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

Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome ) - Drugs In Development, 2022, provides an overview of the Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders) pipeline landscape.

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is an inherited metabolic disease caused by an absence or malfunctioning of certain enzymes needed to breakdown molecules called glycosaminoglycans (GAG). Symptoms include seizures, hyperactivity, liver and spleen enlargement, severe diarrhea or constipation and enlargement of tonsils and adenoids. Treatment includes enzyme replacement therapy (ERT).

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Drugs In Development, 2022, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders) pipeline guide also reviews of key players involved in therapeutic development for Mucopolysaccharidosis III (MPS III) (Sanfilippo 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, Preclinical and Discovery stages are 3, 3, 1, 18 and 4 respectively. Similarly, the Universities portfolio in Preclinical stages comprises 1 molecules, respectively.

Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders).
  • The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome ) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Mucopolysaccharidosis III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome) – Overview
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Companies Involved in Therapeutics Development
Allievex Corp
Amicus Therapeutics Inc
ArmaGen Inc
BioStrategies LC
Denali Therapeutics Inc
Esteve Pharmaceuticals SA
GC Biopharma Corp
JCR Pharmaceuticals Co Ltd
Jupiter Neurosciences Inc
Lacerta Therapeutics Inc
M6P Therapeutics
Neurogt Inc
Orchard Therapeutics Plc
Phoenix Nest Inc
Seelos Therapeutics, Inc.
Swedish Orphan Biovitrum AB
Tega Therapeutics Inc
Ultragenyx Pharmaceutical Inc
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Drug Profiles
AGT-184 – Drug Profile
AGT-187 – Drug Profile
anakinra – Drug Profile
AVP-6 – Drug Profile
AX-313 – Drug Profile
BIOS: MPS IIIA – Drug Profile
DNL-126 – Drug Profile
EGT-101 – Drug Profile
EGT-201 – Drug Profile
GC-1130A – Drug Profile
Gene Therapies to Activate NAGLU for Mucopolysaccharidosis Type IIIB – Drug Profile
Gene Therapy for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Drug Profile
Gene Therapy to Activate NAGLU for Mucopolysaccharidosis III (Sanfilippo Syndrome) – Drug Profile
Gene Therapy to Activate NAGLU for Mucopolysaccharidosis Type IIIB – Drug Profile
JLK-247 – Drug Profile
JOT-109 – Drug Profile
JR-441 – Drug Profile
JR-446 – Drug Profile
LA-027 – Drug Profile
M-041 – Drug Profile
M-043 – Drug Profile
Next Generation MPS IIIA – Drug Profile
Next Generation MPS IIIB – Drug Profile
OTL-201 – Drug Profile
OTL-202 – Drug Profile
Proteins for Mucopolysaccharidosis Type IIIC – Drug Profile
rebisufligene etisparvovec – Drug Profile
Recombinant Enzyme to Replace Sulphamidase for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Drug Profile
tralesinidase alfa – Drug Profile
trehalose – Drug Profile
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Dormant Projects
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Discontinued Products
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Product Development Milestones
Featured News & Press Releases
Feb 10, 2022: JCR pharmaceuticals to present posters on JR-441 at the 18th annual WORLDSymposium 2022
Feb 10, 2022: M6P Therapeutics presents promising preclinical data in lysosomal storage disorders at the 18th Annual WORLDSymposium 2022
Jan 21, 2022: EC grants orphan drug designation to JR-441 for the treatment of mucopolysaccharidosis type III A (MPS IIIA)
Jul 28, 2021: M6P Therapeutics presented data on M041, a recombinant enzyme therapy, for the treatment of Sanfilippo B syndrome at MPS 2021
Dec 21, 2020: Seelos Therapeutics announces issuance of a patent for Trehalose (SLS-005) in Israel
Nov 09, 2020: Seelos Therapeutics to participate in the 3rd International Conference on Sanfilippo Syndrome and related diseases
Aug 25, 2020: Seelos Therapeutics receives European Orphan Drug Designation for SLS-005 (Trehalose) in Sanfilippo syndrome
Jul 21, 2020: Seelos Therapeutics receives positive EMA opinion on orphan drug designation for SLS-005 (Trehalose) in Sanfilippo syndrome
May 15, 2020: Seelos Therapeutics receives rare pediatric disease designation for SLS-005 (Trehalose) in Sanfilippo Syndrome
Apr 30, 2020: Seelos Therapeutics receives Orphan Drug Designation for SLS-005 (Trehalose) in Sanfilippo Syndrome
Apr 21, 2020: JCR to initiate development of new drug candidate JR-446 for Sanfilippo Syndrome Type B using J-Brain Cargo
Mar 11, 2020: Seelos Therapeutics announces European Medicines Agency guidance to design an open-label pivotal study for SLS-005 in Sanfilippo Syndrome
Jan 22, 2020: Seelos Therapeutics announces updates to SLS-005 (trehalose) programs for Europe and U.S.
Dec 13, 2019: Seelos Therapeutics to participate in 33rd annual National MPS Society Family Conference
Dec 04, 2019: Seelos Therapeutics receives notice of allowance for an additional US patent for Trehalose (SLS-005)
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 III (MPS III) (Sanfilippo 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 III (MPS III) (Sanfilippo Syndrome) – Pipeline by Allievex Corp, 2022
Table 12: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Amicus Therapeutics Inc, 2022
Table 13: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by ArmaGen Inc, 2022
Table 14: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by BioStrategies LC, 2022
Table 15: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Denali Therapeutics Inc, 2022
Table 16: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Esteve Pharmaceuticals SA, 2022
Table 17: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by GC Biopharma Corp, 2022
Table 18: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by JCR Pharmaceuticals Co Ltd, 2022
Table 19: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Jupiter Neurosciences Inc, 2022
Table 20: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Lacerta Therapeutics Inc, 2022
Table 21: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by M6P Therapeutics, 2022
Table 22: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Neurogt Inc, 2022
Table 23: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Orchard Therapeutics Plc, 2022
Table 24: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Phoenix Nest Inc, 2022
Table 25: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Seelos Therapeutics, Inc., 2022
Table 26: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Swedish Orphan Biovitrum AB, 2022
Table 27: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Tega Therapeutics Inc, 2022
Table 28: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Pipeline by Ultragenyx Pharmaceutical Inc, 2022
Table 29: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Dormant Projects, 2022
Table 30: Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) – Discontinued Products, 2022
List of Figures
Figure 1: Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome), 2022
Figure 2: Number of Products under Development by Companies, 2022
Figure 3: Number of Products by Targets, 2022
Figure 4: Number of Products by Stage and Targets, 2022
Figure 5: Number of Products by Mechanism of Actions, 2022
Figure 6: Number of Products by Stage and Mechanism of Actions, 2022
Figure 7: Number of Products by Top 10 Routes of Administration, 2022
Figure 8: Number of Products by Stage and Top 10 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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