Sarepta Reports Second Death of Patient Using Its Gene Therapy
Sarepta Therapeutics Inc. said a second patient has died of acute liver failure after being treated with its gene therapy for a rare muscle disorder.
The death comes three months after a teenage boy died after getting the one-time treatment, Elevidys. Both cases occurred in patients with Duchenne muscular dystrophy who are unable to walk, the company said.
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Associated Press
an hour ago
- Associated Press
Innovent Announces Completion of First Participant Dosed in the Seventh Phase 3 Clinical Trial (GLORY-OSA) of Mazdutide in China
SAN FRANCISCO and SUZHOU, China, June 15, 2025 /PRNewswire/ -- Innovent Biologics, Inc. (Innovent) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, cardiovascular and metabolic, autoimmune, ophthalmology and other major disease areas, announced that the first participant has been successfully dosed in a Phase 3 clinical trial (GLORY-OSA) of mazdutide, a dual glucagon (GCG) and glucagon-like peptide-1 (GLP-1) receptor agonist, in Chinese participants with moderate-to-severe obstructive sleep apnea (OSA) and obesity (BMI ≥ 28 kg/m2). This is the seventh Phase 3 clinical study of mazdutide in China, continuing to explore its potential in managing obesity and a range of metabolic syndromes, with the aim of generating more comprehensive and high-quality evidence to support clinical application. GLORY-OSA is a multicenter, randomized, open-label Phase 3 clinical study (NCT06931028) comparing the efficacy and safety of mazdutide 9mg versus placebo in Chinese participants with moderate-to-severe obstructive sleep apnea (OSA) and obesity (BMI ≥ 28 kg/m2).The primary endpoint is the change in apnea-hypopnea index (AHI) from baseline to Week 48. Globally, an estimated 425 million people aged 30 to 69 live with moderate-to-severe OSA, with China bearing the highest burden at approximately 66 million cases[1]. However, the diagnosis rates remain critically low, with less than 1% in China and only 20% in the U.S.[2],[3] The condition disproportionately affects individuals living with obesity, with a prevalence of 40%, increasing to 80.5% among those undergoing bariatric surgery[4],[5]. OSA-associated co-morbidities, including hypertension, cardiac arrhythmias, stroke, and metabolic syndrome, are strongly correlated with OSA severity[6],[7]. Positive airway pressure (PAP) is the first-line treatment for OSA, and certain patients experience transformative benefits from PAP. Challenges exist, however, as many others are unable to accept or adhere to PAP treatment due to the burdensome nature of the therapy. Moreover, current evidence does not strongly support a direct link between PAP and improved cardiometabolic outcomes (e.g., mortality, hypertension, myocardial infarction, stroke)[8]. Recently, the FDA approved Zepbound® (tirzepatide), a dual GLP-1 and glucose-dependent insulinotropic polypeptide (GIP) agonist, as the first and only prescription medicine for moderate-to-severe OSA in adults with obesity[9]. There is currently no approved treatment for OSA in China[1], highlighting a significant unmet medical need. The literature suggests[10],[11],[12] that a 10% reduction in weight is associated with a 26% decrease in AHI, and that each 1-unit reduction in BMI corresponds to a 2-3 unit decrease in AHI. As such, weight loss medications have emerged as potential treatments for OSA. Professor Tianpei Hong, the Principal Investigator of the Study, Peking University Third Hospital, stated, 'Patients with untreated OSA face significantly increased risks of cardiovascular diseases, metabolic disorders, neurocognitive decline, and traffic accidents. Their quality of life and labor capacity are often severely hampered. The OSA proportion of patients living with obesity is large and grows year after year. Despite this, an effective drug treatment regimen for OSA is lacking in China[13]. In our Phase 3 study GLORY-1[14], mazdutide showed good weight loss efficacy with multi-cardiovascular metabolism improvement, and a well-tolerated safety profile, which positions mazdutide as the world's first dual GCG/GLP-1 receptor targeted drug for patients with OSA and obesity. I will work closely with the GLORY-OSA research team to ensure the successful and high-quality completion of this study, with the ultimate goal of obtaining robust clinical evidence and expanding treatment options for Chinese patients with moderate-to-severe OSA and obesity.' Dr. Lei Qian, Senior Vice President of Clinical Development at Innovent, stated, 'We have developed different doses and regimens of mazdutide to address the diverse treatment needs of patients with different indications. The development of mazdutide for OSA specifically targets the treatment needs of Chinese patients with moderate-to-severe OSA and obesity (BMI ≥ 28 kg/m2). In a Phase 2 study, mazdutide 9 mg achieved an 18.6 % weight loss compared to placebo, along with notable cardiovascular and metabolic benefits. The 9 mg regimen also features a simple titration schedule and a favorable safety profile. We are optimistic that the GLORY-OSA study will further validate mazdutide's powerful efficacy and good safety, and provide a promising pharmacologic alternative to PAP, the current standard of care for patients with moderate-to-severe OSA.' About Mazdutide (IBI362) Innovent entered into an exclusive license agreement with Eli Lilly and Company (Lilly) for the development and potential commercialization of OXM3 (also known as mazdutide), a GLP-1R and GCGR dual agonist, in China. As a mammalian oxyntomodulin (OXM) analogue, mazdutide may offer additional benefits beyond those of GLP-1 receptor agonists—such as promoting insulin secretion, lowering blood glucose and reducing body weight—by also activating the glucagon receptor to increase energy expenditure and improve hepatic fat metabolism. Mazdutide has demonstrated excellent weight loss and glucose-lowering effects in clinical studies. It has also shown benefits in reducing waist circumference, blood lipids, blood pressure, blood uric acid, liver enzymes, and liver fat content, as well as improving insulin sensitivity. Currently, Mazdutide has two NDAs accepted for review by NMPA, including for: Mazdutide is currently being evaluated in seven Phase 3 clinical studies, including: Among these, GLORY-1, DREAMS-1, and DREAMS-2 have already met their primary endpoints and the other four studies are currently ongoing. In addition, several new clinical studies of mazdutide are initiated or planned, including: About Innovent Innovent is a leading biopharmaceutical company founded in 2011 with the mission to empower patients worldwide with affordable, high-quality biopharmaceuticals. The company discovers, develops, manufactures and commercializes innovative medicines that target some of the most intractable diseases. Its pioneering therapies treat cancer, cardiovascular and metabolic, autoimmune and eye diseases. Innovent has launched 15 products in the market. It has 3 new drug applications under regulatory review, 4 assets in Phase III or pivotal clinical trials and 15 more molecules in early clinical stage. Innovent partners with over 30 global healthcare companies, including Eli Lilly, Sanofi, Incyte, LG Chem and MD Anderson Cancer Center. Guided by the motto, 'Start with Integrity, Succeed through Action,' Innovent maintains the highest standard of industry practices and works collaboratively to advance the biopharmaceutical industry so that first-rate pharmaceutical drugs can become widely accessible. For more information, visit or follow Innovent on Facebook and LinkedIn. Forward-looking statement This news release may contain certain forward-looking statements that are, by their nature, subject to significant risks and uncertainties. The words 'anticipate', 'believe', 'estimate', 'expect', 'intend' and similar expressions, as they relate to Innovent Biologics ('Innovent'), are intended to identify certain of such forward-looking statements. The Company does not intend to update these forward-looking statements regularly. These forward-looking statements are based on the existing beliefs, assumptions, expectations, estimates, projections and understandings of the management of the Company with respect to future events at the time these statements are made. These statements are not a guarantee of future developments and are subject to risks, uncertainties and other factors, some of which are beyond the Company's control and are difficult to predict. Consequently, actual results may differ materially from information contained in the forward-looking statements as a result of future changes or developments in our business, the Company's competitive environment and political, economic, legal and social conditions. The Company, the Directors and the employees of the Company assume (a) no obligation to correct or update the forward-looking statements contained in this site; and (b) no liability in the event that any of the forward-looking statements does not materialise or turn out to be incorrect. View original content: SOURCE Innovent Biologics
Associated Press
an hour ago
- Associated Press
Adolore BioTherapeutics Announces Publication Demonstrating Biosafety and Efficacy of Kv7 Activating rdHSV-CA8* Analgesic Gene Therapy for Chronic Pain via the Intra-Articular Route in Mice
Findings highlight the advantages of Adolore's approach for the delivery of proprietary gene therapy directly to specialized pain-sensing peripheral nerves (nociceptors) that mediates profound analgesia with the potential to address the great unmet need for non-opioid chronic pain therapies Data further supports the clinical-translational value of Adolore's proprietary non-opioid analgesics for treating chronic non-cancer pain These data support the Company's continuing efforts to progress with IND-enabling studies of ADB-102 gene therapy for the treatment of osteoarthritis (OA) chronic knee pain. DELRAY BEACH, FL / ACCESS Newswire / June 15, 2025 / Adolore BioTherapeutics ('Adolore' or the 'Company'), a biotechnology company focused on developing breakthrough opioid-free gene therapy treatments for chronic pain and neurological disorders, today announced the publication of its manuscript titled, 'Biosafety and Efficacy of Kv7 Activating rdHSV-CA8* Analgesic Gene Therapy for Chronic Pain Via the Intra-Articular Route in Mice1,' in the peer-reviewed journal, Molecular Therapy. Roy Clifford Levitt, MD, Clinical Professor at the University of Miami, Principal Investigator and Program Director of the NIH, NINDS, HEAL Award supporting ADB-102 development for the treatment of chronic knee pain due to OA, and Founder & Executive Chairman of Adolore BioTherapeutics, published biosafety and efficacy data from preclinical studies of Adolore's gene therapy expressing a human carbonic anhydrase-8 variant peptide (CA8*). In model systems, replication-defective, disease-free, herpes simplex virus (rdHSV) gene therapy expressing an analgesic carbonic anhydrase-8 (CA8*) peptide variant corrects somatosensory hyperexcitability by activating Kv7 voltage-gated potassium channels, produces profound, long-lasting analgesia and treats chronic pain from knee OA. In these studies, we provide the first non-Good Laboratory Practices (GLP) biosafety, efficacy, biodistribution, shedding, and histopathology examination of this rdHSV-CA8* via the intra-articular knee route of administration. Naive mice were examined for clinical safety, distribution of virus across all major tissues, knee histopathology, and analgesic efficacy. We observed no signs of persistent toxicity or histopathology, viral genomes remained where they were injected, and there was no evidence of shedding. Profound analgesia persisted for >6 months without functional impairments. These initial biosafety and efficacy data support further development of rdHSV-CA8* for treating chronic knee pain due to moderate-to-severe OA. Dr. Levitt, commented, 'Kv7 voltage-gated potassium channel activators, like rdHSV-CA8* open these channels and hyperpolarize nociceptors making them less excitable to produce profound analgesia. Kv7 activators are well-known to produce potent non-opioid-based analgesia in many human chronic pain conditions. While Kv7 openers are no longer available due to off-target adverse events related to systemic administration, they have been successfully translated from animal models to human chronic pain conditions. Bolstered by our substantial body of published data, we continue to develop our innovative approach to address the significant serious unmet need for safe and effective locally acting pain therapies to replace opioids. Our preclinical data strongly support continued preclinical development toward an IND and clinical studies of ADB-102.' The Company's lead development program for the treatment of chronic pain in knee osteoarthritis is fully funded by a UG3/UH3 grant awarded to the University of Miami by NIH/NINDS HEAL program to support all formal pre-clinical GLP/GMP/GCP development work through a first-in-human study of ADLR-1l01 in patients expected to commence in 2026. 1 Levitt et al., Biosafety and efficacy of Kv7 activating rdHSV-CA8* analgesic gene therapy for chronic pain via the intraarticular route in mice, Molecular Therapy (2025), About Carbonic Anhydrase-8 (CA8*) Gene Therapy CA8* (variants of naturally occurring human carbonic anhydrase-8 analgesic peptides) gene therapies are a novel class of neuronal calcium channel inhibitors that activate Kv7 voltage-gated potassium channels and are administered locally and long-acting. Oral small molecule pain therapeutics that activate Kv7 voltage-gated potassium channels demonstrated proven analgesic efficacy before they were removed from the market due to severe adverse events related to systemic exposure and their metabolism. CA8* gene therapy provides versatile dosing regimens and routes of administration, including intra-articular, intra-neuronal (nerve block), and intradermal injection. This non-opioid CA8* mechanism-of-action addresses neuropathic, inflammatory, and nociceptive pain, which applies to a broad range of chronic pain indications and neurological disorders. These conditions include osteoarthritis, lower back, and cancer pain; diabetes and other forms of peripheral neuropathy; as well as rare pain conditions such as erythromelalgia, a heritable chronic pain condition and epilepsy and hearing loss. About Adolore BioTherapeutics, Inc. Adolore BioTherapeutics, Inc., is a biotechnology company focused on developing novel therapies for treating chronic pain using a revolutionary intra-cellular replication-defective HSV (rdHSV) drug delivery platform that is disease-free, non-toxic, and permits localized peripheral nervous system delivery of proprietary biotherapeutics. This rdHSV gene therapy technology incorporates an established re-dosing strategy and an excellent safety profile. HSV vectors are known for their stability and prolonged gene expression, providing an excellent basis for the long- term treatment of chronic pain conditions and neurological disorders. Our best-in-class CA8* programs are long-acting, locally administered gene therapies that are opioid-free Disease-Modifying Anti-Pain therapies (DMAPs) designed to treat many forms of chronic pain, epilepsy and hearing loss. Leveraging its innovative gene therapy vectors expressing CA8* analgesic peptides (ADLR-1001), Adolore is currently advancing two preclinical development programs: ADB-101 for the treatment of patients' chronic pain caused by erythromelalgia, an orphan disease, and ADB-102, their lead program for the treatment of patients with chronic pain caused by knee OA. Based on substantial compelling preclinical data generated to date, the Company is progressing these programs toward IND filings and first-in-human clinical studies. Adolore has two additional programs: ADB-104 for Drug-Resistant Refractory Focal Epilepsy and ADB-105 for Acute Severe Hearing Loss. For more information, visit Forward-Looking Statements To the extent, this announcement contains information and statements that are not historical, they are considered forward-looking statements within the meaning of the federal securities laws. You can identify forward-looking statements by the use of the words 'believe,' 'expect,' 'anticipate,' 'intend,' 'estimate,' 'project,' 'will,' 'should,' 'may,' 'plan,' 'intend,' 'assume' and other expressions which predict or indicate future events and trends and which do not relate to historical matters. You should not rely on forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, some of which are beyond the control of the Company. These risks and uncertainties include but are not limited to those associated with drug development. These risks, uncertainties, and other factors may cause the actual results, performance, or achievements of the Company to be materially different from the anticipated future results, performance, or achievements expressed or implied by the forward- looking statements. Investor Relations Contact Paul Barone (215)622-4542 [email protected] SOURCE: Adolore Biotherapeutics, Inc. press release
Associated Press
an hour ago
- Associated Press
Samsung Biologics launches drug screening services, Samsung Organoids
INCHEON, South Korea, June 15, 2025 /PRNewswire/ -- Samsung Biologics (KRX: a leading contract development and manufacturing organization (CDMO), today announced the launch of Samsung Organoids – advanced drug screening services to support clients in drug discovery and development. Organoids are three-dimensional cell culture systems engineered to closely mimic human organs, offering clinically relevant insights to better understand specific drug responses and improve lead selection. Organoids are emerging as a new research model due to their high similarity to live tissues and potential applications in biomarker discovery and drug efficacy prediction. Samsung Organoids support precision screening to predict patient responses, streamline preclinical development, and accelerate timelines toward investigational new drug (IND) filings through data-driven analysis of candidate molecules. With the launch, Samsung Biologics expands its business to include preclinical research, with service offerings spanning target discovery, lead selection, preclinical development, and clinical trial planning. Leveraging its expertise in development and manufacturing, the company will provide data-driven, multi-modal insights into the characteristics and mechanisms of candidate molecules. By helping clients address challenges that may arise during the drug discovery phase, Samsung Biologics aims to enhance clinical success rates. 'The latest service launch reflects our unwavering commitment to driving innovation by improving drug success rates and creating new possibilities in personalized medicine,' said John Rim, CEO and President of Samsung Biologics. 'The addition of research services is a significant move for us to create added value for clients by supporting the drug life cycle from start to finish with thorough therapeutic analysis.' About Samsung Biologics Samsung Biologics (KRX: is a leading contract development and manufacturing organization (CDMO), offering end-to-end integrated services that range from late discovery to commercial manufacturing. With a combined biomanufacturing capacity of 784 kL across five plants, Samsung Biologics leverages cutting-edge technologies and expertise to advance diverse modalities, including multispecific antibodies, fusion proteins, antibody-drug conjugates, and mRNA therapeutics. Samsung Biologics operates a global network with facilities and offices in Korea, the U.S., and Japan. Samsung Biologics America supports clients based in the U.S. and Europe, while its Tokyo sales office serves the APAC region. Samsung Biologics continues to invest in new capabilities to maximize operational and quality excellence, ensuring flexibility and agility for clients. The company is committed to the on-time, in-full delivery of safe, high-quality products, as well as making sustainable business decisions for the betterment of society and global health. For more information, please visit Samsung Biologics Media Contact Claire Kim [email protected] View original content to download multimedia: SOURCE Samsung Biologics
