Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Drugs In Development, 2022, provides an overview of the Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders) pipeline landscape.

MPS I (Mucopolysaccharidosis I) is an inherited lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase, a lysosomal enzyme normally required for the breakdown of certain complex carbohydrates known as glycosaminoglycans (GAGs). Symptoms include abnormal bones in the spine, claw hand, cloudy corneas, deafness and heart valve problems. Treatment includes bone marrow transplantation, enzyme therapy and gene therapy.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase III, Phase II, Phase I, IND/CTA Filed, Preclinical, Discovery and Unknown stages are 1, 5, 2, 2, 6, 3 and 1 respectively. Similarly, the Universities portfolio in Discovery stages comprises 2 molecules, respectively.

Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note:Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews latest news related to pipeline therapeutics for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders)
Reasons to Buy
  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) (Genetic Disorders) pipeline depth and focus of Indication therapeutics.
  • Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.


Introduction
Global Markets Direct Report Coverage
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Overview
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Companies Involved in Therapeutics Development
ArmaGen Inc
BioStrategies LC
Chengdu Genevector Biotechnology Co Ltd
Cinnagen Co
Denali Therapeutics Inc
EdiGene Inc
Eloxx Pharmaceuticals Inc
Gain Therapeutics Inc
Immusoft Corp
JCR Pharmaceuticals Co Ltd
Jupiter Neurosciences Inc
Mina Therapeutics Ltd
Orchard Therapeutics Plc
Ossianix Inc
Paradigm Biopharmaceuticals Ltd
RegenxBio Inc
ReqMed Co Ltd
Sigilon Therapeutics Inc
Tega Therapeutics Inc
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Drug Profiles
AGT-181 – Drug Profile
Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I – Drug Profile
DNL-622 – Drug Profile
Drugs for Hurler syndrome – Drug Profile
ELX-02 – Drug Profile
ISP-001 – Drug Profile
JNS-102 – Drug Profile
JWK-008 – Drug Profile
laronidase – Drug Profile
lepunafusp alfa – Drug Profile
MTL-CEBPA – Drug Profile
Oligonucleotide to Activate IDUA for Hurler Syndrome, Unspecified Cancer, Unspecified Muscular Diseases, Unspecified Neurologic Disorders, and Unspecified Non-Genetic Diseases – Drug Profile
OTL-203 – Drug Profile
pentosan polysulfate sodium – Drug Profile
Recombinant Alpha L-Iduronidase Replacement for Mucopolysaccharidosis I (MPS I) – Drug Profile
Recombinant Alpha-L-Iduronidase Replacement for Mucopolysaccharidosis I – Drug Profile
RGX-111 – Drug Profile
SIG-005 – Drug Profile
SIG-XXX: MPS-1 + CNS – Drug Profile
TXB-4LS1 – Drug Profile
X-372 – Drug Profile
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Dormant Projects
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Discontinued Products
Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Product Development Milestones
Featured News & Press Releases
Sep 26, 2022: Paradigm Reports Successful Safety Review in MPS VI Phase 2 Clinical Trial and update on timing of PARA_008 top-line data readout.
Sep 01, 2022: Immusoft announces FDA clearance of IND application for ISP-001 for MPS I, the first engineered B cell therapy to enter into clinical trials
Aug 08, 2022: MPS clinical program update and paradigm to present at the International Conference on lysosomal diseases
Feb 11, 2022: International researchers highlight the need for PPS as a new adjunct therapy treatment for unmet needs of patients with MPS VI
Feb 10, 2022: JCR pharmaceuticals to present posters on JR-171 at the 18th annual WORLDSymposium 2022
Feb 07, 2022: Immusoft to present at WORLDSymposium 2022 Conference
Jan 31, 2022: Sigilon Therapeutics to present preclinical data at the 18th Annual WORLDSymposium on Lysosomal Diseases, MPS-1 and MPS-6
Nov 23, 2021: Positive interim data from phase 2 rare disease trial presented at international medical congress
Oct 05, 2021: JCR Pharmaceuticals: US FDA grants Fast Track Designation for JR-171 for the treatment of Mucopolysaccaridosis Type I (MPSI)
Sep 09, 2021: Sigilon Therapeutics announces acceptance of clinical trial application in the UK for SIG-005 for the treatment of mucopolysaccharidosis type I
Jul 23, 2021: Sigilon Therapeutics presents preclinical data at the 16th International Symposium on MPS and Related Diseases
Mar 30, 2021: EMA grants Orphan Drug Designation to JR-171 for the treatment of Mucopolysaccaridosis Type I (MPS I)
Feb 12, 2021: US FDA grants orphan drug designation to JR-171 for the treatment of Mucopolysaccaridosis Type I (MPS I)
Dec 17, 2020: Sigilon Therapeutics receives Orphan Drug Designation for SIG-005 for the treatment of mucopolysaccharidosis type I
Nov 12, 2020: First patient dosed in Phase 2 clinical trial evaluating PPS in Mucopulysaccharidoses Type-1 (mps-i)
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Table 1: Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), 2022
Table 2: Number of Products under Development by Companies, 2022
Table 3: Number of Products under Development by Universities/Institutes, 2022
Table 4: Products under Development by Companies, 2022
Table 5: Products under Development by Companies, 2022 (Contd..1)
Table 6: Products under Development by Universities/Institutes, 2022
Table 7: Number of Products by Stage and Target, 2022
Table 8: Number of Products by Stage and Mechanism of Action, 2022
Table 9: Number of Products by Stage and Route of Administration, 2022
Table 10: Number of Products by Stage and Molecule Type, 2022
Table 11: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by ArmaGen Inc, 2022
Table 12: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by BioStrategies LC, 2022
Table 13: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Chengdu Genevector Biotechnology Co Ltd, 2022
Table 14: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Cinnagen Co, 2022
Table 15: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Denali Therapeutics Inc, 2022
Table 16: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by EdiGene Inc, 2022
Table 17: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Eloxx Pharmaceuticals Inc, 2022
Table 18: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Gain Therapeutics Inc, 2022
Table 19: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Immusoft Corp, 2022
Table 20: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by JCR Pharmaceuticals Co Ltd, 2022
Table 21: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Jupiter Neurosciences Inc, 2022
Table 22: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Mina Therapeutics Ltd, 2022
Table 23: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Orchard Therapeutics Plc, 2022
Table 24: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Ossianix Inc, 2022
Table 25: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Paradigm Biopharmaceuticals Ltd, 2022
Table 26: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by RegenxBio Inc, 2022
Table 27: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by ReqMed Co Ltd, 2022
Table 28: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Sigilon Therapeutics Inc, 2022
Table 29: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Pipeline by Tega Therapeutics Inc, 2022
Table 30: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Dormant Projects, 2022
Table 31: Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ) – Discontinued Products, 2022
List of Figures
Figure 1: Number of Products under Development for Mucopolysaccharidosis I (MPS I) (Hurler Syndrome ), 2022
Figure 2: Number of Products under Development by Companies, 2022
Figure 3: Number of Products by Top 10 Targets, 2022
Figure 4: Number of Products by Stage and Top 10 Targets, 2022
Figure 5: Number of Products by Top 10 Mechanism of Actions, 2022
Figure 6: Number of Products by Stage and Top 10 Mechanism of Actions, 2022
Figure 7: Number of Products by Routes of Administration, 2022
Figure 8: Number of Products by Stage and Routes of Administration, 2022
Figure 9: Number of Products by Molecule Types, 2022
Figure 10: Number of Products by Stage and Molecule Types, 2022

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