Latest news with #CSLBehring
Yahoo
12-06-2025
- Business
- Yahoo
4 Emerging Graft versus Host Disease Therapies That Could Change the Treatment Landscape
Key companies, such as CSL Behring, Equillium, Biocon, and medac, are advancing their assets through late-stage graft versus host disease clinical trials, driving innovation in the graft versus host disease market and creating significant growth opportunities. LAS VEGAS, June 12, 2025 /PRNewswire/ -- Graft versus host disease (GvHD) is an immune-driven disorder caused by a complex interplay between the donor's and recipient's adaptive immune systems. It typically manifests in two main forms: acute (aGvHD) and chronic (cGvHD). As per DelveInsight's analysis, approximately 60,000 allogeneic transplants and around 55,000 GvHD cases occurred in the 7MM in 2024. These numbers are projected to grow at a notable CAGR over the forecast period from 2025 to 2034. Corticosteroids like prednisone and methylprednisolone are the primary first-line treatments, often combined with other immunosuppressants. Mild GcHD is managed with topical steroids, while systemic cases require stronger immunosuppressive therapy. Several treatments for GvHD have received FDA approval in the US, including ORENCIA (abatacept), JAKAFI/JAKAVI (ruxolitinib), IMBRUVICA (ibrutinib), and REZUROCK (belumosudil), among others. In the upcoming GvHD treatment market landscape, there are a plethora of companies investigating agents in various stages of development. To know more about the graft versus host disease clinical trials market, visit @ Graft versus Host Disease Market DelveInsight estimates that the market size for GvHD is expected to grow from USD 1.4 billion in 2024 with a significant CAGR of 8.2% by 2034. This anticipated growth is driven by advancements in treatment options, greater healthcare access, and a rising prevalence of the condition, which together foster higher demand for innovative and effective therapies. The current pipeline for graft versus host disease includes a range of drugs with diverse mechanisms of action (MoA). CSL964 (Alpha 1 Antitrypsin), EQ001 (Itolizumab; Bmab600), MaaT013, and MC0518 are the most promising drugs that are in the late-stage of development for GvHD treatment. Ongoing research and current trials have the potential to change the GvHD market. Keen to know how the GvHD market will evolve by 2034? Find out @ Graft versus Host Disease (GvHD) Market Forecast Apart from this, several GvHD drugs currently in the early stages of development include RLS-0071 by ReAlta Life Sciences, Vimseltinib by Deciphera Pharmaceuticals, ASC-930 by ASC Therapeutics, RGI-2001 by REGiMMUNE, CYP-001 by Cynata Therapeutics, arsenic trioxide (As2O3) by BioSenic (Medsenic), TRX103 (Tregs) by Tr1X, TCD601 (Siplizumab) by ITB-MED, F-652 by Evive Biotech, RHPRG4 by Lubris BioPharma, XBI302 by Xbiome, RG6287 by Genentech, ALTB-168 by AltruBio, and SER-155 by Seres Therapeutics. Now, let's examine the late-stage pipeline therapies under investigation for GvHD treatment CSL Behring's ZEMAIRA CSL964 Alpha-1 Antitrypsin, an Alpha1-Proteinase Inhibitor (A1-PI) developed by CSL Behring, is being studied for the treatment of steroid-refractory acute graft-versus-host disease (aGvHD) and for the prevention of aGvHD in high-risk patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). It is currently in Phase III clinical trials for the treatment of steroid-refractory aGvHD and is also being evaluated in Phase II/III trials for its potential in preventing aGvHD. Equillium/Biocon's EQ001 EQ001 (Itolizumab; Bmab600) is a first-in-class immune-modulating antibody that targets CD6 to suppress the activation and migration of harmful T cells responsible for releasing pro-inflammatory cytokines in autoimmune and inflammatory conditions such as GvHD, moderate-to-severe uncontrolled asthma, and lupus nephritis. By acting on the CD6-ALCAM signaling pathway, Itolizumab selectively inhibits pathogenic effector T cells (Teffs) while preserving regulatory T cells (Tregs), which are essential for immune system balance. Currently, EQ001 is undergoing a Phase III clinical trial in combination with corticosteroids as a first-line therapy for March 2025, Equillium reported topline Phase III EQUATOR trial results for itolizumab in first-line aGVHD. While the study did not show a meaningful difference in CR or ORR at Day 29 versus placebo, itolizumab demonstrated statistically significant and clinically meaningful improvements in longer-term outcomes, including CR at Day 99, duration of response, and failure-free survival. In April 2025, the FDA declined to grant Breakthrough Therapy designation or support an Accelerated Approval pathway for itolizumab, citing limitations in the EQUATOR study data. Discover which therapies are expected to grab major GvHD market share @ Graft versus Host Disease Treatment Market MaaT Pharma's MaaT013 MaaT013 is a standardized, donor-derived microbiome ecosystem therapy characterized by high richness and diversity. It includes BUTYCORE, a consortium of bacterial species known for producing anti-inflammatory short-chain fatty acids. The therapy is designed to reestablish the balance between the patient's gut microbiome and immune system, aiming to enhance immune tolerance and responsiveness, thereby addressing steroid-resistant, gastrointestinal-dominant aGvHD. MaaT013 has been granted Orphan Drug Designation (ODD) by both the US FDA and the EMA. In December 2024, MaaT Pharma shared encouraging updated results from its Early Access Program at the ASH 2024 Annual Meeting. Subsequently, in January 2025, the company announced promising topline findings from the Phase III ARES trial, where MaaT013 achieved a 62% overall gastrointestinal response rate by Day 28, marking a significant advancement as a third-line treatment for GI-aGvHD. medac's MC0518 MC0518 is an investigational mesenchymal stromal cell (MSC) therapy developed by Medac GmbH, currently undergoing clinical trials for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGvHD) following allogeneic hematopoietic stem cell transplantation. This therapy leverages the immunomodulatory properties of MSCs to mitigate the severe inflammatory responses characteristic of SR-aGvHD, a condition where the donor's immune cells attack the recipient's tissues despite steroid treatment. The IDUNN trial, a pivotal Phase III study, is evaluating the efficacy and safety of MC0518 compared to the best available therapy (BAT) in pediatric and adolescent patients. The primary endpoint is the overall response rate (ORR) at Day 28, with secondary objectives including overall survival (OS) up to 24 months and freedom from treatment failure (FFTF) within six months. Preclinical assessments have demonstrated that MC0518 is well-tolerated, with no evidence of tumorigenicity or significant adverse effects in animal models. Discover more about drugs for GvHD in development @ Graft versus Host Disease Clinical Trials The anticipated launch of these emerging therapies for GvHD 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 GvHD market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. DelveInsight's latest published market report, titled as Graft versus Host Disease 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 GvHD country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The GvHD market report offers epidemiological analysis for the study period 2020–2034 in the 7MM, segmented into: Total allogenic transplant cases Total Graft Versus Host Disease Cases Type-specific Cases of Graft Versus Host Disease Acute Graft Versus Host Disease Cases by Grading Acute Graft Versus Host Disease Cases by Organ Involvement Chronic Graft Versus Host Disease Cases by Grading Chronic Graft Versus Host Disease Cases by Organ Involvement Total Treated Cases of Graft Versus Host Disease Mortality Adjusted Treated Cases of Graft Versus Host Disease The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM GvHD market. Highlights include: 10-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 GvHD 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 GvHD market. Also, stay abreast of the mitigating factors to improve your market position in the GvHD therapeutic space. Related Reports Graft versus Host Disease Epidemiology Forecast Graft versus Host Disease Epidemiology Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted GvHD epidemiology in the 7MM, i.e., the United States, EU4 (Germany, Spain, Italy, France) and the United Kingdom, and Japan. Graft versus Host Disease Pipeline Graft versus Host Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key GvHD companies, including Abbisko Therapeutics, Equillium, Theriva Biologics, Seres Therapeutics, CytoMed Therapeutics, Beijing Tide Pharmaceutical, CTI BioPharma, ViGenCell, Lipella Pharmaceuticals, Cellestia Biotech, Seres Therapeutics, Jiangsu HengRui Medicine Therapeutics, Genentech, AltruBio, Orca Bio, GSK, Amgen, among others. Acute Graft versus Host Disease Pipeline Acute Graft versus Host Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key acute GvHD companies, including MaaT Pharma, Medac, CSL Behring, Humanigen, Ironwood Pharmaceuticals, ReAlta Life Sciences, Roche, Incyte Corporation, among others. Ocular Graft versus Host Disease Pipeline Ocular Graft versus Host Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key ocular GvHD companies, including Cambium Medical Technologies, Glia LLC., Ocular Discovery, Selagine, 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 UsShruti Thakur info@ +14699457679 Logo: View original content: SOURCE DelveInsight Business Research, LLP
Yahoo
10-06-2025
- Health
- Yahoo
Alpha-1 Antitrypsin Deficiency Market Research 2025-2035: Rising Demand for AATD Treatments Fuels Expansion with North America Leading
The global alpha-1 antitrypsin deficiency (AATD) market is expanding, driven by advances in diagnosis, treatment, and growing awareness. Key players like Grifols, CSL Behring, and Pfizer lead innovation. Market growth is fueled by rising disease prevalence and improved therapies but faces high costs. Dublin, June 10, 2025 (GLOBE NEWSWIRE) -- The "Alpha-1 Antitrypsin Deficiency Market - A Global and Regional Analysis: Focus on Treatment, Distribution Channel, and Regional Analysis - Analysis and Forecast, 2025-2035" report has been added to global alpha-1 antitrypsin deficiency (AATD) market is experiencing growth, fueled by advances in diagnosis, treatment options, and increasing awareness of the condition. AATD is a genetic disorder where the body produces insufficient alpha-1 antitrypsin, a protein that protects the lungs and liver. Its deficiency can lead to chronic obstructive pulmonary disease (COPD), emphysema, and liver cirrhosis. The condition often remains underdiagnosed, which presents an opportunity for early detection and primary treatment for AATD is augmentation therapy, which involves the infusion of AAT protein to restore deficient levels in the bloodstream. This therapy is designed to protect the lungs and slow the progression of emphysema. Other treatment options include symptomatic management, such as bronchodilators and corticosteroids. Research continues to explore gene therapies and novel treatments to better address the underlying genetic causes of market is expanding due to rising awareness about AATD, improvements in diagnostic tools, and innovations in treatment options. Key drivers include the increasing prevalence of the disease, particularly in older populations, and the growing availability of specialized therapies. However, challenges such as high treatment costs and the need for long-term care remain significant factors impacting the global alpha-1 antitrypsin deficiency market growth. The North America alpha-1 antitrypsin deficiency market is expected to lead globally due to advanced healthcare infrastructure, high awareness, and significant research investments. The market is primarily driven by augmentation therapy, which involves the administration of alpha-1 proteinase inhibitors to manage disease progression. Enhanced genetic testing and early diagnosis have improved treatment outcomes. Hospitals and specialty clinics serve as key treatment settings, offering specialized care. The region's favorable reimbursement policies and commitment to research and development further support market growth, positioning North America as a dominant force in the global alpha-1 antitrypsin deficiency treatment summary, the global alpha-1 antitrypsin deficiency market is poised for growth, with a strong focus on improving diagnostic accuracy and providing effective treatments, offering significant opportunities for stakeholders in the healthcare and pharmaceutical industries. How Can This Report Add Value to an Organization?Product/Innovation Strategy: Product launches and innovations in the global alpha-1 antitrypsin deficiency market are focused on advancing treatment options to improve patient care. These innovations aim to enhance the efficacy of therapies and streamline the detection and management of the disease. Key players in the global alpha-1 antitrypsin deficiency market, such as Grifols, CSL Behring, Takeda, GlaxoSmithKline plc, and Pfizer Inc., have been involved in offering of therapies for alpha-1 antitrypsin Strategy: Enterprises led by market leaders in the global alpha-1 antitrypsin deficiency market are continuously working on updating their product portfolios with innovative treatments to maintain competitiveness. A detailed competitive benchmarking of the key players in the global alpha-1 antitrypsin deficiency market has been conducted, providing insights into how these companies compare in terms of product offerings, market share, and innovation. This benchmarking provides readers with a clear understanding of the market landscape and the positions of the leading players. Additionally, comprehensive competitive strategies, such as partnerships, agreements, and collaborations, will help readers identify untapped revenue opportunities in the global alpha-1 antitrypsin deficiency Increasing demand for Alpha-1 Antitrypsin Deficiency therapies is anticipated to support the growth of the global alpha-1 antitrypsin deficiency market during the forecast period 2025-2035. The global alpha-1 antitrypsin deficiency market is expected to grow at a significant rate due to advancements in diagnostic technologies, the development of innovative therapies, and increasing awareness among patients and healthcare providers. Recent Developments: Regulatory Activities: In March 2025, Beam Therapeutics announced that the U.S. FDA cleared its Investigational New Drug (IND) application for BEAM-302, a base-editing therapy for AATD. This marks the first clinical advancement of a base-editing approach for AATD, with initial Phase 1/2 trial data showing promising results in correcting the PiZ mutation associated with the disease. Regulatory Activities: In February 2025, Grifols completed enrollment of the second cohort in its Phase 1/2 study for Alpha-1 15%, a subcutaneous AATD treatment. This development could provide patients with a more convenient administration option, enhancing treatment adherence and quality of life. Acquisition: In May 2024, Sanofi completed its acquisition of Inhibrx, Inc., gaining control of INBRX-101, a recombinant human AAT-Fc fusion protein under development for AATD treatment. INBRX-101 aims to normalize serum AAT levels with less frequent dosing, potentially offering a significant advancement in AATD therapy. Market Dynamics The following are the drivers for the global alpha-1 antitrypsin deficiency market: Increasing Awareness and Early Diagnosis Advancements in Treatment Options The global alpha-1 antitrypsin deficiency market is expected to face some limitations too, due to the following challenges: High Treatment Costs Limited Awareness in Emerging Markets Some of the prominent names established in this market are: Grifols CSL Behring Takeda Pharmaceuticals LFB Biotechnologies GlaxoSmithKline plc AstraZeneca Merck Pfizer Inc. Key Topics Covered: Executive SummaryScope and Definition1. Global Alpha-1 Antitrypsin Deficiency Market: Market Outlook1.1 Industry Outlook1.1.1 Market Overview and Ecosystem1.1.2 Market Trends1.1.3 Epidemiological Analysis of Alpha-1 Antitrypsin Deficiency1.1.3.1 By Region1.1.4 Clinical Trials1.1.4.1 By Phase1.1.4.2 By Sponsor Type1.1.5 Regulatory Landscape / Compliance1.1.5.1 Legal Requirement and Framework in the U.S.1.1.5.2 Legal Requirement and Framework in the E.U.1.1.5.3 Legal Requirement and Framework in Japan1.1.5.4 Legal Requirement and Framework in Rest-of-the-World1.2 Market Dynamics1.2.1 Impact Analysis1.2.2 Market Drivers1.2.3 Market Restraints1.2.4 Market Opportunities2. Global Alpha-1 Antitrypsin Deficiency Market, By Treatment, $Million, 2023-20352.1 Overview2.2 Augmentation Therapy2.3 Bronchodilators2.4 Corticosteroids2.5 Oxygen Therapy3. Global Alpha-1 Antitrypsin Deficiency Market, By Distribution Channel, $Million, 2023-20353.1 Overview3.2 Hospitals Pharmacy3.3 Retail Pharmacies3.4 Online Pharmacies4. Global Alpha-1 Antitrypsin Deficiency Market, By Region, $Million, 2023-20354.1 North America4.1.1 Key Findings4.1.2 Market Dynamics4.1.3 Market Sizing and Forecast4.1.3.1 North America Alpha-1 Antitrypsin Deficiency Market (by Distribution Channel)4.1.3.2 North America Alpha-1 Antitrypsin Deficiency Market (by Country)4.1.3.2.1 U.S.4.1.3.2.2 Canada4.2 Europe4.2.1 Key Findings4.2.2 Market Dynamics4.2.3 Market Sizing and Forecast4.2.3.1 Europe Alpha-1 Antitrypsin Deficiency Market (by Distribution Channel)4.2.3.2 Europe Alpha-1 Antitrypsin Deficiency Market (by Country)4.3 Asia-Pacific4.4 Rest-of-the-World5. Global Alpha-1 Antitrypsin Deficiency Market - Competitive Landscape and Company Profiles5.1 Competitive Landscape5.1.1 Growth Share Matrix (2024)5.1.1.1 By Treatment5.1.2 Key Strategies and Developments by Company5.1.2.1 Funding Activities5.1.2.2 Mergers and Acquisitions5.1.2.3 Regulatory Approvals5.1.2.4 Partnerships, Collaborations and Business Expansions5.1.3 Key Developments Analysis5.2 Company Profiles5.2.1 Company Overview5.2.2 Product Portfolio5.2.3 Target Customers/End Users5.2.4 Analyst View6. Research Methodology For more information about this report visit About is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. CONTACT: CONTACT: Laura Wood,Senior Press Manager press@ For E.S.T Office Hours Call 1-917-300-0470 For U.S./ CAN Toll Free Call 1-800-526-8630 For GMT Office Hours Call +353-1-416-8900Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Reuters
10-02-2025
- Business
- Reuters
Australia's CSL reports half-year profit jump on strong demand for immunoglobulin products
Feb 11 (Reuters) - Australia's biotech giant CSL Ltd ( opens new tab reported a 5% rise in its first-half profit on Tuesday, boosted by growth in its core immunoglobulin business across geographies, while low U.S. immunisation rates weighed on its influenza products unit. Strong underlying demand for CSL's immunoglobulin (Ig) products for the treatment of patients with chronic immune system disorders drove up sales. Total revenue from the company's largest division, Behring, for the six months ended December 31 rose 10% to $5.74 billion. CSL Behring includes the Ig products segment, whose sales grew 15% year over year. The Melbourne-based firm has been in the process of improving CSL Behring's gross profit margins by introducing the efficient "RIKA Plasma Donation System" technology across its plasma collection business. The company expects the RIKA roll-out to be completed across CSL Plasma by the end of the financial year, it said. Meanwhile, revenue for CSL Seqirus, the company's influenza-related products division, fell 9%. "CSL Seqirus was negatively impacted by significantly low influenza immunisation rates, particularly in the United States," CEO Paul McKenzie said. The firm said that its underlying net profit after tax attributable for the half year was $2.11 billion, on a constant currency basis, compared with $2.06 billion reported a year ago. On a reported currency basis, its profit was $2.07 billion, missing a Visible Alpha consensus estimate of $2.16 billion. CSL reassured investors that it continues to expect annualised double-digit earnings growth over the medium term. It declared an interim dividend of $1.30 per share, higher than $1.19 per share declared a year ago.
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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