https://www.bioworld.com/rss en-us 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 Researchers from Beacon Therapeutics Holdings Ltd. reported the development of BTX-001, an intravitreal gene therapy designed to target the complement pathway through delivery of a C5 inhibitor. http://www.bioworld.com/articles/731075 Wed, 13 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731075-btx-001-enables-single-dose-treatment-for-geographic-atrophy Eye and DNA Curacle Co. Ltd. inked a potential $1.07 billion deal with U.S.-based “newco” Memento Medicines on May 10, granting the latter exclusive global rights to develop and commercialize preclinical TIE2/VEGF-directed bispecific antibody MT-103 for retinal disorders. http://www.bioworld.com/articles/731224 Tue, 12 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731224-curacle-memento-ink-1b-deal-for-ocular-tie2-vegf-bispecific Eye, currency symbols and globe Curacle Co. Ltd. inked a potential $1.07 billion deal with U.S.-based “newco” Memento Medicines on May 10, granting the latter exclusive global rights to develop and commercialize preclinical TIE2/VEGF-directed bispecific antibody MT-103 for retinal disorders. http://www.bioworld.com/articles/731193 Tue, 12 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731193-curacle-memento-ink-1b-deal-for-ocular-tie2-vegf-bispecific Eye, currency symbols and globe Researchers at Opus Genetics Inc. reported the efficacy of OPGx-BEST1, an AVV-based gene therapy developed to deliver a functional BEST1 transgene to retinal pigment epithelium (RPE) cells to re-establish normal BEST1 expression and activity. http://www.bioworld.com/articles/731014 Tue, 12 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731014-opgx-best1-restores-best1-expression-in-retinal-cells Test tube, dropper, DNA illustration Curacle Co. Ltd. inked a potential $1.07 billion deal with U.S.-based “newco” Memento Medicines on May 10, granting the latter exclusive global rights to develop and commercialize preclinical TIE2/VEGF-directed bispecific antibody MT-103 for retinal disorders. http://www.bioworld.com/articles/731030 Mon, 11 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731030-curacle-memento-ink-1b-deal-for-ocular-tie2-vegf-bispecific Eye, currency symbols and globe In its first substantial M&A transaction since 2021, Bayer AG is buying Perfuse Therapeutics Inc., a firm that has operated quietly, though it reported positive midstage data last year for PER-001, an endothelin antagonist, in glaucoma and diabetic retinopathy. http://www.bioworld.com/articles/730862 Wed, 06 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/730862-bayer-buying-perfuse-for-300m-up-front-in-potential-245b-deal Eye and financial charts The classic origin story for a biotech startup is that of a scientist who nurtures his work out of a university and to commercial success. For Link Biologics Ltd. and its TSG6-based pipeline, the story is the other way around; it began with now-CEO Reuben Dawkins meeting University of Manchester scientists Tony Day and Caroline Milner while he was on the lookout for “great science that needs help to make it to patients.” The three are now co-founders of Link, which spun out of the University of Manchester in 2021 and has four programs in three indications, all based on TSG-6 biology. http://www.bioworld.com/articles/730902 Wed, 06 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/730902-eyes-are-first-prize-for-newco-link-biologics Rays of light beaming from eye Top-line data from the Reveal-2 phase III trial of elegrobart to treat chronic thyroid eye disease (TED) drove Viridian Therapeutics Inc.’s stock up by 33.6%, positioning the company for a BLA filing in the first quarter of 2027. The trial met both primary endpoints at week 24: proptosis responder rate, as measured by exophthalmometry, required for a filing with the U.S. FDA; and the overall responder rate, required for an EMA filing. Doses every four weeks or every eight weeks achieved 50% and 54% PRR, respectively, compared to 15% for placebo. The doses also achieved 47% and 54% ORR vs. 15% for placebo. ORR included those with both proptosis and Clinical Activity Score response, defined as no worsening from baseline in the study eye, without deterioration in the fellow eye. http://www.bioworld.com/articles/730847 Tue, 05 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/730847-viridians-reveal-2-beats-expectations-with-elegrobart-in-ted Eye with digital overlay Sun Pharmaceuticals Industries Ltd. will pay $11.75 billion cash to acquire Organon & Co. The transaction is expected to close in early 2027. The addition of Organon’s women’s health products, legacy drug brands and biosimilars are expected to boost Mumbai, India-based Sun’s global reach. The all-cash buy is also expected to relieve Jersey City, N.J.-based Organon of an $8.6 billion debt pile, accumulated after its debt-laden 2021 spinout from Merck & Co. Inc. http://www.bioworld.com/articles/730787 Tue, 28 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730787-sun-pharma-to-acquire-merck-spinoff-organon-for-1175b Three red darts on target Sun Pharmaceuticals Industries Ltd. will pay $11.75 billion cash to acquire Organon & Co. The transaction is expected to close in early 2027. The addition of Organon’s women’s health products, legacy drug brands and biosimilars are expected to boost Mumbai, India-based Sun’s global reach. The all-cash buy is also expected to relieve Jersey City, N.J.-based Organon of an $8.6 billion debt pile, accumulated after its debt-laden 2021 spinout from Merck & Co. Inc. http://www.bioworld.com/articles/730643 Mon, 27 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730643-sun-pharma-to-acquire-merck-spinoff-organon-for-1175b Three red darts on target