The Hunter Syndrome Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.
“Hunter Syndrome Pipeline Insight, 2024” report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Hunter Syndrome Market.
Some of the key takeaways from the Hunter Syndrome Pipeline Report:
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Companies across the globe are diligently working toward developing novel Hunter Syndrome treatment therapies with a considerable amount of success over the years.
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Hunter Syndrome companies working in the treatment market are Capsida Biotherapeutics, JCR Pharmaceuticals, Takeda, Green Cross Corporation/GC Pharma, Denali Therapeutics, Regenxbio, Sangamo Therapeutics, AVROBIO, and others, are developing therapies for the Hunter Syndrome treatment
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Emerging Hunter Syndrome therapies in the different phases of clinical trials are- CAP 001, JR-141, TAK-609/SHP609/HGT-2310, GC1111 (Hunterase), DNL310, RGX-121, SB-913, AVR-RD-05, and others are expected to have a significant impact on the Hunter Syndrome market in the coming years.
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In February 2024, GC Biopharma, a South Korean biopharmaceutical company led by CEO Eun-Chul Huh, announced on February 14th that it has shared updates on its lysosomal storage disease (LSD) treatments at the WORLDSymposium 2024. During the symposium, GC Biopharma delivered an oral presentation on the Phase III clinical trial results of “Hunterase,” its treatment for Hunter syndrome, and presented posters on the non-clinical results of “GC1130A,” a treatment for Sanfilippo syndrome type A (MPS IIIA), and “GC1134A/HM15421,” a treatment for Fabry disease.
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In November 2023, JCR Pharmaceuticals administered the initial dose to a patient in its Phase I/II clinical trial of JR-441, targeting Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome Type A). This trial is being conducted at the International Center for Lysosomal Disorders (ICLD) at the University Hospital of Hamburg Eppendorf (UKE) in Germany. The study, based at a single center, aims to enroll 12 patients between the ages of one and 18, who are at various stages of the disease.
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In June 2023, Denali Therapeutics Inc. (NASDAQ: DNLI) has reported new interim findings from the ongoing open-label, single-arm Phase 1/2 trial of DNL310 (ETV:IDS) in children diagnosed with MPS II (Hunter syndrome). At the time of the interim analysis, 13 participants who completed two years of treatment showed a significant mean reduction of 64% (p<0.001) in serum neurofilament light (NfL) levels from baseline. The U.S. Food and Drug Administration (FDA) recently advised Denali to evaluate NfL as an exploratory endpoint to assess its potential as a biomarker for diagnosing, predicting outcomes, or gauging treatment response in individuals with neuronopathic MPS II. DNL310 is an investigational brain-penetrant enzyme replacement therapy aimed at addressing the behavioral, cognitive, and physical symptoms of MPS II. The global Phase 2/3 COMPASS study is currently ongoing.
Hunter Syndrome Overview
Hunter Syndrome, also known as Mucopolysaccharidosis II (MPS II), is a rare genetic disorder that affects the body’s ability to break down certain complex molecules called glycosaminoglycans (GAGs).
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Emerging Hunter Syndrome Drugs Under Different Phases of Clinical Development Include:
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CAP 001: Capsida Biotherapeutics
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JR-141: JCR Pharmaceuticals
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TAK-609/SHP609/HGT-2310: Takeda
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GC1111 (Hunterase): Green Cross Corporation/GC Pharma
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DNL310: Denali Therapeutics
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RGX-121: Regenxbio
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SB-913: Sangamo Therapeutics
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AVR-RD-05: AVROBIO
Hunter Syndrome Route of Administration
Hunter Syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
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Oral
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Parenteral
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Intravenous
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Subcutaneous
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Topical
Hunter Syndrome Molecule Type
Hunter Syndrome Products have been categorized under various Molecule types, such as
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Monoclonal Antibody
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Peptides
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Polymer
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Small molecule
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Gene therapy
Hunter Syndrome Pipeline Therapeutics Assessment
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Hunter Syndrome Assessment by Product Type
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Hunter Syndrome By Stage and Product Type
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Hunter Syndrome Assessment by Route of Administration
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Hunter Syndrome By Stage and Route of Administration
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Hunter Syndrome Assessment by Molecule Type
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Hunter Syndrome by Stage and Molecule Type
DelveInsight’s Hunter Syndrome Report covers around 10+ products under different phases of clinical development like
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Late-stage products (Phase III)
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Mid-stage products (Phase II)
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Early-stage product (Phase I)
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Pre-clinical and Discovery stage candidates
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Discontinued & Inactive candidates
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Route of Administration
Further Hunter Syndrome product details are provided in the report. Download the Hunter Syndrome pipeline report to learn more about the emerging Hunter Syndrome therapies
Some of the key companies in the Hunter Syndrome Therapeutics Market include:
Key companies developing therapies for Hunter Syndrome are – REGENXBIO Inc., Takeda Pharmaceutical Company Limited, Triley Bidco Limited (Clinigen Group PLC), JCR Pharmaceuticals, Sangamo Therapeutics, Denali Therapeutics Inc., Bioasis Technologies Inc., Inventiva, GC Pharma (Green Cross Holdings), Esteve, Avrobio Inc., CANbridge Life Sciences Ltd, and others.
Hunter Syndrome Pipeline Analysis:
The Hunter Syndrome pipeline report provides insights into
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The report provides detailed insights about companies that are developing therapies for the treatment of Hunter Syndrome with aggregate therapies developed by each company for the same.
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It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Hunter Syndrome Treatment.
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Hunter Syndrome key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
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Hunter Syndrome Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Hunter Syndrome market.
The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.
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Hunter Syndrome Pipeline Market Drivers
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Increase in the number of the population suffering from hunter syndrome, improving Healthcare Infrastructure, demand for New and Effective Drugs are some of the important factors that are fueling the Hunter Syndrome Market.
Hunter Syndrome Pipeline Market Barriers
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However, high cost of treatment, heterogeneity of the disease, lack of awareness and other factors are creating obstacles in the Hunter Syndrome Market growth.
Scope of Hunter Syndrome Pipeline Drug Insight
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Coverage: Global
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Key Hunter Syndrome Companies: Capsida Biotherapeutics, JCR Pharmaceuticals, Takeda, Green Cross Corporation/GC Pharma, Denali Therapeutics, Regenxbio, Sangamo Therapeutics, AVROBIO, and others
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Key Hunter Syndrome Therapies: CAP 001, JR-141, TAK-609/SHP609/HGT-2310, GC1111 (Hunterase), DNL310, RGX-121, SB-913, AVR-RD-05, and others
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Hunter Syndrome Therapeutic Assessment: Hunter Syndrome current marketed and Hunter Syndrome emerging therapies
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Hunter Syndrome Market Dynamics: Hunter Syndrome market drivers and Hunter Syndrome market barriers
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Table of Contents
1. Hunter Syndrome Report Introduction
2. Hunter Syndrome Executive Summary
3. Hunter Syndrome Overview
4. Hunter Syndrome- Analytical Perspective In-depth Commercial Assessment
5. Hunter Syndrome Pipeline Therapeutics
6. Hunter Syndrome Late Stage Products (Phase II/III)
7. Hunter Syndrome Mid Stage Products (Phase II)
8. Hunter Syndrome Early Stage Products (Phase I)
9. Hunter Syndrome Preclinical Stage Products
10. Hunter Syndrome Therapeutics Assessment
11. Hunter Syndrome Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Hunter Syndrome Key Companies
14. Hunter Syndrome Key Products
15. Hunter Syndrome Unmet Needs
16 . Hunter Syndrome Market Drivers and Barriers
17. Hunter Syndrome Future Perspectives and Conclusion
18. Hunter Syndrome Analyst Views
19. Appendix
20. About DelveInsight
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. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach
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