Table of Content


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
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 investigatiol 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 alysis 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) desigtion 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 registratiol 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 Us
Disclaimer



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


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