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
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
Deli 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
akinra - 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 GLU 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 GLU for Mucopolysaccharidosis III (Sanfilippo Syndrome) - Drug Profile
Product Description
Mechanism Of Action
Gene Therapy to Activate GLU 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
Recombint 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 desigtion to JR-441 for the treatment of mucopolysaccharidosis type III A (MPS IIIA)
Jul 28, 2021: M6P Therapeutics presented data on M041, a recombint 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 Intertiol Conference on Sanfilippo Syndrome and related diseases
Aug 25, 2020: Seelos Therapeutics receives European Orphan Drug Desigtion for SLS-005 (Trehalose) in Sanfilippo syndrome
Jul 21, 2020: Seelos Therapeutics receives positive EMA opinion on orphan drug desigtion for SLS-005 (Trehalose) in Sanfilippo syndrome
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)
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures

List of Figures
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

List of Tables

List of Tables
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 Companies, 2022 (Contd..1)
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