https://www.bioworld.com/rss en-us The saga of Capricor Therapeutics Inc.’s allogeneic cell therapy deramiocel for cardiomyopathy in Duchenne muscular dystrophy (DMD) added another bleak chapter as the U.S. FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted on the question of whether the available data provide “substantial evidence” that the drug works. Balloting turned out 9 no, 3 yes. http://www.bioworld.com/articles/732941 Wed, 29 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732941-resin-peace-capricor-sap-spat-weak-data-foil-dmd-adcom-bid 3D rendering of human cells or embryonic stem cells Kolon Tissuegene Inc.’s TG-C, an allogeneic cell and gene therapy candidate for knee osteoarthritis (OA), missed both co-primary endpoints and all key secondary endpoints in a highly anticipated top-line phase III clinical trial readout. http://www.bioworld.com/articles/733042 Tue, 28 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/733042-kolon-tissuegene-shares-fall-as-tg-c-misses-knee-oa-endpoints 3D illustration of knee joint Dyne Therapeutics Inc. has obtained IND clearance from the FDA for DYNE-302 in facioscapulohumeral muscular dystrophy (FSHD), a rare, genetic disease caused by a mutation in the DUX4 gene. Dyne plans to conduct a phase I trial in ambulatory adults with FSHD. http://www.bioworld.com/articles/732976 Tue, 28 Jul 2026 09:00:00 -0400 https://www.bioworld.com/articles/732976-dyne-302-gets-ind-for-facioscapulohumeral-muscular-dystrophy Medtronic plc’s recent launch of the Viaverte basivertebral nerve ablation system and completion of the acquisition of SPR Therapeutics Inc. broadens its chronic pain offering and strengthens its ability to treat patients across the pain care continuum. http://www.bioworld.com/articles/732865 Fri, 24 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732865-medtronic-builds-out-chronic-pain-continuum-with-viaverte-spr Medtronic Viaverte basivertebral nerve ablation system Viaverte basivertebral nerve ablation system. Credit: Medtronic plc Kolon Tissuegene Inc.’s TG-C, an allogeneic cell and gene therapy candidate for knee osteoarthritis (OA), missed both co-primary endpoints and all key secondary endpoints in a highly anticipated top-line phase III clinical trial readout. http://www.bioworld.com/articles/732754 Wed, 22 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732754-kolon-tissuegene-shares-fall-as-tg-c-misses-knee-oa-endpoints 3D illustration of knee joint For decades, treatment options for osteoarthritis have followed a familiar path: As cartilage deteriorates and pain worsens, conservative therapies eventually give way to joint replacement surgery. Australian regenerative medicine company Integrant Pty Ltd. wants to change that trajectory. Rather than replacing damaged joints, the Sydney-based company is building a platform designed to regenerate cartilage and preserve joint function by combining biologics, medical devices and artificial intelligence. http://www.bioworld.com/articles/732670 Tue, 14 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732670-integrant-targets-cartilage-repair-with-ai-biologics-platform Illustration of leg anatomy with target around knee The U.S. Court of Appeals for the Seventh Circuit revived Teva Pharmaceuticals USA Inc.’s breach-of-contract suit against Eli Lilly and Co. July 13, as it chided the lower court for dismissing the complaint at the pleadings stage. http://www.bioworld.com/articles/732601 Tue, 14 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732601-tevas-beef-with-lilly-over-forteo-settlement-lives-again For decades, treatment options for osteoarthritis have followed a familiar path: As cartilage deteriorates and pain worsens, conservative therapies eventually give way to joint replacement surgery. Australian regenerative medicine company Integrant Pty Ltd. wants to change that trajectory. Rather than replacing damaged joints, the Sydney-based company is building a platform designed to regenerate cartilage and preserve joint function by combining biologics, medical devices and artificial intelligence. http://www.bioworld.com/articles/732517 Thu, 09 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732517-integrant-targets-cartilage-repair-with-ai-biologics-platform Illustration of leg anatomy with target around knee Pending talks with regulators, Satellos Bioscience Inc. may seek accelerated approval in Duchenne muscular dystrophy (DMD) for SAT-3247, which yielded positive results in the adult phase II Trailhead study. Shares of Toronto-based Satellos (NASDAQ:MSLE) closed July 8 at $8.98, up 77 cents, or 9.4%, after the firm made public six-month interim data from Trailhead. http://www.bioworld.com/articles/732431 Wed, 08 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732431-phase-ii-dmd-trailhead-data-hike-satellos-stock Person-in-wheelchair1.jpg Neuracle Medical Technology Co. Ltd. is seeking a Shanghai IPO that could make it China’s first publicly listed invasive brain-computer interface (BCI) company, months after winning approval for the country’s first invasive BCI system. http://www.bioworld.com/articles/732472 Tue, 07 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732472-neuracle-eyes-shanghai-ipo-as-global-bci-funding-surges IPO puzzle pieces Neuracle Medical Technology Co. Ltd. is seeking a Shanghai IPO that could make it China’s first publicly listed invasive brain-computer interface (BCI) company, months after winning approval for the country’s first invasive BCI system. http://www.bioworld.com/articles/732271 Thu, 02 Jul 2026 12:00:00 -0400 https://www.bioworld.com/articles/732271-neuracle-eyes-shanghai-ipo-as-global-bci-funding-surges IPO puzzle pieces Ready or not, the future has arrived. Novel AI and brain-computer interface (BCI) systems are no longer confined to the realm of science fiction. As an increasingly intertwined human-machine model moves closer to adoption in real-world clinical and military practice, technological advances are sparking concerns over public health, ethics and national security. http://www.bioworld.com/articles/732225 Mon, 29 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732225-ascending-bci-systems-deepen-national-security-ethical-concerns Brain made of chip and circuits Amyotrophic lateral sclerosis (ALS)-associated genes provide direct therapeutic targets and reveal pathways that can be used to develop treatments that counteract their harmful molecular effects. Because the underlying causes of most ALS cases remain unknown, identifying disease-associated variants is essential to uncover the mechanisms that drive the disease, as shown at the European Network to Cure ALS (ENCALS) meeting, held in Madrid from June 24 to 26, 2026. http://www.bioworld.com/articles/732185 Fri, 26 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732185-encals-2026-from-genetics-to-advancing-strategies-against-als Illustration for mutations in the DNA leading to brain diseases or neurodegenerative disorders Nippon Shinyaku Co. Ltd.’s NS-035 has been awarded Japanese orphan drug designation for the treatment of Fukuyama congenital muscular dystrophy. http://www.bioworld.com/articles/732135 Thu, 25 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/732135-nippon-shinyakus-ns-035-designated-orphan-drug-in-japan 3d illustration of human body muscle tissue anatomy Neither of Cynata Therapeutics Ltd.’s pivotal trials for its Cymerus stem cell platform met the primary endpoints, with no statistically significant differences between active and control groups on primary or key secondary efficacy measures, CEO Kilian Kelly told investors during a June 22 conference call. http://www.bioworld.com/articles/732155 Tue, 23 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732155-cynatas-cymerus-flops-in-osteoarthritis-gvhd Stem cells Decades of research are helping unravel the “black box” of the brain. The second article in BioWorld’s series on the Brain-Computer Interface (BCI) field looks at how simultaneous breakthroughs in AI technology are pushing the BCI field from a theoretical concept to a potential real-world, clinical option for individuals, particularly in China where the National Medical Products Administration greenlighted the world’s first invasive BCI system – Neuracle Medical Technology Co. Ltd.’s Neural Electronic Opportunity – for clinical use in March 2026. BioWorld’s series on the Brain-Computer Interface (BCI) field looks at how simultaneous breakthroughs in AI technology are pushing the BCI field from a theoretical concept to a potential real-world, clinical option for individuals, particularly in China where the National Medical Products Administration greenlighted the world’s first invasive BCI system – Neuracle Medical Technology Co. Ltd.’s Neural Electronic Opportunity – for clinical use in March 2026.]]> http://www.bioworld.com/articles/732062 Tue, 23 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732062-2026-marks-critical-turning-point-for-bci-technology Patient using Neuracle NEO system A patient grasps a bottle with a prosthetic hand using Neuracle’s NEO system, codeveloped by Tsinghua University School of Medicine. Credit: Tsinghua University Neither of Cynata Therapeutics Ltd.’s pivotal trials for its Cymerus stem cell platform met the primary endpoints, with no statistically significant differences between active and control groups on primary or key secondary efficacy measures, CEO Kilian Kelly told investors during a June 22 conference call. http://www.bioworld.com/articles/732047 Mon, 22 Jun 2026 12:00:00 -0400 https://www.bioworld.com/articles/732047-cynatas-cymerus-flops-in-osteoarthritis-gvhd Stem cells Arthrogryposis multiplex congenita (AMC) is a group of disorders defined by two or more contractures in different body areas; while genes encoding sarcomeric proteins are usually involved in its pathogenesis, the role of the dystrophin complex is not well studied in AMC. Utrophin, encoded by the UTRN gene, is an important fetal dystrophin homologue and was the focus of a recently presented study. UTRN gene, is an important fetal dystrophin homologue and was the focus of a recently presented study.]]> http://www.bioworld.com/articles/731991 Wed, 17 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731991-utrn-gene-involved-in-arthrogryposis-study-finds Applied Biologics LLC has initiated a preclinical research program focused on osteoarthritis and other degenerative diseases. The program marks an expansion of the company’s research and development efforts beyond chronic wounds. http://www.bioworld.com/articles/731856 Fri, 12 Jun 2026 09:00:00 -0400 https://www.bioworld.com/articles/731856-applied-biologics-begins-osteoarthritis-research-program Drug discovery illustration 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