendering of adeno-associated viral vector Another day, another about-face by the U.S. FDA on Uniqure NV’s Huntington’s disease gene therapy. But this latest shift brings good news for the company’s AMT-130, for which the FDA says three-year analysis data from the phase I/II study will be acceptable for an accelerated BLA filing, now expected to be submitted in the third quarter. http://www.bioworld.com/articles/731903 Wed, 17 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731903-flexibility-restored-uniqure-plans-for-bla-filing-in-huntingtons Genetic mutation illustration Opus Genetics Inc. is advancing a pipeline of gene therapies to restore vision and prevent blindness in patients with inherited retinal diseases, with three programs expected to enter clinical testing over the next 12-18 months. http://www.bioworld.com/articles/731993 Wed, 17 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731993-opus-genetics-advances-ocular-gene-therapies-toward-clinic Eye, DNA double helix illustration. Credit: Ernesto del Aguila III, National Human Genome Research Institute, NIH Autism spectrum disorder (ASD), developmental epileptic encephalopathies and other neurodevelopmental disorders are driven by the disruption of genes regulating neuronal proliferation, differentiation and synaptic maturation. Researchers from Shanghai Jiao Tong University School of Medicine generated Csnk2b haploinsufficient (Csnk2b+/-) mice mimicking the most relevant disease features to investigate the effects of reduced gene dosage. Csnk2b haploinsufficient (Csnk2b+/-) mice mimicking the most relevant disease features to investigate the effects of reduced gene dosage.]]> http://www.bioworld.com/articles/731935 Mon, 15 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731935-gene-replacement-rescues-autism-related-phenotypes-in-mice Brain with puzzle piece removed Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional CRISPR/Cas9 editing. http://www.bioworld.com/articles/731851 Wed, 10 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731851-columbia-researchers-use-base-editing-to-modify-human-embryo-genome DNA double helix under a magnifying glass Sensorion SA has selected SENS-601 (GJB2-GT) as its lead program and has filed clinical trial applications to study its use for GJB2-related hearing loss. SENS-601 is an AAV-based gene therapy program, utilizing a gene therapy platform codeveloped with Institut Pasteur. http://www.bioworld.com/articles/731777 Wed, 10 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731777-sensorion-selects-sens-601-as-lead-program-for-hearing-loss 3D illustration showing DNA inside adenovirus Scientists at Columbia University have used base editing to make precise changes in the genomes of human embryos, avoiding the damage to chromosomes that occurs following two-stranded DNA cuts with conventional Crispr-Cas9 editing. http://www.bioworld.com/articles/731708 Tue, 09 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731708-columbia-researchers-use-base-editing-to-modify-human-embryo-genome DNA double helix under a magnifying glass Idefine, The Kleefstra Syndrome Foundation, has established a collaboration with UT Southwestern Medical Center to advance development of a potential gene therapy to treat Kleefstra syndrome, a rare neurodevelopmental disorder caused by changes or loss of the EHMT1 gene, which plays a critical role in brain development and function. EHMT1 gene, which plays a critical role in brain development and function.]]> http://www.bioworld.com/articles/731656 Thu, 04 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731656-idefine-announces-kleefstra-syndrome-gene-therapy-collaboration DNA illustration Rather than reinventing the wheel for every gene therapy that uses genome editing, the U.S. FDA is advising sponsors on leveraging existing knowledge, be it publicly available or platform-based, to more efficiently advance their products across multiple stages of development. http://www.bioworld.com/articles/731529 Tue, 02 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731529-fda-advises-leveraging-whats-already-known-in-gene-therapy-r-and-d DNA double helix illustration with section being removed in red Voyager Therapeutics Inc. has obtained IND clearance from the FDA for VY-1706, the company’s investigational gene therapy for the treatment of Alzheimer’s disease. http://www.bioworld.com/articles/731578 Tue, 02 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731578-voyagers-tau-targeted-gene-therapy-vy-1706-gains-ind-approval Tau neuron illustration Tau protein accumulating on neuron. Credit: Kenneth S. Kosik, University of California - Santa Barbara X-linked Alport syndrome is an inherited kidney disease caused by pathogenic mutations in the COL4A5 gene. Patients develop hematuria, proteinuria and kidney function decline leading to end-stage renal disease. Nionyx Bio Inc. has developed ONYX-101, a novel kidney-targeting therapeutic designed to ensure durable COL4A5 restoration through dual-vector AAV delivery using NYX capsids that were optimized for kidney targeting. COL4A5 gene. Patients develop hematuria, proteinuria and kidney function decline leading to end-stage renal disease. Nionyx Bio Inc. has developed ONYX-101, a novel kidney-targeting therapeutic designed to ensure durable COL4A5 restoration through dual-vector AAV delivery using NYX capsids that were optimized for kidney targeting.]]> http://www.bioworld.com/articles/731296 Wed, 27 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731296-onyx-101-restores-col4a5-expression-in-x-linked-alport-syndrome Illustration of kidneys with DNA double helix Circio Holding ASA and Aavigen GmbH have entered a research collaboration focused on developing circVec-enhanced AAV vectors engineered for targeted, low-dose gene therapies for heart diseases. http://www.bioworld.com/articles/731292 Wed, 27 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731292-circio-and-aavigen-to-collaborate-on-targeted-cardiac-aavs Heart and DNA