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The publisher's latest Pharmaceutical and Healthcare disease pipeline guide Metachromatic Leukodystrophy - Pipeline Review, H1 2020, provides an overview of the Metachromatic Leukodystrophy (Central Nervous System) pipeline landscape.
Metachromatic leukodystrophy is an inherited disorder characterized by the accumulation of fats called sulfatides in cells. Symptoms include vision problems leading to blindness, personality changes, and motor disturbances such as clumsiness, muscle weakness (hypotonia), rigidity, inability to coordinate movement (ataxia), and/or muscle spasms especially of the neck, spine, arms, and legs. Treatment for metachromatic leukodystrophy is symptomatic and supportive.
Report Highlights
The publisher's Pharmaceutical and Healthcare latest pipeline guide Metachromatic Leukodystrophy - Pipeline Review, H1 2020, provides comprehensive information on the therapeutics under development for Metachromatic Leukodystrophy (Central Nervous System), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.
The Metachromatic Leukodystrophy (Central Nervous System) pipeline guide also reviews of key players involved in therapeutic development for Metachromatic Leukodystrophy (MLD) and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase II, Preclinical and Discovery stages are 1, 2, 2 and 1 respectively. Similarly, the Universities portfolio in Phase II and Phase I stages comprises 2 and 1 molecules, respectively.
Metachromatic Leukodystrophy (Central Nervous System) pipeline guide 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. The guide is built using data and information sourced from the publisher'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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
Scope
- The pipeline guide provides a snapshot of the global therapeutic landscape of Metachromatic Leukodystrophy (Central Nervous System).
- The pipeline guide reviews pipeline therapeutics for Metachromatic Leukodystrophy (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 (Central Nervous System) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Metachromatic Leukodystrophy (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 latest news related to pipeline therapeutics for Metachromatic Leukodystrophy (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 portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Metachromatic Leukodystrophy (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 it’s most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Metachromatic Leukodystrophy (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 pipeline.
Table of Contents
- Introduction
- 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
- Magenta Therapeutics Inc
- Orchard Therapeutics Plc
- Passage Bio Inc
- Takeda Pharmaceutical Co Ltd
- 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 and Adrenoleukodystrophy - 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
- HMI-202 - Drug Profile
- Product Description
- Mechanism Of Action
- R&D Progress
- OTL-200 - Drug Profile
- Product Description
- Mechanism Of Action
- R&D Progress
- PBML-04 - Drug Profile
- Product Description
- Mechanism Of Action
- R&D Progress
- spanlecortemlocel - Drug Profile
- Product Description
- Mechanism Of Action
- R&D Progress
- TAK-611 - 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
- Feb 20, 2020: Magenta Therapeutics announces updated phase 2 data on MGTA-456 cell therapy, demonstrating continued durability in inherited metabolic disorders
- Feb 13, 2020: Homology Medicines presents new data characterizing AAVHSCs as potential gene therapies for nervous system disorders
- Feb 10, 2020: Orchard Therapeutics announces presentation on OTL-200 at 16th Annual WORLD Symposium
- Dec 02, 2019: Orchard Therapeutics announces the filing and validation of marketing authorization application by European Medicines Agency for OTL-200 for the treatment of metachromatic leukodystrophy
- Nov 18, 2019: European Medicines Agency grants Orchard Therapeutics accelerated assessment of OTL-200 for patients with Metachromatic Leukodystrophy
- Oct 22, 2019: Orchard Therapeutics presents data from OTL-200 in patients with Metachromatic Leukodystrophy using Cryopreservation
- 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
- Sep 30, 2019: Orchard Therapeutics announces presentation of new clinical data for OTL-200 for Metachromatic Leukodystrophy at upcoming European Society of Gene & Cell Therapy Annual Congress
- Sep 04, 2019: Orchard Therapeutics presents an integrated data analysis demonstrating sustained clinical benefit of OTL-200 for the treatment of Metachromatic Leukodystrophy
- Sep 04, 2019: Magenta Therapeutics announces FDA regenerative medicine advanced therapy (RMAT) designation granted to MGTA-456 for the treatment of Inherited Metabolic Disorders
- Aug 27, 2019: Orchard Therapeutics announces presentation on OTL-200 at Upcoming Society for the Study of Inborn Errors of Metabolism (SSIEM) 2019 Annual Symposium
- May 09, 2019: Magenta Therapeutics presents updated phase 2 clinical data on MGTA-456 cell therapy at American Academy of Neurology Annual Meeting
- Mar 27, 2019: Orchard Therapeutics presents new registrational data demonstrating sustained clinical benefit of OTL-200 for the treatment of MLD
- Mar 11, 2019: Orchard Therapeutics announces acceptance of OTL-200 abstract for MLD at the 45th Annual Meeting of the European Society for Blood and Bone Marrow Transplantation (EBMT)
- Feb 25, 2019: Magenta Therapeutics Presents Clinical and Preclinical Data on MGTA-456 Cell Therapy in Best Abstracts Sessions at Transplant and Cellular Therapy (TCT) Annual Meeting
- Appendix
- Methodology
- Coverage
- Secondary Research
- Primary Research
- Expert Panel Validation
- Contact
- Disclaimer
List of Tables
- Number of Products under Development for Metachromatic Leukodystrophy (MLD), H1 2020
- Number of Products under Development by Companies, H1 2020
- Number of Products under Development by Universities/Institutes, H1 2020
- Products under Development by Companies, H1 2020
- Products under Development by Universities/Institutes, H1 2020
- Number of Products by Stage and Target, H1 2020
- Number of Products by Stage and Mechanism of Action, H1 2020
- Number of Products by Stage and Route of Administration, H1 2020
- Number of Products by Stage and Molecule Type, H1 2020
- Metachromatic Leukodystrophy (MLD) - Pipeline by ArmaGen Inc, H1 2020
- Metachromatic Leukodystrophy (MLD) - Pipeline by Homology Medicines Inc, H1 2020
- Metachromatic Leukodystrophy (MLD) - Pipeline by Magenta Therapeutics Inc, H1 2020
- Metachromatic Leukodystrophy (MLD) - Pipeline by Orchard Therapeutics Plc, H1 2020
- Metachromatic Leukodystrophy (MLD) - Pipeline by Passage Bio Inc, H1 2020
- Metachromatic Leukodystrophy (MLD) - Pipeline by Takeda Pharmaceutical Co Ltd, H1 2020
- Metachromatic Leukodystrophy (MLD) - Dormant Projects, H1 2020
- Metachromatic Leukodystrophy (MLD) - Discontinued Products, H1 2020
List of Figures
- Number of Products under Development for Metachromatic Leukodystrophy (MLD), H1 2020
- Number of Products under Development by Companies, H1 2020
- Number of Products under Development by Universities/Institutes, H1 2020
- Number of Products by Top 10 Targets, H1 2020
- Number of Products by Stage and Top 10 Targets, H1 2020
- Number of Products by Top 10 Mechanism of Actions, H1 2020
- Number of Products by Stage and Top 10 Mechanism of Actions, H1 2020
- Number of Products by Top 10 Routes of Administration, H1 2020
- Number of Products by Stage and Top 10 Routes of Administration, H1 2020
- Number of Products by Top 10 Molecule Types, H1 2020
- Number of Products by Stage and Top 10 Molecule Types, H1 2020
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- ArmaGen Inc
- Homology Medicines Inc
- Magenta Therapeutics Inc
- Orchard Therapeutics Plc
- Passage Bio Inc
- Takeda Pharmaceutical Co Ltd
