https://www.bioworld.com/rss en-us 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 Beijing Jingwei-Yankang Biomedical Co. Ltd. has reported new phenyl-substituted heterocyclic compounds acting as retinol-binding protein 4 (RBP4) inhibitors with potential in the treatment of age-related macular degeneration (AMD). http://www.bioworld.com/articles/732494 Wed, 08 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732494-beijing-jingwei-yankang-biomedical-divulges-new-rbp4-inhibitors 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 Researchers at Duke University have demonstrated that retinal endothelial cells (RECs) generated from human pluripotent stem cells can restore retinal vascularization in a preclinical model of ischemic retinal injury. http://www.bioworld.com/articles/732330 Wed, 01 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732330-lab-grown-retinal-endothelial-cells-repair-eye-vasculature Illustration demonstrating structure of the human eye and organization of retinal cells The oversubscribed $330 million series B round secured by Ollin Biosciences Inc. marks more than another large venture round in ophthalmology. It also highlights an emerging biotech financing model in which Chinese pharma companies discover and clinically validate promising drugs before handing global development to well-capitalized U.S. startups backed by blue-chip venture investors. http://www.bioworld.com/articles/732350 Tue, 30 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732350-ollins-330m-series-b-underscores-rise-of-china-origin-newco-model Close-up of a human eye featuring a dollar sign in the iris With the U.S. FDA go-ahead granted June 26 for Viridian Therapeutics Inc.’s IGF-1R antagonist Lumvoa (veligrotug-vvze, or “veli”) as a new thyroid eye disease (TED) therapy – due to launch immediately, the company said – Wall Street will be watching near-term payer dynamics. The drug is set to take on similarly targeted Tepezza (teprotumumab-trbw), owned by Amgen Inc. and approved in January 2020 to treat TED. http://www.bioworld.com/articles/732231 Mon, 29 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732231-new-viridian-meridian-hopes-veli-high-in-ted-post-fda-win Illustration of a road with three destination markers Viridian Therapeutics Inc.’s U.S. FDA clearance of Lumvoa (veligrotug-vvze) to treat thyroid eye disease (TED) includes labeling for chronic as well as active forms, and fewer infusions – plus fast, durable effects – should give the IGF-1R antagonist leverage in competing with similarly targeted TED drug Tepezza (teprotumumab-trbw), owned by Amgen Inc. and approved in January 2020. http://www.bioworld.com/articles/732326 Sun, 28 Jun 2026 00:00:00 -0400 https://www.bioworld.com/articles/732326-fda-says-yes-to-viridians-lumvoa-in-ted Close up of man's eye The oversubscribed $330 million series B round secured by Ollin Biosciences Inc. marks more than another large venture round in ophthalmology. It also highlights an emerging biotech financing model in which Chinese pharma companies discover and clinically validate promising drugs before handing global development to well-capitalized U.S. startups backed by blue-chip venture investors. http://www.bioworld.com/articles/732160 Thu, 25 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732160-ollins-330m-series-b-underscores-rise-of-china-origin-newco-model Close-up of a human eye featuring a dollar sign in the iris Lu-AG-22515, also known as velaprumig, is a recombinant fusion protein that targets CD40 ligand (CD40L) and human serum albumin (HSA), thereby blocking the binding between CD40 and CD40L, a known signaling pathway involved in several autoimmune diseases. Researchers at H. Lundbeck A/S evaluated the pharmacokinetic (PK) and pharmacodynamic (PD) profile of Lu-AG-22515 in cynomolgus monkeys, as well as the impact of its inhibitory effect in the preclinical setting of thyroid eye disease (TED). http://www.bioworld.com/articles/732119 Tue, 23 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732119-lundbecks-lu-ag-22515-shows-promise-in-thyroid-eye-disease Close up of eye and vision test 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 Retinitis pigmentosa (RP) is an inherited retinal dystrophy that causes loss of vision. Pathogenic variants in proteins involved in RNA splicing are the second most common cause of autosomal dominant RP, with mutations in PRPF31 being the most prevalent. Additionally, mutations in spliceosomal small nuclear RNAs (snRNAs) U4 and U6 have recently been linked to RP. PRPF31 being the most prevalent. Additionally, mutations in spliceosomal small nuclear RNAs (snRNAs) U4 and U6 have recently been linked to RP.]]> http://www.bioworld.com/articles/732034 Fri, 19 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732034-new-intronic-prpf31-mutation-causing-rp-can-be-targeted-with-aso Eye and DNA illustration Memento Medicines Inc. has announced its launch with a $93 million series A financing and the licensing of a lead program for retinal diseases. http://www.bioworld.com/articles/732033 Fri, 19 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732033-memento-medicines-launches-with-focus-on-ophthalmology Eye with blue graphic overlays 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 Fresh off a deal with Travere Therapeutics Inc. for civorebrutinib worth more than $1.14 billion, Everest Medicines Ltd. went on a shopping spree, striking three deals in six days to expand its pipeline and geographic reach across Asia Pacific. http://www.bioworld.com/articles/731982 Tue, 16 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731982-three-deals-in-six-days-everest-builds-late-stage-apac-portfolio Hands holding gears Ethyreal Bio Inc. has come out of stealth mode in order to disclose preclinical data for ETHY-001, its monoclonal antibody targeting thyroid stimulating hormone receptor, at the Endocrine Society’s 2026 annual meeting on June 15. http://www.bioworld.com/articles/731803 Thu, 11 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731803-ethyreal-launches-to-treat-the-underlying-cause-of-graves-ted Neck exam Ethyreal Bio Inc. has launched with $101 million in financing and a focus on developing precision therapies for thyroid diseases with high unmet need. http://www.bioworld.com/articles/731836 Thu, 11 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731836-ethyreal-bio-launches-with-focus-on-thyroid-diseases Fresh off a deal with Travere Therapeutics Inc. for civorebrutinib worth more than $1.14 billion, Everest Medicines Ltd. went on a shopping spree, striking three deals in six days to expand its pipeline and geographic reach across Asia Pacific. http://www.bioworld.com/articles/731721 Wed, 10 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731721-three-deals-in-six-days-everest-builds-late-stage-apac-portfolio Hands holding gears 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 Kunming Taita Biotech Co. Ltd. has patented polypeptides acting as angiogenesis inhibitors reported to be useful for the treatment of eye disorders. http://www.bioworld.com/articles/731790 Wed, 10 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731790-kunming-taita-biotech-discovers-new-angiogenesis-inhibitors 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 City Therapeutics Inc. has completed a $99.5 million series B financing to support its pipeline of RNA interference (RNAi) therapeutics and accelerate its next-generation RNAi engineering platform. http://www.bioworld.com/articles/731762 Tue, 09 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731762-city-therapeutics-advances-rnai-pipeline-with-series-b-financing Tavo Biotherapeutics Inc. has successfully closed a $17 million series A financing, with the proceeds directed to advancing the company’s pipeline of innovative therapies targeting glaucoma and retinal disease. http://www.bioworld.com/articles/731597 Wed, 03 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731597-tavo-biotherapeutics-closes-series-a-to-advance-ocular-pipeline Close-up of a human eye featuring a dollar sign in the iris The much-awaited phase III readout from Oculis Holding AG’s OCS-01 in diabetic macular edema (DME) fell short of expectations, delaying the possibility of a topical eye drop option for DME patients and prompting the company to shuffle its pipeline priorities, with a focus on late-stage programs targeting optic neuropathies and dry eye disease. http://www.bioworld.com/articles/731510 Mon, 01 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731510-oculis-shifts-focus-after-phase-iii-dme-disappointment Woman using eyedrops Daewoong Pharmaceutical Co. Ltd. announced after South Korean market hours May 21 that it gained successive rights to Turn Biotechnologies Inc.’s mRNA-based cellular rejuvenation platform from Hanall Biopharma Co. Ltd. http://www.bioworld.com/articles/731469 Tue, 26 May 2026 11:00:00 -0400 https://www.bioworld.com/articles/731469-daewoong-takes-over-turn-bios-longevity-platform-from-hanall DNA double helix with sand hour glass Merck Sharp & Dohme LLC (MSD) has reported new plasma kallikrein (KLKB1) inhibitors potentially useful for the treatment of diabetic macular edema, cancer, hereditary angioedema, uveitis, pancreatitis, arthritis, nephropathy and inflammatory disorders and among others. http://www.bioworld.com/articles/731283 Tue, 26 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731283-msd-divulges-new-klkb1-inhibitors Daewoong Pharmaceutical Co. Ltd. announced after South Korean market hours May 21 that it gained successive rights to Turn Biotechnologies Inc.’s mRNA-based cellular rejuvenation platform from Hanall Biopharma Co. Ltd. http://www.bioworld.com/articles/731389 Fri, 22 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731389-daewoong-takes-over-turn-bios-longevity-platform-from-hanall DNA double helix with sand hour glass There are real world demonstrations of how autonomous artificial intelligence agents are poised to disrupt biomedical research, according to two papers published May 19 in Nature. Each describes an AI system that assists across the piece, from generating hypotheses to designing experiments, analyzing the data and refining hypotheses in the light of new data. Nature. Each describes an AI system that assists across the piece, from generating hypotheses to designing experiments, analyzing the data and refining hypotheses in the light of new data.]]> http://www.bioworld.com/articles/731370 Tue, 19 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731370-artificial-intelligence-agents-get-ready-to-take-on-the-scientific-method AI Agent illustration There are real world demonstrations of how autonomous artificial intelligence agents are poised to disrupt biomedical research, according to two papers published May 19 in Nature. Each describes an AI system that assists across the piece, from generating hypotheses to designing experiments, analyzing the data and refining hypotheses in the light of new data. Nature. Each describes an AI system that assists across the piece, from generating hypotheses to designing experiments, analyzing the data and refining hypotheses in the light of new data.]]> http://www.bioworld.com/articles/731327 Tue, 19 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731327-artificial-intelligence-agents-get-ready-to-take-on-the-scientific-method AI Agent illustration Cell replacement therapy is currently being investigated to restore the retinal pigment epithelium (RPE) layer and treat vision decline due to dry age-related macular degeneration (AMD). The Neural Stem Cell Institute and collaborating institutions developed an approach to RPE replacement using an adult RPE stem cell source derived from the RPE layer of donated cadaver eyes. http://www.bioworld.com/articles/731142 Mon, 18 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731142-ind-enabling-studies-of-adult-rpe-stem-cell-product-for-dry-amd Senior eye exam 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