Mucopolysaccharidosis Type I Pipeline Drugs and Companies Insight Report, 2023 Updates: Analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments

Mucopolysaccharidosis Type I Overview

Mucopolysaccharidosis type I (MPS I) is a rare genetic disorder that affects many parts of the body (multisystem). MPS I is member of a group of hereditary metabolic diseases known as the Mucopolysaccharidosis which, in turn, are part of a larger group of diseases known as lysosomal storage disorders (LSDs). Lysosomes function as the primary digestive and recycling units within cells.

“Mucopolysaccharidosis Type I Pipeline Insight, 2023“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Mucopolysaccharidosis Type I Market.

The Mucopolysaccharidosis Type I 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.

Some of the key takeaways from the Mucopolysaccharidosis Type I Pipeline Report:

• Companies across the globe are diligently working toward developing novel Mucopolysaccharidosis Type I treatment therapies with a considerable amount of success over the years. Mucopolysaccharidosis Type I Key players such as – Chengdu Genevector Biotechnology, EdiGene Inc., Sigilon Therapeutics, REGENXBIO Inc., JCR Pharmaceuticals, Paradigm Biopharma, Jupiter Neurosciences, and others, are developing therapies for the Mucopolysaccharidosis Type I treatment 
• Mucopolysaccharidosis Type I Emerging therapies such as – JWK-008, oligonucleotide based RNA base editing therapeutics, SIG 005, RGX-111, JR-171, Pentosan Polysulphate, JNS102, and others are expected to have a significant impact on the Mucopolysaccharidosis Type I market in the coming years.   
• In February 2022, REGENXBIO Inc. announced positive interim data are being presented at the 18th Annual WORLD Symposium™ from five patients in the ongoing Phase I/II trial and one patient from a single-patient Investigational New Drug (IND) application of RGX-111 for the treatment of severe Mucopolysaccharidosis Type I (MPS I)
• As of December 20, 2021, RGX-111 is reported to be well-tolerated in the five patients enrolled in the Phase I/II clinical trial with no drug-related serious adverse events (SAEs). Time of post-administration follow-up ranges from three weeks to 56 weeks

Route of Administration

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

Molecule Type

Products have been categorized under various Molecule types, such as

• Oligonucleotide
• Peptide
• Small molecule

Mucopolysaccharidosis Type I Pipeline Therapeutics Assessment

• Mucopolysaccharidosis Type I Assessment by Product Type
• Mucopolysaccharidosis Type I By Stage and Product Type
• Mucopolysaccharidosis Type I Assessment by Route of Administration
• Mucopolysaccharidosis Type I By Stage and Route of Administration
• Mucopolysaccharidosis Type I Assessment by Molecule Type
• Mucopolysaccharidosis Type I by Stage and Molecule Type

DelveInsight’s Mucopolysaccharidosis Type I 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)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Some of the key companies in the Mucopolysaccharidosis Type I Therapeutics Market include:

Key companies developing therapies for Mucopolysaccharidosis Type I are – Shire (Takeda Pharmaceutical Company Limited), Denali Therapeutics, ArmaGen, REGENXBIO Inc, Sangamo Therapeutics, BioMarin, Lysogene., beona Therapeutics Inc., and others.

Emerging Mucopolysaccharidosis Type I Drugs Under Different Phases of Clinical Development Include:

• JWK-008: Chengdu Genevector Biotechnology
• Research programme: oligonucleotide based RNA base editing therapeutics: EdiGene Inc.
• SIG 005: Sigilon Therapeutics
• RGX-111: REGENXBIO Inc.
• JR-171: JCR Pharmaceuticals
• Pentosan Polysulphate: Paradigm Biopharma
• JNS102: Jupiter Neurosciences

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Mucopolysaccharidosis Type I Pipeline Analysis:

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 key 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.

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.

Further Mucopolysaccharidosis Type I product details are provided in the report. Download the Mucopolysaccharidosis Type I pipeline report to learn more about the emerging Mucopolysaccharidosis Type I therapies

Mucopolysaccharidosis Type I Pipeline Market Drivers

• Increase in prevalence of Mucopolysaccharidosis type I
• Further understanding in pathogenesis

Mucopolysaccharidosis Type I Pipeline Market Barriers

• Lack of awareness of signs and symptoms of the disorder
• Challenges in predicting disease severity in newborn screening

Scope of Mucopolysaccharidosis Type I Pipeline Drug Insight    

• Coverage: Global
• Key Mucopolysaccharidosis Type I Companies: Chengdu Genevector Biotechnology, EdiGene Inc., Sigilon Therapeutics, REGENXBIO Inc., JCR Pharmaceuticals, Paradigm Biopharma, Jupiter Neurosciences, and others
• Key Mucopolysaccharidosis Type I Therapies: JWK-008, oligonucleotide based RNA base editing therapeutics, SIG 005, RGX-111, JR-171, Pentosan Polysulphate, JNS102, and others
• Mucopolysaccharidosis Type I Therapeutic Assessment: Mucopolysaccharidosis Type I current marketed and Mucopolysaccharidosis Type I emerging therapies
• Mucopolysaccharidosis Type I Market Dynamics: Mucopolysaccharidosis Type I market drivers and Mucopolysaccharidosis Type I market barriers 

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Table of Contents

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

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