Latest news with #HemophiliaA
Business Wire
06-05-2025
- Business
- Business Wire
Demonstrating the Potential of Genetic Medicine
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- SonoThera ™, a biotechnology company dedicated to treating the root cause of human diseases through developing the next generation of genetic medicines, today announced it will present new data at the 28 th annual American Society of Gene and Cell Therapy (ASGCT) meeting being held May 13-17 th in New Orleans, Louisiana. This data continues to demonstrate our unique capabilities, including targeted full-length human dystrophin expression in muscle, and delivering oversized genetic payloads in a durable, safe, redosable and noninvasive manner. Share An oral presentation will focus on the treatment of Hemophilia A while new data in the poster session targets the treatment of Duchenne Muscular Dystrophy (DMD). Both presentations at ASGCT feature SonoThera's proprietary, nonviral, non-invasive approach using ultrasound-mediated delivery, which enables broad, highly targeted biodistribution of diverse genetic medicine formats without size restrictions, and which enables genetic medicines that can be re-dosed, are safe and well tolerated and are cost-effective. 'We're pleased to be presenting new data at ASGCT highlighting the potential of ultrasound mediated delivery in solving the key challenges in genetic medicine,' said Kenneth Greenberg, PhD, CEO of SonoThera. 'This data continues to demonstrate our unique capabilities, including targeted full-length human dystrophin expression in muscle, and delivering oversized genetic payloads in a durable, safe, redosable and noninvasive manner. We look forward to sharing our latest data at this year's annual meeting.' Presentation Details: Oral Presentation: Development of a Novel, Non-Viral Genetic Therapy for Hemophilia A Utilizing Durable, Redosable, and Titratable Approach of Ultrasound-Mediated Delivering of an Oversized Episomal hFVIII DNA Vector. Session Title: Liver Targeted Genetic Therapies Presenter: Ivan Krivega, PhD Session Date/Time: Wednesday, May 14, 2025, 2:30pm – 2:45pm CT. Room: 288-290 Poster Presentation: Non-Viral Genetic Medicine for Targeted Delivery of Full-Length Dystrophin to Skeletal, Cardiac, and Diaphragm Muscles in DMD Mouse Models and Non-Human Primates. Session: Disease Models and Clinical Applications: B8 - Musculo-skeletal Diseases Presenter: Ivan Krivega, PhD Session Date/Time: Thursday, May 15, 2025, 5:30pm – 7:00pm CT Location: Poster Hall, Hall I2 About SonoThera™ SonoThera is a biotechnology company dedicated to treating the root cause of human diseases through developing the next generation of genetic medicines. Our nonviral technology is designed to overcome all prevailing limitations of genetic medicine, enabling a pipeline of products which leverages our novel capabilities. Using ultrasound-mediated delivery (UMD), we are developing a proprietary, non-invasive approach which enables broad, highly targeted biodistribution, delivery of diverse genetic payloads without size restriction, in a redosable manner designed to be safe, well-tolerated and cost-effective. Founded by Drs. Kenneth Greenberg, Michael Davidson, and Steve Feinstein, SonoThera is headquartered in South San Francisco.
Technical.ly
04-04-2025
- Business
- Technical.ly
Spark Therapeutics files notice to lay off 300 employees this year
Cell and gene therapy standout Spark Therapeutics is undergoing its second shakeup in a month, with plans to lay off about half of its workforce. Several hundred people will be affected. A WARN notice, which companies file to provide advance notice of layoffs, reported 298 eliminated positions in the Philadelphia region. A Spark Therapeutics spokesperson told the Philadelphia Business Journal on Thursday it would be laying off 337 of its almost 650 employees. These changes are expected to occur in three waves: in May, July and at the end of 2025. The remaining 310 employees will be incorporated into parent company Roche, a multinational pharmaceutical company. Spark first announced on January 30 the decision to integrate more of its work into Roche, spokesperson Denise Bradley told The impacted employees will be eligible for severance, outplacement services and will be able to apply for other roles within Roche. Spark's plans for its University City-based Gene Therapy Innovation Center in Philadelphia have not changed, Bradley said. Last month, Roche classified the former startup as a financial loss following the end of its trial for a hemophilia A gene therapy treatment, the Philadelphia Inquirer reported. The company is still working on a new Hemophilia A gene product, Spark previously told The layoff announcement comes less than a year after Spark's previous workforce reduction, when it let less than 50 of its employees go in July 2024. At the end of last year, the company welcomed Roche veteran Sylke Poehling as its new CEO, replacing Ron Phillip, who had been in the role since 2022. Spark's year of downsizing Recent struggles at Spark, which was founded by Jeffrey Marrazzo in 2013 and was considered a big Philadelphia success story, indicate the need for the gene therapy sector to make manufacturing more cost effective, Rebecca Grant, senior director of life sciences and innovation for the city's Department of Commerce, previously told But the company itself heavily contributed to the development of the industry as a whole. 'They really created a lot of recognition for gene therapy and innovation,' Grant said. 'Now more people understand what gene therapy means and how it can literally cure disease.' In 2021, the company announced plans for a 500,000-square-foot Gene Therapy Innovation Center in University City. At the time, Spark said the new site would house over 500 jobs. The Innovation Center is still expected to be completed next year, Spark spokesperson Bradley previously told The Penn spinout is known for developing the first FDA-approved gene therapy, Luxturna. Pharma giant Roche acquired Spark in 2019 for $4.8 billion, the largest VC-backed exit in Philadelphia at the time. 'Gene therapy is not a huge sector, and Spark was a trailblazer,' Dean Miller, president of the Philadelphia Alliance for Capital and Technologies, previously said. '[It's] never easy when your trailblazer starts to disappear a little further.' Sarah Huffman is a 2022-2024 corps member for Report for America, an initiative of The Groundtruth Project that pairs young journalists with local newsrooms. This position is supported by the Lenfest Institute for Journalism.
Globe and Mail
28-01-2025
- Business
- Globe and Mail
Hemophilia A FDA Approvals, Clinical Trials, Pipeline Insights, Drugs and Companies
DelveInsight's, 'Hemophilia A Pipeline Insight' report provides comprehensive insights about 40+ companies and 40+ pipeline drugs in Hemophilia A pipeline landscape. It covers the Hemophilia A pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Hemophilia A therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space. Explore the comprehensive insights by DelveInsight and stay ahead in understanding the Hemophilia A Treatment Landscape. Click here to read more @ Hemophilia A Pipeline Outlook Key Takeaways from the Hemophilia A Pipeline Report In January 2025:- CSL Behring:- A Phase 3, Open-label, Multicenter, Pharmacokinetics, Efficacy, and Safety Study of a Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Previously Treated Patients (PTPs) With Hemophilia A. For bridging the available global clinical data of rVIII-SingleChain, with the Chinese population, the aim of this study in China is to investigate the pharmacokinetics (PK) of rVIII-SingleChain after an initial and repeat dose and to assess efficacy and safety during 2 to 3 times weekly prophylaxis treatment with rVIII-SingleChain in male Chinese PTPs with severe hemophilia A (FVIII activity less than [<] 1%). In January 2025:- Octapharma:-Female patients with mild to moderate haemophilia A will often need FVIII concentrates to provide haemostatic cover during major surgery. This prospective, open-label, non-controlled, single-arm, multinational, multicentre study aims to evaluate the overall perioperative haemostatic efficacy of Nuwiq in women/girls over 12 with haemophilia A undergoing major surgery requiring FVIII treatment. In January 2025:- Bayer:- A Phase 1/2 Open-label Safety and Dose-finding Study of BAY2599023 (DTX201), an Adeno-associated Virus (AAV) hu37-mediated Gene Transfer of B-domain Deleted Human Factor VIII, in Adults With Severe Hemophilia A. By replacing the defective gene with a healthy copy the human body may produce clotting factor on its own. Hemophilia A is a bleeding disorder in which the human body does not have enough clotting factor VIII, a protein that controls bleeding. Researcher want to find the optimal dose of BAY 2599023 (DTX201) so that the body may produce enough clotting factor on its own. DelveInsight's Hemophilia A pipeline report depicts a robust space with 40+ active players working to develop 40+ pipeline therapies for Hemophilia A treatment. The leading Hemophilia A Companies such as Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd., Bayer, Ultragenix Pharmaceutical, Spark Therapeutics, Octapharma, ApcinteX Ltd., Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Expression Therapeutics LLC, CSL Behring, and others. Promising Hemophilia A Pipeline Therapies such as Emicizumab, rFVIIa, aPCC, BAX 888, OBIZUR, BIIB031 (rFVIIIFc), Recombinant Human Coagulation FVIII, and others. Discover groundbreaking developments in Hemophilia A therapies! Gain in-depth knowledge of key Hemophilia A clinical trials, emerging drugs, and market opportunities @ Hemophilia A Clinical Trials Assessment Hemophilia A Emerging Drugs Profile AGN-193408: Allergan Allergan is conducting a multicenter, open-label, dose escalation (Cohort 1) to masked, randomized, parallel-group (Cohort 2) study to evaluate the safety and efficacy of AGN-193408 SR in participants with open-angle glaucoma or ocular hypertension. It is an implant containing preservative-free AGN-193408 dispersed in a biodegradable polymer matrix. OCTA101: Octapharma Octapharma is evaluating OCTA101 (a human-cl rhFVIII and recombinant human von Willebrand Factor fragment dimer) under a Phase 1/2 study, which will be a dose escalation study in adults in 5 cohorts, with the main purpose to assess the safety of subcutaneous injection of OCTA101 (in previously treated adult patients with severe hemophilia A. The study also aims to assess the pharmacokinetics (PK) characteristics, dose proportionality, and subcutaneous bioavailability of OCTA101 compared with intravenous administration of Nuwiq (Human-cl rh FVIII), in order to define the prophylactic treatment (dose and injection interval) that would result in protective trough levels of FVIII:C for future Phase 3 studies. By Data Monitoring Committee recommendation, patients enrolled in cohorts 1, 2 and 3 will proceed to 3-month prophylactic treatment to receive daily dosing of OCTA101 for 3 months. Valoctocogene roxaparvovec: BioMarin Pharmaceuticals Valoctocogene roxaparvovec, is an investigational gene therapy in clinical trials for the treatment of Hemophilia A and has not been determined to be safe or effective. Valoctocogene roxaparvovec is administered as a single infusion. The ongoing clinical trials will determine if the new gene will enable the body to produce factor VIII. Following infusion, clinical trial subjects are being evaluated to determine the safety profile, changes in Factor VIII activity levels, changes in factor replacement usage, changes in reported bleeds requiring factor replacement, and quality of life measures, among other endpoints. Hemophilia A Companies Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd., Bayer, Ultragenix Pharmaceutical, Spark Therapeutics, Octapharma, ApcinteX Ltd., Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Expression Therapeutics LLC, CSL Behring, and others. Hemophilia A pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as Oral Intravenous Subcutaneous Molecule Type Hemophilia A Products have been categorized under various Molecule types such as Small molecules Gene Therapies Bispecific antibodies Recombinant proteins Fusion Proteins Coagulants Blood coagulation factor replacements Transform your understanding of the Hemophilia A Pipeline! See the latest progress in drug development and clinical research @ Hemophilia A Market Drivers and Barriers, and Future Perspectives Scope of the Hemophilia A Pipeline Report Coverage- Global Hemophilia A Companies- Hoffmann-La Roche, Chugai Pharmaceutical, Shire, Pfizer, BioMarin Pharmaceutical, Sinocelltech Ltd., Bayer, Ultragenix pharmaceutical, Spark Therapeutics, Octapharma, ApcinteX Ltd., Chia Tai Tianqing Pharmaceutical Group Co., Ltd., Expression Therapeutics LLC, CSL Behring, and others. Hemophilia A Pipeline Therapies- Emicizumab, rFVIIa, aPCC, BAX 888, OBIZUR, BIIB031 (rFVIIIFc), Recombinant Human Coagulation FVIII, and others. Hemophilia A Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination Hemophilia A Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III Stay Ahead in Genetic Disorders Research–Access the Full Hemophilia A Pipeline Analysis Today! @ Hemophilia A Drugs and Companies Table of Content 1. Introduction 2. Executive Summary 3. Hemophilia A: Overview 4. Pipeline Therapeutics 5. Therapeutic Assessment 6. Hemophilia A - DelveInsight's Analytical Perspective 7. In-depth Commercial Assessment 8. Hemophilia A Collaboration Deals 9. Late Stage Products (Pre-registration) 10. Valoctocogene roxaparvovec: BioMarin Pharmaceutical 11. Late Stage Products (Phase III) 12. Giroctocogene fitelparvovec: Pfizer/ Sangamo Therapeutics 13. Mid Stage Products (Phase II) 14. NNC0365-3769 A (Mim8): Novo Nordisk A/S 15. Early Stage Products (Phase I/II) 16. BAX 888: Baxalta / Shire 17. BAY2599023: Bayer / Ultragenix pharmaceutical 18. Early Stage Products (Phase I) 19. Gene therapy: Expression Therapeutics, LLC 20. Drug profiles in the detailed report….. 21. Hemophilia A- Market Drivers and Barriers 22. Appendix About Us DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve. Media Contact Company Name: DelveInsight Business Research LLP Contact Person: Yash Bhardwaj Email: Send Email Phone: 9650213330 Address: 304 S. 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