https://www.bioworld.com/rss en-us 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 New regulations tighten regulatory oversight of China’s investigator-initiated trials (IITs) but legitimize the pathway that will be open to other modalities beyond cell and gene therapies. http://www.bioworld.com/articles/731468 Tue, 26 May 2026 11:00:00 -0400 https://www.bioworld.com/articles/731468-chinas-new-iit-rules-expected-to-wipe-out-gray-market Stem cells AAV-based therapies for Duchenne muscular dystrophy (DMD) have shown efficacy, but have limitations such as poor delivery to target tissues and toxicity associated with the vector. Gemma Biotherapeutics Inc. has developed a gene therapy candidate, GB-703, which uses a new myotropic, integrin-binding AAV capsid containing a codon-optimized, deimmunized hybrid payload. http://www.bioworld.com/articles/731275 Tue, 26 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731275-gemma-biotherapeutics-gb-703-shows-promise-for-dmd Illustration of DNA double helix and motorized wheel chair New regulations tighten regulatory oversight of China’s investigator-initiated trials (IITs) but legitimize the pathway that will be open to other modalities beyond cell and gene therapies. http://www.bioworld.com/articles/731388 Fri, 22 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731388-chinas-new-iit-rules-expected-to-wipe-out-gray-market Stem cells Defects in antigen presentation lead to resistance to cancer immunotherapy, where type I conventional dendritic cells (cDC1s) are crucial drivers of antitumor immunity and their presence is tied to favorable responses and better outcomes. Intratumoral delivery of adenoviral vector, Ad5-PIB, encoding PU.1, IRF8 and BATF3 reprograms tumor cells into cDC1-like antigen-presenting cells and has shown synergy with immune checkpoint blockade (ICB) therapy at exerting antitumor immunity. Asgard Therapeutics AB has developed AT-108, a lead candidate developed for durable efficacy. http://www.bioworld.com/articles/731255 Fri, 22 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731255-asgards-at-108-shows-promise-as-immunotherapeutic Dendritic cells Sangamo Therapeutics Inc. discussed gene regulation approaches for neurodegenerative diseases when presenting findings on their clinical candidate ST-506 for the treatment of prion disease. http://www.bioworld.com/articles/731252 Fri, 22 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731252-sangamo-presents-primate-data-for-prion-suppressor-st-506 Brain and DNA Researchers at the University of London and collaborating institutions have developed a gene and cell therapy approach that enables sustained systemic frataxin protein delivery, improving motor performance and tissue pathology, and supporting a promising translational strategy for long-term disease stabilization in Friedreich’s ataxia patients. http://www.bioworld.com/articles/731178 Wed, 20 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731178-hspcs-delivering-tissue-penetrating-frataxin-ameliorate-friedreichs-ataxia-symptoms Stem cells A new strategy aims to improve gene therapy for Pompe disease by optimizing both the genetic component that restores the function of a deficient lysosomal enzyme and the vector that delivers it to the target tissue while avoiding the liver. The findings suggest that combining an optimized transgene with a targeted capsid could significantly enhance the effectiveness of gene therapy for Pompe disease. http://www.bioworld.com/articles/731174 Wed, 20 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731174-detargeted-targeted-gene-therapy-improves-activity-in-pompe Acid alpha-glucosidase molecular structure isolated on black Type 2 diabetes is marked by insulin resistance coupled with insufficient insulin secretion due to early β-cell dysfunction and progressive loss of β-cell mass. Pdx1 and MafA, critical for maintaining β-cell function, are progressively reduced under metabolic stress and in patients, driving disease progression. Researchers at the University of Pittsburgh have reported efficacy data demonstrating successful pancreatic delivery of GPX-002, an AAV-Pdx1/MafA construct, in HFD mice. http://www.bioworld.com/articles/731140 Mon, 18 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731140-gpx-002-restores-cell-function-in-a-diabetes-model Pancreas anatomy illustration The American Society of Gene & Cell Therapy (ASGCT) and Orphan Therapeutics Accelerator (OTXL) have announced the public launch of CGTxchange, an AI-enhanced clearinghouse and marketplace built to help reactivate cell and gene therapy programs that have been shelved despite strong scientific and clinical evidence. http://www.bioworld.com/articles/731124 Fri, 15 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731124-launch-of-cgtxchange-to-reactivate-cell-and-gene-therapy-programs Dimly lit archive room filled with cardboard storage boxes Directed evolution has become a central pillar in gene therapy. This engineering strategy enables the generation of more efficient variants of genetic editors and delivery vectors. Molecular diversification methods are increasingly sophisticated and are now accelerated by machine learning and AI tools, as showcased at the 29th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) held in Boston this week. http://www.bioworld.com/articles/731119 Fri, 15 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731119-asgct-2026-directed-evolution-in-gene-therapy DNA and genome editing illustration How the U.S. FDA might view the latest Duchenne muscular dystrophy (DMD) phase III data with gene therapy RGX-202 became the question for Regenxbio Inc., shares of which (NASDAQ:RGNX) closed May 14 at $6.25, down 38%, or $3.80, after the results were made public. http://www.bioworld.com/articles/731196 Thu, 14 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731196-will-fda-show-affinity-for-fast-yes-to-regenxbio-in-dmd Green arrow up red arrow down Chengdu Origen Biotechnology Co. Ltd. and Vanotech Ltd. have announced IND clearance by the FDA for KHN-921 for the treatment of hypertrophic cardiomyopathy (HCM) associated with MYBPC3 mutations. MYBPC3 mutations.]]> http://www.bioworld.com/articles/731095 Thu, 14 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731095-khn-921-gains-ind-clearance-for-emmybpc3-em-associated-hcm Abstract blue human heart with red cardio pulse line and red circle