Mucopolysaccharidosis Type III Pipeline 2024: Therapies Under Investigation, Clinical Trials Milestones, and FDA Approvals by DelveInsight | Takeda Pharma, BioMarin Pharma, Ultragenyx Pharma, Sarepta

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Mucopolysaccharidosis Type III pipeline constitutes 12+ key companies continuously working towards developing 12+ Mucopolysaccharidosis Type III treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.
"Mucopolysaccharidosis Type III Pipeline Insight, 2024 [https://www.delveinsight.com/sample-request/mucopolysaccharidosis-type-i-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]" 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 December 2024, MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) have announced the commencement of a Phase I/II clinical trial for JR-446 in Japan, following the administration of the first dose to a participant. JR-446 is a proprietary -N-acetylglucosaminidase designed to penetrate the blood-brain barrier (BBB) and is being developed as a potential treatment for mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B or MPS IIIB).

*
In November 2024, GC Biopharma and Novel Pharma have announced that the first patient in the U.S. has been dosed in a multinational clinical trial of 'GC1130A,' a novel treatment for Sanfilippo syndrome type A (MPS IIIA). The companies have received IND clearance to conduct a Phase I clinical trial for 'GC1130A' in the U.S., Korea, and Japan and are currently running the multinational study. This Phase I trial aims to assess the safety, tolerability, and efficacy of 'GC1130A' over a two-year period in children aged two to six diagnosed with MPS IIIA.

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.

Get a Free Sample PDF Report to know more about Mucopolysaccharidosis Type III Pipeline Therapeutic Assessment-

https://www.delveinsight.com/report-store/mucopolysaccharidosis-type-i-pipeline-insight [https://www.delveinsight.com/report-store/mucopolysaccharidosis-type-i-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

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 [https://www.delveinsight.com/sample-request/mucopolysaccharidosis-type-i-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

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 [https://www.delveinsight.com/sample-request/mucopolysaccharidosis-type-i-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

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

Request for Sample PDF Report for Mucopolysaccharidosis Type III Pipeline Assessment and clinical trials [https://www.delveinsight.com/sample-request/mucopolysaccharidosis-type-i-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Table of Contents

1

Mucopolysaccharidosis Type III Report Introduction

2

Mucopolysaccharidosis Type III Executive Summary

3

Mucopolysaccharidosis Type III Overview

4

Mucopolysaccharidosis Type III- Analytical Perspective In-depth Commercial Assessment

5

Mucopolysaccharidosis Type III Pipeline Therapeutics

6

Mucopolysaccharidosis Type III Late Stage Products (Phase II/III)

7

Mucopolysaccharidosis Type III Mid Stage Products (Phase II)

8

Mucopolysaccharidosis Type III Early Stage Products (Phase I)

9

Mucopolysaccharidosis Type III Preclinical Stage Products

10

Mucopolysaccharidosis Type III Therapeutics Assessment

11

Mucopolysaccharidosis Type III Inactive Products

12

Company-University Collaborations (Licensing/Partnering) Analysis

13

Mucopolysaccharidosis Type III Key Companies

14

Mucopolysaccharidosis Type III Key Products

15

Mucopolysaccharidosis Type III Unmet Needs

16

Mucopolysaccharidosis Type III Market Drivers and Barriers

17

Mucopolysaccharidosis Type III Future Perspectives and Conclusion

18

Mucopolysaccharidosis Type III Analyst Views

19

Appendix

20

About DelveInsight

*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.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Gaurav Bora
Email:Send Email [https://www.abnewswire.com/email_contact_us.php?pr=mucopolysaccharidosis-type-iii-pipeline-2024-therapies-under-investigation-clinical-trials-milestones-and-fda-approvals-by-delveinsight-takeda-pharma-biomarin-pharma-ultragenyx-pharma-sarepta]
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/



This release was published on openPR.