DelveInsight’s, “Mucopolysaccharidosis Type I Pipeline Insight 2023” report provides comprehensive insights about 12+ companies and 12+ pipeline drugs in the 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.
Key Takeaways from the Mucopolysaccharidosis Type I Pipeline Report
- DelveInsight’s Mucopolysaccharidosis Type I pipeline report depicts a robust space with 12+ active players working to develop 12+ pipeline therapies for Mucopolysaccharidosis Type I treatment.
- The leading companies working in the Mucopolysaccharidosis Type I Market include JCR Pharmaceuticals, REGENXBIO, Talaris Therapeutics Inc., Orchard Therapeutics, Paradigm Biopharma, Jupiter Neurosciences, and others.
- Promising Mucopolysaccharidosis Type I Pipeline Therapies in the various stages of development include JR-171 (lepunafusp alfa), Adalimumab, AGT-181, RGX-111, SB-318, Cyclosporine A (CsA), Azathioprine (Aza), and others.
- November 2023: Masonic Cancer Center, University of Minnesota announced a study of Phase 2 clinical trials for IMD Preparative Regimen. This single-institution, phase II study is designed to test the ability to achieve donor hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM) using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe osteopetrosis (OP).
- October 2023: JCR Pharmaceuticals Co. Ltd announced a study of Phase 1 & 2 clinical trials for JR-441. A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients. Phase I/II Study of Weekly Infusions of JR-441 in Patients With Mucopolysaccharidosis Type IIIA.
- October 2023: Paul Szabolcs announced a study of Phase 2 clinical trials for Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan and Thiotepa. The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.
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In the Mucopolysaccharidosis Type I Pipeline Report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Mucopolysaccharidosis type I (MPS I) collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Mucopolysaccharidosis Type I Overview
Mucopolysaccharidosis type I (MPS I) is a progressive multisystem disorder with features ranging over a continuum of severity. While affected individuals have traditionally been classified as having one of three MPS I syndromes (Hurler syndrome, Hurler-Scheie syndrome, or Scheie syndrome), no easily measurable biochemical differences have been identified and the clinical findings overlap.
Find out more about Mucopolysaccharidosis Type I Therapeutics Assessment @ Mucopolysaccharidosis Type I Preclinical and Discovery Stage Products
Mucopolysaccharidosis Type I Emerging Drugs Profile
- JR 171: JCR Pharmaceuticals
- RGX 111: REGENXBIO
Mucopolysaccharidosis Type I Pipeline Therapeutics Assessment
There are approx. 12+ key companies which are developing the Mucopolysaccharidosis type I therapies. The Mucopolysaccharidosis type I companies which have their Mucopolysaccharidosis type I (MPS I) drug candidates in the most advanced stage, i.e. Phase I/II, JCR Pharmaceuticals.
DelveInsight’s Mucopolysaccharidosis Type I Pipeline Report covers around 12+ products under different phases of clinical development like
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
Mucopolysaccharidosis type 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
- Intra-articular
- Intraocular
- Intrathecal
- Intravenous
- Ophthalmic
- Oral
- Parenteral
- Subcutaneous
- Topical
- Transdermal
Mucopolysaccharidosis Type I Pipeline Products have been categorized under various Molecule types such as
- Oligonucleotide
- Peptide
- Small molecule
Learn more about the emerging Mucopolysaccharidosis Type I Pipeline Therapies @ Mucopolysaccharidosis Type I Clinical Trials Assessment
Scope of the Mucopolysaccharidosis Type I Pipeline Report
- Coverage- Global
- 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
- Mucopolysaccharidosis Type I Companies- JCR Pharmaceuticals, REGENXBIO, Talaris Therapeutics Inc., Orchard Therapeutics, Paradigm Biopharma, Jupiter Neurosciences, and others.
- Mucopolysaccharidosis Type I Pipeline Therapies- JR-171 (lepunafusp alfa), Adalimumab, AGT-181, RGX-111, SB-318, Cyclosporine A (CsA), Azathioprine (Aza), and others.
Dive deep into rich insights for new drugs for Mucopolysaccharidosis Type I Treatment, Visit @ Mucopolysaccharidosis Type I Market Drivers and Barriers, and Future Perspective
Table of Content
- 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)
- Drug name : Company name
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase I/II)
- JR 171: JCR Pharmaceuticals
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Drug name : Company name
- 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
For further information on the Mucopolysaccharidosis Type I Pipeline therapeutics, reach out to Mucopolysaccharidosis Type I Unmet Needs and Analyst Views
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