Metachromatic Leukodystrophy (MLD) – Pipeline Review, H2 2019

“Global Markets Direct’s, ‘Metachromatic Leukodystrophy (MLD) – Pipeline Review, H2 2019’, provides an overview of the Metachromatic Leukodystrophy (MLD) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Metachromatic Leukodystrophy (MLD), complete with analysis by stage of development, drug target, 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 therapeutic development for Metachromatic Leukodystrophy (MLD) and features dormant and discontinued projects.

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.”

Scope


– The report provides a snapshot of the global therapeutic landscape of Metachromatic Leukodystrophy (MLD)

– The report reviews pipeline therapeutics for Metachromatic Leukodystrophy (MLD) 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 Metachromatic Leukodystrophy (MLD) therapeutics and enlists all their major and minor projects

– The report assesses Metachromatic Leukodystrophy (MLD) therapeutics based on drug target, 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 related to pipeline therapeutics for Metachromatic Leukodystrophy (MLD)”

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 important and diverse types of therapeutics under development for Metachromatic Leukodystrophy (MLD)

- 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 Metachromatic Leukodystrophy (MLD) pipeline depth and focus of Indication therapeutics

- 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

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

“ArmaGen Inc

EnhanX Biopharm Inc

Homology Medicines Inc

Magenta Therapeutics Inc

Orchard Therapeutics Plc

Recursion Pharmaceuticals Inc

Takeda Pharmaceutical Co Ltd”

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Metachromatic Leukodystrophy (MLD) Overview

Metachromatic Leukodystrophy (MLD) Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Metachromatic Leukodystrophy (MLD) Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Metachromatic Leukodystrophy (MLD) Companies Involved in Therapeutics Development

ArmaGen Inc

Homology Medicines Inc

Recursion Pharmaceuticals Inc

RegenxBio Inc

Shire Plc

Metachromatic Leukodystrophy (MLD) Drug Profiles

AGT-183 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

DUOC-01 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene therapy for Metachromatic Leukodystrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Arylsulfatase A for Metachromatic Leukodystrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

GSK-2696274 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

MGTA-456 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

SHP-611 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Metachromatic Leukodystrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Stem Cell Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Metachromatic Leukodystrophy (MLD) Dormant Projects

Metachromatic Leukodystrophy (MLD) Discontinued Products

Metachromatic Leukodystrophy (MLD) Product Development Milestones

Featured News & Press Releases

May 03, 2018: Orchard Therapeutics OTL-200 Receives Rare Pediatric Disease Designation from FDA for Treatment of Metachromatic Leukodystrophy

Apr 05, 2018: Magenta Therapeutics Announces First Patient Transplanted with MGTA-456 in Phase 2 Study in Inherited Metabolic Disorders

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

“List of Tables

Number of Products under Development for Metachromatic Leukodystrophy (MLD), H2 2019

Number of Products under Development by Companies, H2 2019

Number of Products under Development by Universities/Institutes, H2 2019

Products under Development by Companies, H2 2019

Products under Development by Universities/Institutes, H2 2019

Number of Products by Stage and Target, H2 2019

Number of Products by Stage and Mechanism of Action, H2 2019

Number of Products by Stage and Route of Administration, H2 2019

Number of Products by Stage and Molecule Type, H2 2019

Metachromatic Leukodystrophy (MLD) – Pipeline by ArmaGen Inc, H2 2019

Metachromatic Leukodystrophy (MLD) – Pipeline by EnhanX Biopharm Inc, H2 2019

Metachromatic Leukodystrophy (MLD) – Pipeline by Homology Medicines Inc, H2 2019

Metachromatic Leukodystrophy (MLD) – Pipeline by Magenta Therapeutics Inc, H2 2019

Metachromatic Leukodystrophy (MLD) – Pipeline by Orchard Therapeutics Plc, H2 2019

Metachromatic Leukodystrophy (MLD) – Pipeline by Recursion Pharmaceuticals Inc, H2 2019

Metachromatic Leukodystrophy (MLD) – Pipeline by Takeda Pharmaceutical Co Ltd, H2 2019

Metachromatic Leukodystrophy (MLD) – Dormant Projects, H2 2019

Metachromatic Leukodystrophy (MLD) – Discontinued Products, H2 2019”

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