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.

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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.

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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

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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

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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

1. Introduction
2. Executive Summary
3. Mucopolysaccharidosis type I (MPS I): Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Mucopolysaccharidosis type I (MPS I)– DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. OTL 203: Orchard Therapeutics
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II)
11. Drug Name: Company Name
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I)
14. JR 171: JCR Pharmaceuticals
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. Drug name : Company name
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Mucopolysaccharidosis type I (MPS I) Key Companies
21. Mucopolysaccharidosis type I (MPS I) Key Products
22. Mucopolysaccharidosis type I (MPS I)- Unmet Needs
23. Mucopolysaccharidosis type I (MPS I)- Market Drivers and Barriers
24. Mucopolysaccharidosis type I (MPS I)- Future Perspectives and Conclusion
25. Mucopolysaccharidosis type I (MPS I) Analyst Views
26. Mucopolysaccharidosis type I (MPS I) Key Companies
27. Appendix

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