1h Free Analyst Time
The publisher'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.Speak directly to the analyst to clarify any post sales queries you may have.
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
The publisher'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 the publisher'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.
Table of Contents
- Introduction
- 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
- Product Description
- Mechanism Of Action
- AGT-187 - Drug Profile
- Product Description
- Mechanism Of Action
- anakinra - Drug Profile
- Product Description
- Mechanism Of Action
- AVP-6 - Drug Profile
- Product Description
- Mechanism Of Action
- AX-313 - Drug Profile
- Product Description
- Mechanism Of Action
- BIOS: MPS IIIA - Drug Profile
- Product Description
- Mechanism Of Action
- DNL-126 - Drug Profile
- Product Description
- Mechanism Of Action
- EGT-101 - Drug Profile
- Product Description
- Mechanism Of Action
- EGT-201 - Drug Profile
- Product Description
- Mechanism Of Action
- GC-1130A - Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapies to Activate NAGLU for Mucopolysaccharidosis Type IIIB - Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapy for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapy to Activate NAGLU for Mucopolysaccharidosis III (Sanfilippo Syndrome) - Drug Profile
- Product Description
- Mechanism Of Action
- Gene Therapy to Activate NAGLU for Mucopolysaccharidosis Type IIIB - Drug Profile
- Product Description
- Mechanism Of Action
- JLK-247 - Drug Profile
- Product Description
- Mechanism Of Action
- JOT-109 - Drug Profile
- Product Description
- Mechanism Of Action
- JR-441 - Drug Profile
- Product Description
- Mechanism Of Action
- JR-446 - Drug Profile
- Product Description
- Mechanism Of Action
- LA-027 - Drug Profile
- Product Description
- Mechanism Of Action
- M-041 - Drug Profile
- Product Description
- Mechanism Of Action
- M-043 - Drug Profile
- Product Description
- Mechanism Of Action
- Next Generation MPS IIIA - Drug Profile
- Product Description
- Mechanism Of Action
- Next Generation MPS IIIB - Drug Profile
- Product Description
- Mechanism Of Action
- OTL-201 - Drug Profile
- Product Description
- Mechanism Of Action
- OTL-202 - Drug Profile
- Product Description
- Mechanism Of Action
- Proteins for Mucopolysaccharidosis Type IIIC - Drug Profile
- Product Description
- Mechanism Of Action
- rebisufligene etisparvovec - Drug Profile
- Product Description
- Mechanism Of Action
- Recombinant Enzyme to Replace Sulphamidase for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome) - Drug Profile
- Product Description
- Mechanism Of Action
- tralesinidase alfa - Drug Profile
- Product Description
- Mechanism Of Action
- trehalose - Drug Profile
- Product Description
- Mechanism Of Action
- 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
- Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), 2022
- Number of Products under Development by Companies, 2022
- Number of Products under Development by Universities/Institutes, 2022
- Products under Development by Companies, 2022
- Products under Development by Universities/Institutes, 2022
- Number of Products by Stage and Target, 2022
- Number of Products by Stage and Mechanism of Action, 2022
- Number of Products by Stage and Route of Administration, 2022
- Number of Products by Stage and Molecule Type, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Allievex Corp, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Amicus Therapeutics Inc, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by ArmaGen Inc, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by BioStrategies LC, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Denali Therapeutics Inc, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Esteve Pharmaceuticals SA, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by GC Biopharma Corp, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by JCR Pharmaceuticals Co Ltd, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Jupiter Neurosciences Inc, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lacerta Therapeutics Inc, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by M6P Therapeutics, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Neurogt Inc, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Orchard Therapeutics Plc, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Phoenix Nest Inc, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Seelos Therapeutics, Inc., 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Swedish Orphan Biovitrum AB, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Tega Therapeutics Inc, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Ultragenyx Pharmaceutical Inc, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects, 2022
- Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products, 2022
- Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), 2022
- Number of Products under Development by Companies, 2022
- Number of Products by Top 10 Targets, 2022
- Number of Products by Stage and Top 10 Targets, 2022
- Number of Products by Top 10 Mechanism of Actions, 2022
- Number of Products by Stage and Top 10 Mechanism of Actions, 2022
- Number of Products by Top 10 Routes of Administration, 2022
- Number of Products by Stage and Top 10 Routes of Administration, 2022
- Number of Products by Top 10 Molecule Types, 2022
- Number of Products by Stage and Top 10 Molecule Types, 2022
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- 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
