Metachromatic Leukodystrophy (MLD) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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Metachromatic leukodystrophy (MLD) is an inherited disorder characterized by the accumulation of fats called sulfatides in cells. The Metachromatic Leukodystrophy pipeline market research report provides comprehensive information on the therapeutics under development for Metachromatic Leukodystrophy, 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 the latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Metachromatic Leukodystrophy and features dormant and discontinued projects.

Metachromatic Leukodystrophy Pipeline Products Market Segmentation by Targets

The key target in the Metachromatic Leukodystrophy pipeline products market is Arylsulfatase A.

Metachromatic Leukodystrophy Pipeline Products Market Segmentation by Mechanisms of Action

The key mechanisms of action in the Metachromatic Leukodystrophy pipeline products market are Arylsulfatase A Activator and Arylsulfatase A Replacement. Arylsulfatase A Activator leads the Metachromatic Leukodystrophy pipeline products market in terms of MoA.

Metachromatic Leukodystrophy Pipeline Products Market Analysis by Mechanisms of Action

Metachromatic Leukodystrophy Pipeline Products Market Analysis by Mechanisms of Action

For more mechanisms of action insights into the Metachromatic Leukodystrophy pipeline products market, download a free report sample

Metachromatic Leukodystrophy Pipeline Products Market Segmentation by Routes of Administration

The key routes of administration in the Metachromatic Leukodystrophy pipeline products market are intravenous, intrathecal, intracerebral, and parenteral. Intravenous leads the Metachromatic Leukodystrophy pipeline products market in terms of RoA.

Metachromatic Leukodystrophy Pipeline Products Market Analysis by Routes of Administration

Metachromatic Leukodystrophy Pipeline Products Market Analysis by Routes of AdministrationFor more routes of administration insights into the Metachromatic Leukodystrophy pipeline products market, download a free report sample

Metachromatic Leukodystrophy Pipeline Products Market Segmentation by Molecule Types

The key molecule types in the Metachromatic Leukodystrophy pipeline products market are gene therapy, gene-modified cell therapy, small molecule, cell therapy, fusion protein, and recombinant enzyme. Gene therapy leads the Metachromatic Leukodystrophy pipeline products market in terms of molecule type.

Metachromatic Leukodystrophy Pipeline Products Market Analysis by Molecule Types

Metachromatic Leukodystrophy Pipeline Products Market Analysis by Molecule TypesFor more molecule type insights into the Metachromatic Leukodystrophy pipeline products market, download a free report sample

MLD Pipeline Products Market - Competitive Landscape

Some of the leading companies in the MLD pipeline products market are Homology Medicines Inc, Affinia Therapeutics, ArmaGen Inc, Gain Therapeutics Inc, Orchard Therapeutics Plc, Recursion Pharmaceuticals Inc, and Takeda Pharmaceutical Co Ltd. Homology Medicines Inc is the leading company in the MLD pipeline products market.

Metachromatic Leukodystrophy Pipeline Products Market Analysis by Companies

Metachromatic Leukodystrophy Pipeline Products Market Analysis by Companies

To know more about the leading players in the Metachromatic Leukodystrophy pipeline products market, download a free report sample

MLD Pipeline Products Market Report Overview

Key Targets Arylsulfatase A
Key Mechanisms of Action Arylsulfatase A Activator and Arylsulfatase A Replacement
Key Routes of Administration Intravenous, Intrathecal, Intracerebral, and Parenteral
Key Molecule Type Gene Therapy, Gene-Modified Cell Therapy, Small Molecule, Cell Therapy, Fusion Protein, and Recombinant Enzyme
Leading Companies Homology Medicines Inc, Affinia Therapeutics, ArmaGen Inc, Gain Therapeutics Inc, Orchard Therapeutics Plc, Recursion Pharmaceuticals Inc, and Takeda Pharmaceutical Co Ltd

Scope

  • The pipeline guide provides a snapshot of the global therapeutic landscape of Metachromatic Leukodystrophy (MLD) (Central Nervous System).
  • The pipeline guide reviews pipeline therapeutics for Metachromatic Leukodystrophy (MLD) (Central Nervous System) by companies and universities/research institutes based on information derived from company and industry-specific sources.
  • The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
  • The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
  • The pipeline guide reviews key companies involved in Metachromatic Leukodystrophy (MLD) (Central Nervous System) therapeutics and enlists all their major and minor projects.
  • The pipeline guide evaluates Metachromatic Leukodystrophy (MLD) (Central Nervous System) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
  • The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
  • The pipeline guide reviews the latest news related to pipeline therapeutics for Metachromatic Leukodystrophy (MLD) (Central Nervous System)

Reasons to Buy

  • Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
  • Recognize emerging players with potentially strong product portfolios and create effective counter-strategies to gain competitive advantage.
  • Find and recognize significant and varied types of therapeutics under development for Metachromatic Leukodystrophy (MLD) (Central Nervous System).
  • Classify potential new clients or partners in the target demographic.
  • Develop tactical initiatives by understanding the focus areas of leading companies.
  • Plan mergers and acquisitions meritoriously by identifying key players and their most promising pipeline therapeutics.
  • Formulate corrective measures for pipeline projects by understanding Metachromatic Leukodystrophy (MLD) (Central Nervous System) 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.
  • Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from the pipeline.

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

Affinia Therapeutics

ArmaGen Inc

Gain Therapeutics Inc

Homology Medicines Inc

Orchard Therapeutics Plc

Recursion Pharmaceuticals Inc

Takeda Pharmaceutical Co Ltd

Metachromatic Leukodystrophy (MLD) – Drug Profiles

AFTX-001 – Drug Profile

Product Description

Mechanism Of Action

AGT-183 – Drug Profile

Product Description

Mechanism Of Action

atidarsagene autotemcel – Drug Profile

Product Description

Mechanism Of Action

cebsulfase alfa – Drug Profile

Product Description

Mechanism Of Action

DUOC-01 – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy – Drug Profile

Product Description

Mechanism Of Action

Gene Therapy to Activate Arylsulfatase A for Metachromatic Leukodystrophy – Drug Profile

Product Description

Mechanism Of Action

HMI-202 – Drug Profile

Product Description

Mechanism Of Action

HMI-204 – Drug Profile

Product Description

Mechanism Of Action

Metachromatic Leukodystrophy – Drug Profile

Product Description

Mechanism Of Action

Small Molecules to Activate Arylsulfatase A for Metachromatic Leukodystrophy – Drug Profile

Product Description

Mechanism Of Action

Metachromatic Leukodystrophy (MLD) – Dormant Projects

Metachromatic Leukodystrophy (MLD) – Discontinued Products

Metachromatic Leukodystrophy (MLD) – Product Development Milestones

Featured News & Press Releases

Feb 13, 2020: Homology Medicines presents new data characterizing AAVHSCs as potential gene therapies for nervous system disorders

Oct 21, 2019: Homology Medicines presents data from investigational MLD gene therapy program HMI-202 at the American Society of Human Genetics (ASHG) 2019 Meeting

Nov 07, 2018: Homology Medicines presents data on platform’s ability to target the central and peripheral nervous system and therapeutic potential in the rare disease Metachromatic Leukodystrophy

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

Table

List of Tables

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

Metachromatic Leukodystrophy (MLD) – Pipeline by Affinia Therapeutics, 2022

Metachromatic Leukodystrophy (MLD) – Pipeline by ArmaGen Inc, 2022

Metachromatic Leukodystrophy (MLD) – Pipeline by Gain Therapeutics Inc, 2022

Metachromatic Leukodystrophy (MLD) – Pipeline by Homology Medicines Inc, 2022

Metachromatic Leukodystrophy (MLD) – Pipeline by Orchard Therapeutics Plc, 2022

Metachromatic Leukodystrophy (MLD) – Pipeline by Recursion Pharmaceuticals Inc, 2022

Metachromatic Leukodystrophy (MLD) – Pipeline by Takeda Pharmaceutical Co Ltd, 2022

Metachromatic Leukodystrophy (MLD) – Dormant Projects, 2022

Metachromatic Leukodystrophy (MLD) – Discontinued Products, 2022

Figures

List of Figures

Number of Products under Development for Metachromatic Leukodystrophy (MLD), 2022

Number of Products under Development by Companies, 2022

Number of Products under Development by Universities/Institutes, 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

Frequently asked questions

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