FST

SEATTLE--( BUSINESS WIRE )-- Oisín Biotechnologies (Oisín), a privately held biotechnology company focused on mitigating the effects of age-related diseases, today announced the publication of preclinical data from its follistatin (FST) gene therapy program to mitigate muscle loss in an article titled “Safe and Effective Delivery of DNA and RNA Using Proteolipid Vehicles” in Cell. Scientists at Oisín, OncoSenX , the University of Alberta , Dalhousie University , and Aegis Life, Inc. , contributed to the published research led by Entos ® Pharmaceuticals , inventor of the FAST-PLV™ platform. The article is available from Cell online today and in print on September 19th. “When we set out to build cutting-edge genetic medicines to address age-related diseases, we knew the ability to deliver nucleic acids safely and effectively to a broad array of cell types would be essential to our success,” said Matthew Scholz, chief executive officer, Oisín Biotechnologies. “We placed an early bet on the Entos FAST-PLV system because it has the potential to overcome the biodistribution, tolerability, and repeat-dosing limitations of other commonly used delivery systems. The data published today in Cell validate that choice and provide important proof-of-principle for our follistatin gene therapy program. As we utilize the FAST-PLV system in our other ongoing development programs, the remarkable flexibility of the platform continues to support the broader potential of our genetic medicine approach to addressing age-related diseases.” The published data include results from studies conducted by Oisín scientists evaluating the impact of administering a single dose of FAST-PLV FST gene therapy in mice, over time. After 15 weeks, the mice administered with FST gene therapy had significantly greater grip strength and body weight increases than the control group. At 34 weeks, the FST gene therapy mice showed significant increases in muscle size, especially in the hind limbs and throughout the torso, that correlated with a significant increase in cross-sectional muscle fiber area compared with the control group. “Muscle loss is associated with age-related frailty, which affects the mobility and quality of life for millions of elderly people,” said Henry Garcia, Ph.D., head of Aging R&D at Oisín. “With an aging population and the increasing use of GLP-1 class agonists to treat diabetes and obesity, there is a growing need for a therapy that can safely and effectively mitigate the side effect of muscle loss and, ideally, rebuild muscle.” FAST-PLVs form the basis of the Entos Fusogenix™ PLV delivery platform. This technology is licensed to Oisin for the development of age-related disease applications. In July 2024 , Oisín announced the first close of a $15 million Series A financing round, to advance its age-related disease therapy portfolio. About Oisín Biotechnologies Oisín is a privately held biotechnology company developing genetic medicines to address age-related diseases. Headquartered in Seattle, Washington, Oisín utilizes the Entos ® Fusogenix™ PLV™ delivery platform to deliver DNA directly to cells throughout the body, addressing the damage and detrimental effects caused by the aging process itself. The company’s first therapies focus on eliminating unwanted fat cells and building muscle mass. For more information, visit www.oisinbio.com or follow Oisín on X and LinkedIn . About Entos Pharmaceuticals Inc. A new reality in genetic medicine lies ahead, one that will be ushered in with the advent of safe, effective, and re-dosable nucleic acid delivery technologies. Since its inception in 2016, Entos ® has been dedicated to advancing next-generation genetic medicines using our proprietary Fusogenix TM PLV TM drug delivery system. The Fusogenix PLV platform is formulated with FAST proteins to enable the delivery of nucleic acid to target cells through direct fusion. Entos is pioneering the development of life-changing medicines for patients and has partnered with global companies, such as Eli Lilly, to accelerate and expand the impact of our platform. Entos Pharmaceuticals Inc. is headquartered in Edmonton, Canada, with its wholly owned U.S. and U.K. subsidiaries based in San Diego, California and London, United Kingdom, respectively. For more information, visit www.entospharma.com , or follow Entos on LinkedIn . Entos ® word mark and design logo, Fusogenix TM and PLV TM are registered trademarks of Entos Pharmaceuticals Inc. All other trademarks and registered trademarks are the property of their respective owners.

Pfizer's mini-dystrophin gene therapy fordadistrogene movaparvovec failure in the Duchenne muscular dystrophy treatment opens new opportunities for several pharmaceutical companies such as Sarepta Therapeutics (SRP-5051), Santhera Pharmaceuticals/ReveraGen BioPharma (Vamorolone), Taiho Pharmaceutical (TAS-205), FibroGen (Pamrevlumab), and others, to advance their therapies and potentially dominate the DMD market. LAS VEGAS, June 17, 2024 /PRNewswire/ -- Duchenne muscular dystrophy is the most prevalent type of muscular dystrophy found in children. This genetic condition leads to ongoing muscle weakening and degeneration. It is one among nine different forms of muscular dystrophy. According to DelveInsight's estimates, the US accounted for around ~17K total prevalent cases of Duchenne muscular dystrophy in 2023. The highest cases were found in the children of the 5-9 age group, these numbers are expected to grow by the year 2034. As per DelveInsight's analysis, the most commonly associated comorbidities accompanied with DMD included the highest cases of scoliosis accounting for around ~20% of cases followed by ADHD, cardiomyopathy, and obsessive-compulsive disorders accounting for around ~19%, ~18%, and ~15% cases respectively in the US in the year 2023. The standard of care for DMD typically involves a combination of corticosteroids and surgical interventions. Prednisone and deflazacort are commonly prescribed glucocorticoids that have been utilized for over two decades to increase muscular strength in DMD patients. Surgery may be necessary to address contractures, spinal curvature, or scoliosis, which can affect breathing and overall mobility. Additionally, interventions such as pacemakers or other cardiac devices may be utilized to improve heart function. Sarepta Therapeutics is a key player in the DMD market, boasting three approved assets and two in the current pipeline. Despite its dominance, emerging competition is evident. The historical success rate for market entry varies across the 7MM. Notably, the DMD market exhibits geographic disparities in regulatory approvals, with instances of products approved in one jurisdiction and rejected in another, despite identical clinical data, reflecting the distinct standards of regulatory bodies such as EMA and FDA. Learn more about the FDA-approved DMD drugs @ Drugs for Duchenne Muscular Dystrophy Treatment The current US DMD treatment market possesses approved products, EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), and VILTEPSO (viltolarsen), ELEVIDYS (delandistrogene moxeparvovec) in patients with DMD. In January 2024, Santhera Pharmaceuticals announced the introduction of its AGAMREE (vamorolone) to treat DMD patients aged four years and above in Germany. Santhera Pharmaceuticals has officially entered the commercial stage of its biopharma journey with the launch of this drug in Germany. ELEVIDYS, the first FDA-approved gene therapy for DMD, received accelerated approval in June 2023. However, ICER recently challenged the potential conversion of Sarepta's gene therapy to full approval, citing concerns about the surrogate endpoint used in the approval process. In the EU4 and the UK, the current market is dominated by steroid therapies along with an approved medication targeting DMD patients with the nonsense mutation, TRANSLARNA (ataluren). In Japan, the only approved treatment is VILTEPSO (viltolarsen). ELEVIDYS is a prescription gene therapy designed for ambulatory children aged 4 to 5 years with DMD and a confirmed dystrophin gene mutation. It received accelerated approval. Patients will take oral corticosteroids before and after receiving ELEVIDYS and will have weekly blood tests to monitor liver enzyme levels for three months post-treatment. In June 2023, the USFDA approved ELEVIDYS as the first gene therapy for treating pediatric patients aged 4-5 years with Duchenne Muscular Dystrophy and a confirmed DMD gene mutation. VYONDYS 53, developed by Sarepta Therapeutics, is prescribed for treating DMD in patients with a specific mutation of the DMD gene that can be addressed by exon 53 skipping. This approval is under the accelerated approval pathway, based on the observed increase in dystrophin production in the skeletal muscle of patients treated with VYONDYS 53. The FDA approved VYONDYS 53 (golodirsen) in December 2019. Additionally, the FDA granted VYONDYS 53 (golodirsen) New Chemical Entity (NCE) exclusivity until December 2024 and Orphan Drug Exclusivity until December 2026. To know more about DMD treatment options, visit @ New Treatment for Duchenne Muscular Dystrophy The DMD market is crowded with so many companies including FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, Cumberland Pharmaceuticals, Sarepta Therapeutics, Antisense Therapeutics, Capricor Therapeutics, and others are running clinical trials to improve the treatment space. The DMD pipeline possesses potential drugs in mid-stage developments to be launched shortly. Some of the promising drugs in the pipeline include Pizuglanstat (TAS-205), SRP-5051, Allogeneic Cardiosphere-derived Cells (CAP-1002), ITF2357 (Givinostat), and others that hold the potential to create a significant positive shift in the DMD market size. Discover which therapies are expected to grab major DMD market share @ Duchenne Muscular Dystrophy Market Report Italfarmaco is developing Givinostat (ITF2357), an oral small molecule that functions as a histone deacetylase inhibitor with potential anti-inflammatory, anti-angiogenic, and antineoplastic properties. ITF2357 increases the production of follistatin protein in muscle cells by inhibiting the HDAC enzyme, leading to increased muscle mass and prevention of muscle degeneration. This action counters the effects of myostatin, helping to alleviate the symptoms of DMD. Givinostat is currently in Phase III trials. The FDA has granted it Orphan Drug, Rare Pediatric Disease, and Fast Track designations for DMD treatment. Additionally, the European Commission has designated Givinostat as an Orphan Medicinal Product for DMD treatment. TAS-205 (pizuglanstat) is a selective inhibitor of hematopoietic prostaglandin D synthase (HPGDS), developed by Taiho Pharmaceutical. It is being advanced as a treatment for Duchenne muscular dystrophy (DMD), effective irrespective of the type of dystrophin gene mutation. By inhibiting HPGDS, which worsens the inflammatory response in the muscles of DMD patients, it helps control the decline in motor function. Currently, it is in Phase III of clinical trials. Discover more about drugs for DMD in development @ Duchenne Muscular Dystrophy Clinical Trials The anticipated launch of these emerging therapies for Duchenne muscular dystrophy are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Duchenne muscular dystrophy market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight estimates that the market size for DMD is expected to grow from USD 2.1 billion in 2023 with a significant CAGR by 2034. The growth of the DMD market is expected to be mainly driven by increased diagnosed incidence, patient awareness, and a robust clinical pipeline during the forecast period (2024–2034). DelveInsight's latest published market report titled as Duchenne Muscular Dystrophy Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the DMD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The DMD market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Total Prevalent Cases of Duchenne Muscular Dystrophy Age-specific Cases of Duchenne Muscular Dystrophy Ambulatory and Non-ambulatory Cases of Duchenne Muscular Dystrophy Mutation-specific Cases of Duchenne Muscular Dystrophy Associated Comorbidities in Duchenne Muscular Dystrophy The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM DMD market. Highlights include: 11-year Forecast 7MM Analysis Epidemiology-based Market Forecasting Historical and Forecasted Market Analysis upto 2034 Emerging Drug Market Uptake Peak Sales Analysis Key Cross Competition Analysis Industry Expert's Opinion Access and Reimbursement Download this DMD market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the DMD market. Also, stay abreast of the mitigating factors to improve your market position in the DMD therapeutic space. Related Reports Duchenne Muscular Dystrophy Epidemiology Forecast Duchenne Muscular Dystrophy Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted Duchenne muscular dystrophy epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan. Duchenne Muscular Dystrophy Pipeline Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Duchenne muscular dystrophy companies, including Santhera Pharmaceuticals, Sarepta Therapeutics, Italfarmaco, Wave Life Sciences Ltd, FibroGen, Edgewise Therapeutics, Pfizer, Daiichi Sankyo, Sarepta Therapeutics, Inc., ENCell, Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, Hansa Biopharma, Ultragenyx Pharmaceutical, Dyne Therapeutics, Entrada Therapeutics, AAVogen, PepGen, Antisense Therapeutics, BioMarin Pharmaceutical, Avidity Biosciences, Sarepta Therapeutics, Dyne Therapeutics, Solid Biosciences Inc, Regenxbio, Stealth BioTherapeutics, among others. Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Nonsense Mutation Duchenne Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key nonsense mutation Duchenne muscular dystrophy companies, including Taiho Pharmaceutical, Solid Biosciences, Capricor, Nippon Shinyaku, among others. Becker Muscular Dystrophy Pipeline Becker Muscular Dystrophy Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key becker muscular dystrophy companies, including Epirium Bio, Ultragenyx, Strykagen, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP

The test will be used by providers and consumers to monitor muscle growth. BOCA RATON, Fla., March 13, 2024 /PRNewswire/ -- Physicians Lab, a provider of at-home lab testing for integrative medicine and wellness, launched a new at-home lab test that measures the analytes myostatin and follistatin. The test will show the current myostatin and follistatin levels and the ratio of myostatin to follistatin. The new test will be useful for monitoring muscle loss or growth in individuals. Continue Reading "Our research team has delivered another test that's convenient, reliable and has a fast turnaround time," Jaime Rios, CEO of Physicians Lab, said. "Our method is minimally invasive. We can provide valuable insight into a person's muscle development without burdening them by taking a large blood sample. Our team is excited to expand Physicians Lab solutions and to offer this at-home lab test to integrative providers and directly to health-conscious consumers." Physicians Lab provides convenient at-home lab testing that is precise, reliable and timely. Post this Myostatin, a member of the transforming growth factor-beta (TGF-β) superfamily, is a potent regulator of muscle growth. It acts primarily to inhibit muscle development, maintaining muscle size by inhibiting both the growth and differentiation of muscle cells. Assessing myostatin levels can provide valuable understanding of muscle homeostasis and the pathological mechanisms underlying muscle wasting diseases. Follistatin, serves as an antagonist to myostatin and other members of the TGF-β family. By binding to and inhibiting these growth factors, follistatin promotes muscle growth and has a critical role in muscle repair and regeneration. It's involved in a variety of biological processes beyond muscle growth, including reproduction and the regulation of inflammation. "GLP-1 has become a popular weight loss intervention," Rios said. "Understanding myostatin levels could be beneficial to patients on GLP-1 agonists like semaglutide, since up to 40 percent of the weight loss during therapy could come from lean mass loss, which includes muscle." Physicians Lab uses the technique called bloodspot microsampling. This method allows for the collection of blood samples in a minimally invasive manner. It requires only a small volume of blood that can be obtained from a simple finger prick. To determine the values of myostatin and follistatin, Physicians Lab uses a high-sensitivity Enzyme-Linked Immunosorbent Assay (ELISA) technique. This ensures accurate quantification of myostatin and follistatin levels in these small-volume samples. For more information about Physicians Lab and at-home lab testing solutions, visit . About Physicians Lab Physicians Lab, located in Boca Raton, FL, provides convenient at-home lab testing that is precise, reliable and timely. Physicians Lab serves physicians focused on integrative medicine and wellness for an aging population by shipping easy-to-use lab kits to patients' homes. Physicians Lab also delivers superior retail solutions direct to consumers who are actively seeking to manage their health and wellness. Contact: Jaime Rios, CEO (877) 326-5969 [email protected] SOURCE Physicians Lab