List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) - Overview
N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) - Therapeutics Development
Products under Development by Stage of Development
Products under Development by Therapy Area
Products under Development by Indication
Products under Development by Companies
N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) - Therapeutics Assessment
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) - Companies Involved in Therapeutics Development
Abeona Therapeutics Inc
Amicus Therapeutics Inc
ArmaGen Inc
Denali Therapeutics Inc
Esteve Pharmaceuticals SA
JCR Pharmaceuticals Co Ltd
Lysogene SAS
Swedish Orphan Biovitrum AB
N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) - Drug Profiles
ABO-102 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AGT-184 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
EGT-101 - 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
JR-441 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LYSSAF-301 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LYSSAF-302 - 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
SOBI-003 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) - Discontinued Products
N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) - Product Development Milestones
Featured News & Press Releases
Feb 25, 2020: Lysogene receives FDA fast track designation for LYS-SAF302 gene therapy in MPS IIIA
Feb 12, 2020: Abeona Therapeutics announces presentation on its MPS IIIA drug candidate ABO-102 at WORLDSymposium
Dec 20, 2019: Abeona Therapeutics receives European Medicines Agency PRIME Designation for ABO-102 Gene Therapy in MPS IIIA
Dec 18, 2019: Lysogene announces the publication of an article in the scientific journal "molecular therapy methods & clinical development” demonstrating the potential of its drug candidate LYS-SAF302
Oct 21, 2019: Abeona Therapeutics announces presentations at the 27th European Society of Gene and Cell Therapy (ESGCT) Congress
Jul 25, 2019: Abeona Therapeutics announces positive interim data from the ABO-102 phase 1/2 gene therapy clinical trial in MPS IIIA
Jul 25, 2019: Abeona Therapeutics announces positive interim data from the ABO-102 phase 1/2 gene therapy clinical trial in MPS IIIA
Jun 11, 2019: Lysogene announces first European patient treated in AAVance, Phase 2/3 clinical trial investigating LYS-SAF302, a gene therapy for the treatment of MPS IIIA (Sanfilippo Syndrome Type A)
Mar 22, 2019: Lysogene announces presentations at upcoming scientific and family conferences
Feb 14, 2019: Lysogene and Sarepta Therapeutics Announce Dosing of the First Patient in AAVance, a Phase 2/3 Clinical Trial Investigating LYS-SAF302, a Gene Therapy for the Treatment of MPS IIIA (Sanfilippo Syndrome Type A)
Jan 31, 2019: Abeona Therapeutics to present new supportive data for Novel Gene Therapies at WORLDSymposium
Dec 06, 2018: Abeona Therapeutics provides update on Sanfilippo Syndrome drug candidate ABO-102 at 2018 R&D Day
Sep 14, 2018: JCR to initiate development of a new drug candidate for sanfilippo syndrome Type A using J-Brain Cargo
Sep 05, 2018: Lysogene Announces FDA approval of IND Application to Initiate Phase 2-3 Clinical Trial in MPS IIIA
Aug 10, 2018: First patient dosed in phase 1/2 study evaluating SOBI003 for treatment of mucopolysaccharidosis type IIIA (MPS IIIA)
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
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List of Tables
Number of Products under Development by Stage of Development, H1 2020
Number of Products under Development by Therapy Areas, H1 2020
Number of Products under Development by Indication, H1 2020
Number of Products under Development by Companies, H1 2020
Products under Development by Companies, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Pipeline by Abeona Therapeutics Inc, H1 2020
Pipeline by Amicus Therapeutics Inc, H1 2020
Pipeline by ArmaGen Inc, H1 2020
Pipeline by Denali Therapeutics Inc, H1 2020
Pipeline by Esteve Pharmaceuticals SA, H1 2020
Pipeline by JCR Pharmaceuticals Co Ltd, H1 2020
Pipeline by Lysogene SAS, H1 2020
Pipeline by Swedish Orphan Biovitrum AB, H1 2020
Discontinued Products, H1 2020

List of Figures
Number of Products under Development by Stage of Development, H1 2020
Number of Products under Development by Therapy Areas, H1 2020
Number of Products under Development by Top 10 Indications, H1 2020
Number of Products by Mechanism of Actions, H1 2020
Number of Products by Stage and Mechanism of Actions, H1 2020
Number of Products by Routes of Administration, H1 2020
Number of Products by Stage and Routes of Administration, H1 2020
Number of Products by Molecule Types, H1 2020
Number of Products by Stage and Molecule Types, H1 2020

Companies Mentioned
• Abeona Therapeutics Inc
• Amicus Therapeutics Inc
• ArmaGen Inc
• Denali Therapeutics Inc
• Esteve Pharmaceuticals SA
• JCR Pharmaceuticals Co Ltd
• Lysogene SAS
• Swedish Orphan Biovitrum AB