Mucopolysaccharidosis Type I Pipeline Appears Robust With 8+ Key Pharma Companies Actively Working in the Therapeutics Segment
DelveInsight's, 'Mucopolysaccharidosis Type I Pipeline Insight 2025' report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Mucopolysaccharidosis Type I pipeline landscape. It covers the Mucopolysaccharidosis Type I pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Mucopolysaccharidosis Type I pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Discover the latest drugs and treatment options in the Mucopolysaccharidosis Type I Pipeline. Dive into DelveInsight's comprehensive report today! @ Mucopolysaccharidosis Type I Pipeline Outlook
Key Takeaways from the Mucopolysaccharidosis Type I Pipeline Report
In May 2025, JCR Pharmaceuticals Co. Ltd announced a JR-171-101 study (NCT04227600) and fulfill all eligibility criteria can be enrolled in this JR-171-102 study. In the JR-171-102 study, subjects will receive either 2.0 or 4.0 mg/kg/week of JR-171 intravenously at the same doses received at Week 12 of the JR-171-101 study.
DelveInsight's Mucopolysaccharidosis Type I Pipeline report depicts a robust space with 8+ active players working to develop 8+ pipeline therapies for Mucopolysaccharidosis Type I treatment.
The leading Mucopolysaccharidosis Type I Companies such as Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others.
Promising Mucopolysaccharidosis Type I Pipeline Therapies such as Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, Cyclosporine A (CsA), Azathioprine (Aza), RGX-111, SBC-103, and others.
Stay ahead with the most recent pipeline outlook for Mucopolysaccharidosis Type I. Get insights into clinical trials, emerging therapies, and leading companies with DelveInsight @ Mucopolysaccharidosis Type I Treatment Drugs
Mucopolysaccharidosis Type I Emerging Drugs Profile
OTL 203: Orchard Therapeutics
OTL-203 is an investigational ex vivo autologous gene therapy being investigated for the treatment of MPS-I. It uses a modified virus to insert a functional copy of the IDUA gene into a patient's cells. OTL-203 is being developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. OTL-203 has received rare pediatric disease designation from the FDA. Through an ongoing proof-of-concept clinical trial, OTL-203 is being evaluated as a potential treatment for patients with the most severe form of MPS-I, known as Hurler syndrome. OTL-203 is an investigational therapy and has not been approved by any regulatory agency or health authority. Currently, the drug is in Phase III stage of its development for the treatment of Mucopolysaccharidosis Type I.
JR 171: JCR Pharmaceuticals
JR-171, is an investigational drug for the treatment of mucoplysaccharidosis type I (MPS I, or Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain-barrier (BBB)-penetrating form recombinant α-L-iduronidase that was developed using JCR's proprietary J-Brain Cargo® BBB technology. JR-171 received the orphan drug designation from the US Food Drug Administration (FDA) in February 2021. The trial is also scheduled for enrolling patients in the US. Currently, the drug is in Phase I/II stage of its development for the treatment of Mucopolysaccharidosis Type I.
The Mucopolysaccharidosis Type I Pipeline Report Provides Insights into
The report provides detailed insights about companies that are developing therapies for the treatment of Mucopolysaccharidosis Type I with aggregate therapies developed by each company for the same.
It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Mucopolysaccharidosis Type I Treatment.
Mucopolysaccharidosis Type I Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
Mucopolysaccharidosis Type I 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.
Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Mucopolysaccharidosis Type I market
Explore groundbreaking therapies and clinical trials in the Mucopolysaccharidosis Type I Marketed and Pipeline Drugs. Access DelveInsight's detailed report now! @ New Mucopolysaccharidosis Type I Drugs
Mucopolysaccharidosis Type I Companies
Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others.
Mucopolysaccharidosis type I (MPS I) 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
Parenteral
Topical
Mucopolysaccharidosis Type I Products have been categorized under various Molecule types such as
Recombinant fusion proteins
Small molecule
Monoclonal antibody
Peptide
Polymer
Gene therapy
Unveil the future of Mucopolysaccharidosis Type I Treatment. Learn about new drugs, pipeline developments, and key companies with DelveInsight's expert analysis @ Mucopolysaccharidosis Type I Market Drivers and Barriers
Scope of the Mucopolysaccharidosis Type I Market Report
Coverage- Global
Mucopolysaccharidosis Type I Companies- Orchard Therapeutics, JCR Pharmaceuticals, REGENXBIO and others.
Mucopolysaccharidosis Type I Pipeline Therapies- Laronidase, Antihistamine, Antipyretic, JR-171, AGT-181, Cyclosporine A (CsA), Azathioprine (Aza), RGX-111, SBC-103, and others.
Mucopolysaccharidosis Type I Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
Mucopolysaccharidosis Type I Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
Get the latest on Mucopolysaccharidosis Type I Therapies and clinical trials. Download DelveInsight's in-depth pipeline report today! @ Mucopolysaccharidosis Type I Companies, Key Products and Unmet Needs
Table of Contents
Introduction
Executive Summary
Mucopolysaccharidosis type I (MPS I): Overview
Pipeline Therapeutics
Therapeutic Assessment
Mucopolysaccharidosis type I (MPS I)– DelveInsight's Analytical Perspective
Late Stage Products (Phase III)
OTL 203: Orchard Therapeutics
Drug profiles in the detailed report…..
Mid Stage Products (Phase II)
Drug Name: Company Name
Drug profiles in the detailed report…..
Early Stage Products (Phase I)
JR 171: JCR Pharmaceuticals
Drug profiles in the detailed report…..
Preclinical and Discovery Stage Products
Drug name : Company name
Drug profiles in the detailed report…..
Inactive Products
Mucopolysaccharidosis type I (MPS I) Key Companies
Mucopolysaccharidosis type I (MPS I) Key Products
Mucopolysaccharidosis type I (MPS I)- Unmet Needs
Mucopolysaccharidosis type I (MPS I)- Market Drivers and Barriers
Mucopolysaccharidosis type I (MPS I)- Future Perspectives and Conclusion
Mucopolysaccharidosis type I (MPS I) Analyst Views
Mucopolysaccharidosis type I (MPS I) Key Companies
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.
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CBC
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The most recent rally in silver futures for July delivery took the price 22.16% higher from $27.845 on April 7 to a high of $34.015 on April 25. Over around the same period, SLV rose 15.24% from $26.57 to $30.62 per share. The reason for SLV's underperformance is that the silver ETF can miss highs or lows when the stock market is closed, as the volatile silver futures trade around the clock. With silver over the $32 per ounce level, the critical upside target is the $37.58 technical resistance level. Silver's relative value versus gold, the fundamental deficit, and the path of least resistance of silver prices since the 2020 low all point to a break above the resistance and a challenge of the 2011 and 1980 highs. However, buying on price weakness will likely remain optimal in the volatile silver market.
