Mucopolysaccharidosis Type III Pipeline Insights 2024: Therapies, Clinical Trials, and Market Trends by DelveInsight | Takeda Pharma, BioMarin Pharma, Ultragenyx Pharma, Sarepta Therapeutics, Abeona

“Mucopolysaccharidosis Type III Pipeline Insight, 2024“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Mucopolysaccharidosis Type III Market.

The Mucopolysaccharidosis Type III 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 III Pipeline Report:

• Companies across the globe are diligently working toward developing novel Mucopolysaccharidosis Type III treatment therapies with a considerable amount of success over the years. 

• Mucopolysaccharidosis Type III companies working in the treatment market are Genevector Biotechnology, EdiGene Inc., therapeutics EdiGene Inc., Sigilon Therapeutics, REGENXBIO Inc., JCR Pharmaceuticals, Paradigm Biopharma, Jupiter Neurosciences, and others, are developing therapies for the Mucopolysaccharidosis Type III treatment 

• Emerging Mucopolysaccharidosis Type III therapies in the different phases of clinical trials are JWK-008 Chengdu, Research programme: 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 III market in the coming years.   

• In November 2023, JCR Pharmaceuticals has administered the first dose to a patient in the Phase I/II clinical trial of JR‑441 for treating Mucopolysaccharidosis Type IIIIIA (MPS IIIA), also known as Sanfilippo Syndrome Type A. The trial is being conducted at the International Center for Lysosomal Disorders (ICLD) at the University Hospital of Hamburg Eppendorf (UKE) in Germany.

• In Jan 2023, The FDA has approved the investigational new drug (IND) application for OTL-203, a hematopoietic stem cell (HSC) gene therapy being developed for the treatment of the Hurler subtype of Mucopolysaccharidosis Type III (MPS-IH), according to Orchard Therapeutics (Nasdaq: ORTX), a global leader in gene therapy. In the second half of 2023, the business anticipates starting a global registrational study to evaluate OTL-203’s effectiveness and safety to current standard of care

Mucopolysaccharidosis Type III Overview

Mucopolysaccharidosis Type IIIII (MPS III), also known as Sanfilippo syndrome, is a rare genetic disorder that affects the body’s ability to break down specific complex carbohydrates called glycosaminoglycans (GAGs). This condition leads to the accumulation of GAGs in the cells, particularly in the brain and central nervous system, causing progressive neurological damage.

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

• JWK-008 Chengdu: Genevector Biotechnology

• Research programme: oligonucleotide based RNA base editing therapeutics EdiGene Inc.

• JR 171: JCR Pharmaceuticals

• RGX 111: REGENXBIO

• SIG 005: Sigilon Therapeutics

• RGX-111: REGENXBIO Inc.

• JR-171: JCR Pharmaceuticals

• Pentosan Polysulphate: Paradigm Biopharma

• JNS102: Jupiter Neurosciences

Route of Administration

Mucopolysaccharidosis Type III 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 III Pipeline Therapeutics Assessment

• Mucopolysaccharidosis Type III Assessment by Product Type

• Mucopolysaccharidosis Type III By Stage and Product Type

• Mucopolysaccharidosis Type III Assessment by Route of Administration

• Mucopolysaccharidosis Type III By Stage and Route of Administration

• Mucopolysaccharidosis Type III Assessment by Molecule Type

• Mucopolysaccharidosis Type III by Stage and Molecule Type

DelveInsight’s Mucopolysaccharidosis Type III 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

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

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

Key companies developing therapies for Mucopolysaccharidosis Type III are – Takeda Pharmaceutical Company Limited, BioMarin Pharmaceuticals, Inc., Ultragenyx Pharmaceutical Inc., Sarepta Therapeutics, Abeona Therapeutics Inc., Eloxx Pharmaceuticals, Esteve, Immusoft Corporation, Inventiva, GC Pharma, JCR Pharmaceuticals Co Ltd., RegenxBio Inc., Sangamo Therapeutics, Inc., Bioasis Technologies Inc., Paradigm Biopharmaceuticals Ltd., and others.

Mucopolysaccharidosis Type III Pipeline Analysis:

The Mucopolysaccharidosis Type III pipeline report provides insights into 

• The report provides detailed insights about companies that are developing therapies for the treatment of Mucopolysaccharidosis Type III 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 III Treatment.

• Mucopolysaccharidosis Type III key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

• Mucopolysaccharidosis Type III 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 III 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.

Download Sample PDF Report to know more about Mucopolysaccharidosis Type III drugs and therapies

Mucopolysaccharidosis Type III Pipeline Market Drivers

• Increase in prevalence of Mucopolysaccharidosis Type III, further understanding in pathogenesis are some of the important factors that are fueling the Mucopolysaccharidosis Type III Market.

Mucopolysaccharidosis Type III Pipeline Market Barriers

• However, lack of awareness of signs and symptoms of the disorder, challenges in predicting disease severity in newborn screening and other factors are creating obstacles in the Mucopolysaccharidosis Type III Market growth.

Scope of Mucopolysaccharidosis Type III Pipeline Drug Insight    

• Coverage: Global

• Key Mucopolysaccharidosis Type III Companies: Genevector Biotechnology, EdiGene Inc., therapeutics EdiGene Inc., Sigilon Therapeutics, REGENXBIO Inc., JCR Pharmaceuticals, Paradigm Biopharma, Jupiter Neurosciences, and others

• Key Mucopolysaccharidosis Type III Therapies: JWK-008 Chengdu, Research programme: oligonucleotide based RNA base editing therapeutics, SIG 005, RGX-111, JR-171, Pentosan Polysulphate, JNS102, and others

• Mucopolysaccharidosis Type III Therapeutic Assessment: Mucopolysaccharidosis Type III current marketed and Mucopolysaccharidosis Type III emerging therapies

• Mucopolysaccharidosis Type III Market Dynamics: Mucopolysaccharidosis Type III market drivers and Mucopolysaccharidosis Type III 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.

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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