Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline Review, H1 2020
Summary
Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Mucopolysaccharidosis II (MPS II) (Hunter Syndrome ) - Pipeline Review, H1 2020, provides an overview of the Mucopolysaccharidosis II (MPS II) (Hunter 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 II (MPS II) (Hunter Syndrome ) - Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Mucopolysaccharidosis II (MPS II) (Hunter 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 II (MPS II) (Hunter 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, Discovery and Unknown stages are 2, 6, 1, 9, 4 and 1 respectively.
Mucopolysaccharidosis II (MPS II) (Hunter 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 II (MPS II) (Hunter Syndrome ) (Genetic Disorders).
- The pipeline guide reviews pipeline therapeutics for Mucopolysaccharidosis II (MPS II) (Hunter 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 II (MPS II) (Hunter Syndrome ) (Genetic Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Mucopolysaccharidosis II (MPS II) (Hunter 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 II (MPS II) (Hunter 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 II (MPS II) (Hunter 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 II (MPS II) (Hunter 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
List of Tables
List of Figures
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
Products under Development by Companies
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
Abeona Therapeutics Inc
Allievex Corp
Amicus Therapeutics Inc
ArmaGen Inc
Denali Therapeutics Inc
Esteve Pharmaceuticals SA
JCR Pharmaceuticals Co Ltd
Jupiter Orphan Therapeutics Inc
Lacerta Therapeutics Inc
Lysogene SAS
Orchard Therapeutics Plc
Phoenix Nest Inc
Seelos Therapeutics, Inc.
Swedish Orphan Biovitrum AB
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Drug Profiles
ABO-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ABO-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AGT-184 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AGT-187 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
anakinra - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EGT-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EGT-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Mucopolysaccharidosis III (Sanfilippo Syndrome) - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate HGSNAT for Mucopolysaccharidosis Type IIIC - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate NAGLU for Mucopolysaccharidosis Type IIIB - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate NAGLU for Mucopolysaccharidosis Type IIIB - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate SGSH for Mucopolysaccharidosis Type IIIA - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JOT-109 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JR-441 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
JR-446 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LYSSAF-302 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OTL-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OTL-202 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Proteins for Mucopolysaccharidosis Type IIIC - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant N-Acetylgalactosamine-6-Sulfatase Replacement for Mucopolysaccharidosis Type IIID - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Sulphamidase Replacement for Mucopolysaccharidosis III (Sanfilippo Syndrome) - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tralesinidase alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
trehalose - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
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
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)
Aug 22, 2019: Seelos Therapeutics announces FDA acceptance of IND application for SLS-005 for mucopolysaccharidosis type III (sanfilippo syndrome)
Jul 17, 2019: Seelos Therapeutics announces Investigational New Drug application submission for SLS-005
Jul 15, 2019: Seelos Therapeutics announces receipt of a grant for funding of SLS-005 Program
Jun 06, 2019: Seelos Therapeutics provides update on its pipeline product SLS-005
Feb 14, 2019: Bioblast Pharma announces collaboration with Team Sanfilippo to evaluate trehalose for the treatment of Sanfilippo syndrome
Feb 04, 2019: BioMarin announces presentation of data on drug candidate BMN 250 at 15th Annual WORLDSymposium 2019
Nov 07, 2018: Phoenix Nest Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center (LA BioMed) and Washington University in St. Louis announce a New NIH Grant for Sanfilippo Syndrome (MPSIIID)
Sep 14, 2018: JCR to initiate development of a new drug candidate for sanfilippo syndrome Type A using J-Brain Cargo
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
Tables and Figures
List of Tables
Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H1 2020
Number of Products under Development by Companies, H1 2020
Products under Development by Companies, H1 2020
Products under Development by Companies, H1 2020 (Contd..1), H1 2020
Number of Products by Stage and Target, H1 2020
Number of Products by Stage and Mechanism of Action, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Abeona Therapeutics Inc, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Allievex Corp, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Amicus Therapeutics Inc, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by ArmaGen Inc, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Denali Therapeutics Inc, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Esteve Pharmaceuticals SA, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by JCR Pharmaceuticals Co Ltd, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Jupiter Orphan Therapeutics Inc, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lacerta Therapeutics Inc, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Lysogene SAS, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Orchard Therapeutics Plc, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Phoenix Nest Inc, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Seelos Therapeutics, Inc., H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Pipeline by Swedish Orphan Biovitrum AB, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Dormant Projects, H1 2020
Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) - Discontinued Products, H1 2020
Companies Mentioned
Abeona Therapeutics Inc
Allievex Corp
Amicus Therapeutics Inc
ArmaGen Inc
Denali Therapeutics Inc
Esteve Pharmaceuticals SA
JCR Pharmaceuticals Co Ltd
Jupiter Orphan Therapeutics Inc
Lacerta Therapeutics Inc
Lysogene SAS
Orchard Therapeutics Plc
Phoenix Nest Inc
Seelos Therapeutics, Inc.
Swedish Orphan Biovitrum AB
Reason to Buy
- Current and future of Pharma & Healthcare Market outlook in the developed and emerging markets
- Highlights key business priorities in order to assist companies to realign their business strategies
- The segment that is expected to dominate the Pharma & Healthcare Market
- Regions that are expected to witness the fastest growth during the forecast period
- Identify the latest developments, Pharma & Healthcare Market shares, and strategies employed by the major market players
- Save and reduce time carrying out entry-level research by identifying the growth, size, leading players and segments in the global Pharma & Healthcare Market
- Develop/modify business expansion plans by using substantial growth offering developed and emerging markets