N Sulphoglucosamine Sulphohydrolase – Pipeline Review, H1 2020

Global Markets Direct’s, ‘N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) – Pipeline Review, H1 2020’, provides in depth analysis on N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) targeted pipeline therapeutics. The report provides comprehensive information complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1) targeted therapeutics development and features dormant and discontinued projects. The report analyses the pipeline products from therapy areas Genetic Disorders and Central Nervous System under development targeting N Sulphoglucosamine Sulphohydrolase (Sulfoglucosamine Sulfamidase or Sulphamidase or SGSH or EC 3.10.1.1).

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report 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. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report 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.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

*Updated report will be delivered in 48 hours of order confirmation.

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

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

Table of Contents

Table of Contents

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

Contact Us

Disclaimer

List of Tables

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

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

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