Latest news with #DilatedCardiomyopathy
Yahoo
15-07-2025
- Business
- Yahoo
Rocket Pharmaceuticals Receives FDA IND Clearance for BAG3-DCM Gene Therapy RP-A701
Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) is one of the best low priced pharma stocks to buy now. On June 30, Rocket Pharmaceuticals announced that the US FDA cleared its Investigational New Drug/IND application for RP-A701. RP-A701 is a first-in-class AAVrh.74-based gene therapy candidate designed to treat BAG3-associated Dilated Cardiomyopathy (BAG3-DCM). BAG3-DCM is a rare and inherited heart condition caused by mutations in the BAG3 gene (Bcl2-associated athanogene 3). This leads to early-onset, rapidly progressing heart failure characterized by progressive ventricular enlargement and impaired systolic function, resulting in high morbidity and premature mortality. Scientific equipment in a lab setting, revealing the cutting edge of biotechnology research. A loss of BAG3 leads to the accumulation of misfolded and damaged proteins, impairing the heart's ability to contract. Rocket Pharmaceuticals estimates that ~30,000 individuals in the US are affected by BAG3-DCM. Current therapies don't prevent disease progression and are associated with complications. Rocket Pharmaceuticals Inc. (NASDAQ:RCKT) is a late-stage biotechnology company that develops gene therapies for rare diseases in the US. While we acknowledge the potential of RCKT as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey.
Yahoo
18-06-2025
- Business
- Yahoo
Dilated Cardiomyopathy (DCM) Market View 2025: Marketed Drugs, Pricing and Reimbursement, Pipeline, Clinical Trials, Epidemiology, Commercial and Deals Landscape
Discover the evolving landscape of Dilated Cardiomyopathy Therapeutics. In 2024, anticipate over 3 million DCM cases in 16 countries. Key treatments include beta-blockers and ACE inhibitors, while 34 molecules progress in the pipeline. Explore market insights, competitive analyses, and future industry catalysts. Dublin, June 18, 2025 (GLOBE NEWSWIRE) -- The "Dilated Cardiomyopathy (DCM): Market View" report has been added to report presents a data-driven overview of the current and future competitive landscape in Dilated Cardiomyopathy (DCM) Therapeutics. In 2024, there are expected to be over three million diagnosed prevalent cases of DCM across 16 countries included in the forecast. Treatment is primarily focused on symptom management, using beta-blockers, ACE inhibitors, ARBs, MRAs, and SGLT2 inhibitors. Currently, 34 molecules are in development for DCM across various pipeline stages. Over the past decade, the U.S. led in clinical trial activity with nearly 110 trial sites. In terms of deal activity, acquisitions were most common in North America, while licensing agreements dominated in South and Central America, Africa, and the Asia-Pacific regions. Reasons to Buy Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Develop business strategies by understanding the trends shaping and driving the Dilated Cardiomyopathy Therapeutics market. Drive revenues by understanding the key trends, innovative products and technologies, and companies likely to impact the global Dilated Cardiomyopathy Therapeutics market in the future. Formulate effective sales and marketing strategies by understanding the competitive landscape and analyzing the performance of various competitors. Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage. Organize your sales and marketing efforts by identifying the market categories that present the maximum opportunities for consolidations, investments, and strategic partnerships. Components of the report include: Disease Landscape Disease Overview Epidemiology Overview Treatment Overview Marketed Products Assessment Breakdown by Mechanism of Action, Route of Administration Product Profiles with Sales Forecast Pricing and Reimbursement Assessment Annual Therapy Cost Time to Pricing and Time to Reimbursement Pipeline Assessment Breakdown by Development Stage, Mechanism of Action, Molecule Type, Route of Administration Product Profiles with Sales Forecast Late-to-mid-stage Pipeline Drugs Phase Transition Success Rate and Likelihood of Approval Clinical Trials Assessment Breakdown of Trials by Phase, Status, Virtual Components, Sponsors, Geography, and Endpoint Status Enrolment Analytics, Site Analytics, Feasibility Analysis Deals Landscape Mergers, Acquisitions, and Strategic Alliances by Region Overview of Recent Deals Commercial Assessment Key Market Players Future Market Catalysts Key Topics Covered: 1 Preface1.1 Contents1.2 Report Scope1.3 List of Tables and Figures1.4 Abbreviations2 Key Findings3 Disease Landscape3.1 Disease Overview3.2 Epidemiology Overview3.3 Treatment Overview4 Marketed Drugs Assessment4.1 Leading Marketed Drugs4.2 Overview by Mechanism of Action4.3 Overview by Molecule Type4.4 Product Profiles and Sales Forecast5 Pricing and Reimbursement Assessment5.1 Annual Cost of Therapy5.2 Time to Pricing and Reimbursement6 Pipeline Drugs Assessment6.1 Mid-to-late-stage Pipeline Drugs6.2 Overview by Development Stage6.3 Overview by Mechanism of Action6.4 Overview by Molecule Type6.5 Drug Specific Phase Transition Success Rate (PTSR) and Likelihood of Approval (LoA)6.6 Therapy Area and Indication-specific PTSR and LoA7 Clinical Trials Assessment7.1 Historical Overview7.2 Overview by Phase7.3 Overview by Status7.4 Overview by Phase for Ongoing and Planned Trials7.5 Trials with Virtual Components7.6 Overview of Trials by Geography7.7 Single-Country and Multinational Trials by Region7.8 Top 20 Sponsors with Breakdown by Phase7.9 Top 20 Sponsors with Breakdown by Status7.10 Overview by Endpoint Status7.11 Overview by Race and Ethnicity7.12 Enrollment Data7.13 Top 20 countries for Trial Sites7.14 Top 20 Sites Globally7.15 Feasibility Analysis - Geographic Overview7.16 Feasibility Analysis - Benchmark Models8 Deals Landscape8.1 Mergers, Acquisitions, and Strategic Alliances by Region8.2 Recent Mergers, Acquisitions, and Strategic Alliances9 Commercial Assessment9.1 Key Market Players10 Future Market Catalysts11 Appendix For more information about this report visit About is the world's leading source for international market research reports and market data. 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