https://www.bioworld.com/rss en-us Edgewise Therapeutics Inc. is pulling in $1.55 billion up front by selling its muscular dystrophy business, including its fast skeletal myosin inhibitor, sevasemten (EDG-5506), to Servier SA. The deal is potentially worth up to $2.65 billion when including a potential $1.1 billion in milestone payments. http://www.bioworld.com/articles/731512 Mon, 01 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/731512-edgewise-sells-muscular-dystrophy-assets-to-servier-for-265b Purple pie chart 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 Showing a significant efficacy signal in a phase II trial, Relay Therapeutics Inc.’s zovegalisib (RLY-2608) achieved a 60% volumetric response in patients with PIK3CA-driven vascular anomalies (VAs). The isoform-selective PI3Ka inhibitor is in late-stage clinical trials with various combinations for P13Ka-mutated, HR+/HER2- advanced breast cancer, with VAs representing a second indication for which Leerink Partners analyst Andrew Berens forecasts $2.8 billion in peak revenues. http://www.bioworld.com/articles/731325 Tue, 19 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731325-relays-zovegalisib-hits-in-phase-ii-vascular-anomalies-trial Blood vessels, veins and arteries 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 Star Sports Medicine Co. Ltd. debuted on the Hong Kong Stock Exchange with an HK$829.55 million (US$105.86 million) IPO May 5, with shares closing about 118% higher on the first day. http://www.bioworld.com/articles/731227 Tue, 12 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/731227-china-med-tech-raises-star-sports-105m-ipo-cofoe-nabs-133m ​Star Sports Medicine HKEX IPO ceremony Star Sports Medicine's IPO ceremony on May 5. Credit: HKEX; x.com – @HKEXGroup At the European Congress of Endocrinology in Prague, researchers from Juvena Therapeutics Inc. presented the effects of JUV-161, a fusion protein consisting of human insulin-like growth factor 2 linked to human serum albumin, in preclinical models of myotonic dystrophy type 1 (DM1) and sarcopenia. http://www.bioworld.com/articles/731013 Tue, 12 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/731013-juv-161-reverses-structural-and-functional-muscle-decline Illustration of muscle anatomy Researchers from Rottapharm Biotech Srl have reported preclinical efficacy data on CR-10049, a long-acting osteoarthritis-targeted multikinase inhibitor, designed to treat both osteoarthritis pain and inflammatory joint degeneration. http://www.bioworld.com/articles/730997 Mon, 11 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/730997-first-long-acting-intra-articular-multikinase-inhibitor-for-oa X-ray image of hand and wrist A lower-than-expected increase in dystrophin over baseline in the first and lowest-dose cohort of a phase I/II study of ENTR-601-44 in Duchenne muscular dystrophy (DMD) caused shares of Entrada Therapeutics Inc. to plunge more than 57%, despite the cohort meeting the safety and tolerability primary objective. http://www.bioworld.com/articles/730967 Thu, 07 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/730967-entrada-sinks-on-mixed-dmd-data-with-entr-601-44 Red arrow pointing downward on a stock market ticker Italian family-owned Angelini Pharma SpA is making its first move into the U.S. market, acquiring rare diseases specialist Catalyst Pharma Inc. in an all-cash deal worth $4.1 billion. The acquisition gives Angelini ownership of three marketed drugs for treating epilepsy and neuromuscular diseases that had combined sales of $589 million in 2025, a 19.8% increase over 2024. http://www.bioworld.com/articles/730965 Thu, 07 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/730965-italys-angelini-pays-41b-cash-for-rare-disease-specialist-catalyst Neurology illustration Star Sports Medicine Co. Ltd. debuted on the Hong Kong Stock Exchange with an HK$829.55 million (US$105.86 million) IPO May 5, with shares closing about 118% higher on the first day. http://www.bioworld.com/articles/730868 Wed, 06 May 2026 12:00:00 -0400 https://www.bioworld.com/articles/730868-china-med-tech-raises-star-sports-105m-ipo-cofoe-nabs-133m ​Star Sports Medicine HKEX IPO ceremony Star Sports Medicine's IPO ceremony on May 5. Credit: HKEX; x.com – @HKEXGroup Researchers from Alzecure Pharma AB and collaborators reported preclinical efficacy data on ACD-137, a selective negative allosteric TrkA modulator in models of peripheral neuropathy and osteoarthritis. http://www.bioworld.com/articles/730906 Wed, 06 May 2026 09:00:00 -0400 https://www.bioworld.com/articles/730906-alzecures-trka-modulator-combines-pain-relief-with-joint-protection Joint pain illustration Changchun Genescience Pharmaceuticals Co. Ltd. has reported new parathyroid hormone receptor 1 (PTHR1) agonists potentially useful for the treatment of arthritis, fracture, hyperphosphatemia and more. http://www.bioworld.com/articles/730524 Mon, 20 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730524-changchun-genescience-pharmaceuticals-divulges-new-pthr1-agonists Roche Holding AG is making good on its promise to try and convince the EMA of the benefits of Elevidys (delandistrogene moxeparvovec), announcing a further global phase III trial of the Duchenne muscular dystrophy gene therapy. http://www.bioworld.com/articles/730404 Fri, 17 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/730404-roche-plots-route-to-ema-approval-for-dmd-gene-therapy Green strand of DNA The loss of regenerative capacity in mammals over the course of evolution may be linked to certain environmental conditions rather than to a genetic limitation. Tissue stiffness around an amputated area, oxygen availability, or epigenetic regulation could determine this ability, according to two simultaneously published but independent studies published in Science, as reported by BioWorld yesterday. Science, as reported by BioWorld yesterday.]]> http://www.bioworld.com/articles/730344 Fri, 10 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730344-regeneration-in-mammals-is-controlled-by-environmental-conditions Confocal cross section of a regenerating tadpole limb. Confocal cross section of a regenerating tadpole limb, highlighting cellular boundaries (red) and nuclei (grey). Credit: Georgios Tsissios. The loss of regenerative capacity in mammals over the course of evolution may be linked to certain environmental conditions rather than to a genetic limitation. Tissue stiffness around an amputated area, oxygen availability, or epigenetic regulation could determine this ability, according to two simultaneously published but independent studies published in Science today. Science today.]]> http://www.bioworld.com/articles/730322 Thu, 09 Apr 2026 14:00:00 -0400 https://www.bioworld.com/articles/730322-regeneration-in-mammals-is-controlled-by-environmental-conditions Confocal cross section of a regenerating tadpole limb. Confocal cross section of a regenerating tadpole limb, highlighting cellular boundaries (red) and nuclei (grey). Credit: Georgios Tsissios. Renovare Therapeutics Inc. has announced its formal launch from stealth mode with a focus on developing regenerative therapies for musculoskeletal diseases, including osteoarthritis. http://www.bioworld.com/articles/730192 Wed, 08 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730192-renovare-launches-to-advance-osteoarthritis-therapies Joint pain Fortitude Biomedicines Inc. has disclosed that its lead program, FORT-202, is a first-in-class T-cell-targeting bispecific antibody for the potential treatment of axial spondyloarthritis. As a bispecific antibody, FORT-202 is designed to address multiple disease-causing pathways and is expected to significantly improve therapeutic efficacy. http://www.bioworld.com/articles/730188 Wed, 08 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730188-fortitude-unveils-lead-program-for-axial-spondyloarthritis Spinal cord Ernexa Therapeutics Inc. is advancing its lead program, ERNA-101, toward an IND submission in the third quarter of this year and the initiation of a first-in-human trial in the fourth quarter for the treatment of platinum-resistant ovarian cancer. http://www.bioworld.com/articles/730095 Thu, 02 Apr 2026 09:00:00 -0400 https://www.bioworld.com/articles/730095-ernexa-eyes-clinic-with-erna-101-for-ovarian-cancer Phase II data disclosed March 31 by Pepgen Inc. in myotonic dystrophy type 1 (DM1) hobbled the stock but might have been much different if not for one outlier in the 5-mg/kg multiple ascending dose cohort of the ongoing phase II Freedom2-DM1 trial – and Wall Street is pondering what the hitch means for the Boston-based firm as well as the competitive space. http://www.bioworld.com/articles/729979 Wed, 01 Apr 2026 12:00:00 -0400 https://www.bioworld.com/articles/729979-assay-what-eyes-on-pepgens-dm1-phase-ii Limb exam Researchers from the China Pharmaceutical University and Guangdong Pharmaceutical University (China) have unveiled the crucial role of the alternative splicing of E2A in myogenic progression and demonstrated that PTBP1, by controlling E2A alternative splicing, is a critical regulator of myogenesis. http://www.bioworld.com/articles/729724 Thu, 19 Mar 2026 09:00:00 -0400 https://www.bioworld.com/articles/729724-ptbp1-identified-as-potential-target-for-duchenne-muscular-dystrophy Light micrograph of skeletal muscle. Qyuns Therapeutics Co. Ltd. has moved closer to its first commercial product after China’s National Medical Products Administration (NMPA) accepted its NDA for IL-17 antibody crusekitug (QX-002N) for treating ankylosing spondylitis (AS), a chronic inflammatory disease that affects the spine and sacroiliac joints. http://www.bioworld.com/articles/729710 Tue, 17 Mar 2026 11:00:00 -0400 https://www.bioworld.com/articles/729710-chinas-qyuns-moves-il-17-biologic-toward-first-approval Illustration of inflamed spine Facioscapulohumeral muscular dystrophy (FSHD) is a muscle wasting disease caused by aberrant expression of double homeobox protein 4 (DUX4). When DUX4 is activated in skeletal muscle, it triggers myocyte cell death after several transcriptional changes, thus genetic DUX4 silencing arises as a promising approach for treating FHSD. http://www.bioworld.com/articles/729673 Tue, 17 Mar 2026 09:00:00 -0400 https://www.bioworld.com/articles/729673-sfl-0821-shows-promise-for-facioscapulohumeral-muscular-dystrophy Illustration of motor neuron connecting to muscle fiber Korea Research Institute of Chemical Technology has prepared and tested new serine/threonine-protein kinase LATS1 and LATS2 inhibitors reported to be useful for the treatment of sarcopenia, muscular dystrophy, inflammatory myopathy, congenital myopathy, myotonia, myofascial pain syndrome, motor neuron diseases and muscle injury. http://www.bioworld.com/articles/729639 Fri, 13 Mar 2026 09:00:00 -0400 https://www.bioworld.com/articles/729639-korea-research-institute-of-chemical-technology-reports-lats1-and-lats2-inhibitors With top-line pivotal data with gene therapy RGX-202 for Duchenne muscular dystrophy (DMD) due in the next quarter, Regenxbio Inc. rolled out positive interim data from the phase I/II Affinity trial at the Muscular Dystrophy Association Clinical and Scientific Conference (MDA) in Orlando, Fla., where Bridgebio Pharma Inc., Capricor Therapeutics Inc., and Solid Biosciences Inc. also had clinical findings to talk about. http://www.bioworld.com/articles/729478 Thu, 12 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729478-mda-vinay-away-aa-in-play-for-regenxbio-gene-therapy Illustration of man holding magnifying glass to human body model showing muscle anatomy Qyuns Therapeutics Co. Ltd. has moved closer to its first commercial product after China’s National Medical Products Administration (NMPA) accepted its NDA for IL-17 antibody crusekitug (QX-002N) for treating ankylosing spondylitis (AS), a chronic inflammatory disease that affects the spine and sacroiliac joints. http://www.bioworld.com/articles/729467 Wed, 11 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729467-chinas-qyuns-moves-il-17-biologic-toward-first-approval Illustration of inflamed spine The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal to consider PTC Therapeutics Inc.’s Translarna (ataluren) for the treatment of nonsense mutation disease. http://www.bioworld.com/articles/729463 Wed, 11 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729463-fda-blacklist-in-dmd-legislator-slams-ptc-turndown Photo of magnifying glass inspecting the text FDA Japan has approved the world’s first therapies derived from induced pluripotent stem cells (iPSCs), marking a major milestone for regenerative medicine and, potentially, a turning point in treating Parkinson’s disease. http://www.bioworld.com/articles/729586 Tue, 10 Mar 2026 12:00:00 -0400 https://www.bioworld.com/articles/729586-japan-approves-first-ipsc-therapy-for-parkinsons-disease Induced pluripotent stem cells Japan has approved the world’s first therapies derived from induced pluripotent stem cells (iPSCs), marking a major milestone for regenerative medicine and, potentially, a turning point in treating Parkinson’s disease. http://www.bioworld.com/articles/729360 Fri, 06 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729360-japan-approves-first-ipsc-therapy-for-parkinsons-disease Induced pluripotent stem cells Pepgen Inc. is forging ahead with tests of PGN-EDODM1 in other territories after the U.S. FDA placed a partial hold on the Freedom2-DM1 phase II trial, a multiple ascending-dose, randomized, placebo-controlled experiment in myotonic dystrophy type 1 (DM1). http://www.bioworld.com/articles/729340 Thu, 05 Mar 2026 12:00:00 -0500 https://www.bioworld.com/articles/729340-fda-curtails-pepgens-freedom2-operate-in-dm1 Person holding weight with assistance Innovacell Inc. launched a ¥14.16 billion (US$91.2 million) stock sale on the Tokyo Stock Exchange Feb. 24, ending a near two-year lull of biotech listings in Japan while signaling a dynamic year ahead for cell-based therapeutics. http://www.bioworld.com/articles/729194 Tue, 24 Feb 2026 12:00:00 -0500 https://www.bioworld.com/articles/729194-innovacells-91m-tokyo-ipo-to-fund-incontinence-cell-therapies Glass yen/yuan symbol