DelveInsight’s Hunter Syndrome Pipeline Insights report provides a comprehensive outlook of the pipeline therapies that are in different clinical and preclinical stages of development, their launch, and how the market is expected to transform in the aftermath across the Hunter Syndrome domain.
The Hunter Syndrome Pipeline Analysis report offers a 360° view of the therapeutics landscape by development stage, product type, route of administration, molecule type, and MOA for Hunter Syndrome emerging therapies. The pipeline report lays down the business opportunities, threats, prospective collaborations, strong competitors, and growth strategies.
Some of the key highlights from the Hunter Syndrome Pipeline report:
- Globally, some of the key companies in the Hunter Syndrome Therapeutics Landscape include JCR Pharmaceuticals, Denali Therapeutics, AVRO Bio, Regenxbio, and several others.
- Hunter Syndrome pipeline is robust and possesses multiple potential drugs in late and mid-stage developments to be launched in the near future. Some of the key Hunter Syndrome pipeline therapies include AVR-RD-05, RGX-121, DNL310, JR-141, and many more.
- Among the different therapies in the Hunter Syndrome pipeline, the JCR Pharmaceuticals’s JR-141 is in the most advanced stage of development. The company has received Orphan Drug Designation from both the FDA and the EMA. Furthermore, In February 2021, FDA granted Fast Track Designation to the drug and also granted IND clearance to initiate our global Phase III trial of JR-141 in the US.
- Denali Therapeutics is developing DNL310, a recombinant IDS enzyme engineered to cross the blood-brain barrier and to replace the IDS enzyme, and treat neuropathic and systemic forms of the disease. In March 2021, Denali Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to (DNL310) for the treatment of patients with Hunter syndrome (MPS II).
- Regenxbio is working on RGX-121, an investigational gene therapy product candidate for the treatment of Mucopolysaccharidosis Type II. In August 2016, Regenxbio announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to RGX-121.
- Gene therapy is also expected to provide a new dimension to the Hunter Syndrome treatment. The gene therapies that can target and subsequently edit specific stretches of a genetic code can also prove to be a big competition in near future for ERT-based therapies.
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Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is a rare genetic disorder that results in large sugar molecules called glycosaminoglycans (or GAGs or mucopolysaccharides) build up in body tissues. Elaprase is the first and only treatment approved for people suffering from Hunter syndrome. Currently, several companies are diligently involved in developing the Hunter Syndrome therapies.
- AVR-RD-05
- RGX-121
- DNL310
- JR-141
Key Companies in Hunter Syndrome Therapeutics Landscape
- AVRO Bio
- Regenxbio
- JCR Pharmaceuticals
- Denali Therapeutics
Hunter Syndrome Pipeline Therapeutics Assessment
By Product Type
- Mono
- Combination
- Mono/Combination
By Stage
- Discovery
- Pre-clinical
- Phase I
- Phase II
- Phase III
- Pre-registration
By Route of Administration
- Subcutaneous
- Intravenous
- Intramuscular
By Molecule Type
- Recombinant fusion proteins
- Gene therapies
- Enzymes
- Product Type
By Mechanism of Action
- Iduronate Sulfatase Replacements
- Gene transference
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Scope of the Report
- Coverage: Global
- Key Market Players: AVRO Bio, Regenxbio, JCR Pharmaceuticals, Denali Therapeutics, and others.
- Key Pipeline Therapies: AVR-RD-05, RGX-121, DNL310, JR-141, and several others.
Table of Content
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1 |
Report Introduction |
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2 |
Hunter Syndrome |
|
3 |
Hunter Syndrome Current Treatment Patterns |
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4 |
Hunter Syndrome – DelveInsight’s Analytical Perspective |
|
5 |
Therapeutic Assessment |
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6 |
Hunter Syndrome Late Stage Products (Phase-III) |
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7 |
Hunter Syndrome Mid Stage Products (Phase-II) |
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8 |
Early Stage Hunter Syndrome Products (Phase-I) |
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9 |
Hunter Syndrome Pre-clinical Products and Discovery Stage Products |
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10 |
Inactive Products |
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11 |
Dormant Products |
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12 |
Hunter Syndrome Discontinued Products |
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13 |
Hunter Syndrome Product Profiles |
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14 |
Hunter Syndrome Key Companies |
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15 |
Hunter Syndrome Key Products |
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16 |
Dormant and Discontinued Products |
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17 |
Hunter Syndrome Unmet Needs |
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18 |
Hunter Syndrome Future Perspectives |
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19 |
Hunter Syndrome Analyst Review |
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20 |
Appendix |
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21 |
Report Methodology |
Know more of what’s covered in the Hunter Syndrome Pipeline Assessment report
Key Questions Answered in the Hunter Syndrome Report
- What are the current options for Hunter Syndrome treatment?
- How many companies are developing therapies for the treatment of Hunter Syndrome?
- What are the principal therapies developed by these companies in the industry?
- How many therapies are developed by each company for the treatment of Hunter Syndrome?
- How many Hunter Syndrome emerging therapies are in early-stage, mid-stage, and late stage of development for the treatment of Hunter Syndrome?
- Out of total pipeline products, how many therapies are given as a monotherapy and in combination with other therapies?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and major licensing activities that will impact the Hunter Syndrome market?
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Contact Information:
Shruti Thakur info@delveinsight.com +1(919)321-6187