Table of Content


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
Abeo Therapeutics Inc
Allievex Corp
Amicus Therapeutics Inc
ArmaGen Inc
Deli 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
akinra - 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 HGST for Mucopolysaccharidosis Type IIIC - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate GLU for Mucopolysaccharidosis Type IIIB - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate GLU 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
Recombint N-Acetylgalactosamine-6-Sulfatase Replacement for Mucopolysaccharidosis Type IIID - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombint 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 desigtion for SLS-005 (Trehalose) in Sanfilippo Syndrome
Apr 30, 2020: Seelos Therapeutics receives Orphan Drug Desigtion 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 tiol MPS Society Family Conference
Dec 04, 2019: Seelos Therapeutics receives notice of allowance for an additiol 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 Investigatiol 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



List of Figures


Number of Products under Development for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ), H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products by Top 10 Targets, H1 2020
Number of Products by Stage and Top 10 Targets, H1 2020
Number of Products by Top 10 Mechanism of Actions, H1 2020
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2020
Number of Products by Top 10 Routes of Administration, H1 2020
Number of Products by Stage and Top 10 Routes of Administration, H1 2020
Number of Products by Top 10 Molecule Types, H1 2020
Number of Products by Stage and Top 10 Molecule Types, H1 2020


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