N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) - Pipeline Review, H1 2018

June 2018 | 48 pages | ID: N32CEF6CF94EN
Global Markets Direct

Abstracts

N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) - Pipeline Review, H1 2018

SUMMARY

N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) pipeline Target constitutes close to 6 molecules. The latest report N Sulphoglucosamine Sulphohydrolase - Pipeline Review, H1 2018, outlays comprehensive information on the N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type.

N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) - N-sulphoglucosamine sulphohydrolase is an enzyme encoded by the SGSH gene. This enzyme is involved in the lysosomal degradation of heparan sulfate. Mutations in this gene are associated with Sanfilippo syndrome A, one type of the lysosomal storage disease mucopolysaccaridosis III. The molecules developed by companies in Phase II, IND/CTA Filed and Preclinical stages are 4, 1 and 1 respectively. Report covers products from therapy areas Genetic Disorders which include indications Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ).

Furthermore, this report also reviews key players involved in N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) targeted therapeutics development with respective active and dormant or discontinued projects. Driven by data and information sourced from 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.

Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

SCOPE

• The report provides a snapshot of the global therapeutic landscape for N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1)
• The report reviews N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) targeted therapeutics under development by companies and universities/research institutes based on information derived from company and industry-specific sources
• The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages
• The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities
• The report reviews key players involved in N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) targeted therapeutics and enlists all their major and minor projects
• The report assesses N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) targeted therapeutics based on mechanism of action (MoA), route of administration (RoA) and molecule type
• The report summarizes all the dormant and discontinued pipeline projects
• The report reviews latest news and deals related to N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) targeted therapeutics

REASONS TO BUY

• Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies
• Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
• Identify and understand the targeted therapy areas and indications for N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1)
• Identify the use of drugs for target identification and drug repurposing
• Identify potential new clients or partners in the target demographic
• Develop strategic initiatives by understanding the focus areas of leading companies
• Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics
• Devise corrective measures for pipeline projects by understanding N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) development landscape
• 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

Contents

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
ArmaGen Inc
Laboratorios Del Dr Esteve SA
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
LYSSAF-301 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LYSSAF-302 - 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
May 18, 2018: Abeona Therapeutics Provides Clinical Update on MPS IIIA Gene Therapy Trial at the 21st Annual ASGCT Meeting
Apr 23, 2018: Abeona Announces FDA Grants RMAT Designation to ABO-102 Gene Therapy in MPS IIIA
Feb 08, 2018: Abeona Therapeutics Reports Top-Line Data from Phase 1/2 Gene Therapy Trial in MPS IIIA
Feb 06, 2018: Lysogene gets PIP Green Light from European Medicines Agency on MPS IIIA Pivotal Study; Paving Way for Clinical Trial Application Approvals
Feb 01, 2018: Lysogene to Present Five Year Clinical Data in MPS IIIA at the 14th Annual WORLDSymposium
Jan 23, 2018: FDA accepts investigational new drug application and grants Fast Track status for SOBI003 for the treatment of MPS IIIA
Nov 09, 2017: Abeona Enrolls First Subject in Spain in Ongoing Phase 1/2 Clinical Trial in MPS IIIA
Oct 11, 2017: Abeona Therapeutics Enrolls First Two Patients in Pivotal Expansion of Phase 1/2 Clinical Trial in MPS IIIA
Oct 06, 2017: Abeona Announces Top-Line One Year Data from ABO-102 MPS IIIA Trial at ARM’s Cell & Gene Meeting on the Mesa
Oct 05, 2017: Lysogene to Host Key Opinion Leader Meeting on Gene Therapy for Patients with Sanfilippo Syndrome Type A in New York City
Sep 28, 2017: Abeona Therapeutics and Brammer Bio Announce Collaboration for Commercial Translation of ABO-102
Aug 17, 2017: Abeona Therapeutics Provides Update on ABO-102 at R&D Day on October 11, 2017
Aug 16, 2017: Abeona Therapeutics Announces Pivotal Expansion of ABO-102 Gene Therapy Clinical Trials in Sanfilippo Syndrome Type A
Jul 05, 2017: FDA grants SOBI003 Orphan Drug Designation for the treatment of MPS IIIA
May 30, 2017: Lysogene Completes Enrollment in First International Pivotal Observational Study in MPS IIIA
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List Of Tables

LIST OF TABLES

Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indication, H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Pipeline by Abeona Therapeutics Inc, H1 2018
Pipeline by ArmaGen Inc, H1 2018
Pipeline by Laboratorios Del Dr Esteve SA, H1 2018
Pipeline by Lysogene SAS, H1 2018
Pipeline by Swedish Orphan Biovitrum AB, H1 2018
Discontinued Products, H1 2018

List Of Figures

LIST OF FIGURES

Number of Products under Development by Stage of Development, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018

COMPANIES MENTIONED

Abeona Therapeutics Inc
ArmaGen Inc
Laboratorios Del Dr Esteve SA
Lysogene SAS
Swedish Orphan Biovitrum AB