Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Overview
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Companies Involved in Therapeutics Development
Applied Genetic Technologies Corp
bluebird bio Inc
Magenta Therapeutics Inc
MedDay SA
Minoryx Therapeutics sl
NeuroVia Inc
Orpheris Inc
Poxel SA
SOM Biotech SL
SwanBio Therapeutics Ltd
Viking Therapeutics Inc
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Drug Profiles
biotin - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
dimethyl fumarate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DUOC-01 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
elivaldogene autotemcel - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Adrenomyeloneuropathy and Neurodegenerative Diseases - 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 ABCD1 for Adrenoleukodystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate ABCD1 for Adrenoleukodystrophy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
leriglitazone hydrochloride - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NV-1934 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OP-101 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PXL-065 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules for Metabolic Disorders and Rare Diseases - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SOM-1201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
spanlecortemlocel - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
temsirolimus - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
VK-0214 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Dormant Projects
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - 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
Sep 20, 2019: bluebird bio reports new data for Lenti-D gene therapy
Sep 04, 2019: Magenta Therapeutics announces FDA regenerative medicine advanced therapy (RMAT) desigtion granted to MGTA-456 for the treatment of Inherited Metabolic Disorders
Sep 03, 2019: bluebird bio to present data from clinical development program of Lenti-DTM Gene Therapy for Cerebral Adrenoleukodystrophy (CALD) at the 13th European Pediatric Neurology Society (EPNS) Congress
May 09, 2019: Magenta Therapeutics presents updated phase 2 clinical data on MGTA-456 cell therapy at American Academy of Neurology Annual Meeting
Dec 02, 2018: Magenta Therapeutics presents new data from phase 2 study of MGTA-456 cell therapy in patients with inherited metabolic disorders
Nov 02, 2018: Magenta presents preclinical data on MGTA-456 at ASH annual meeting 2018
Sep 05, 2018: bluebird bio presents updated data from phase 2/3 starbeam study of investigatiol Lenti-D gene therapy for CALD and initial data from observatiol study ALD-103 of allogeneic hematopoietic stem cell transplant in CALD at 2018 SSIEM
Aug 03, 2018: bluebird bio to Present Updated Data for Lenti-D Gene Therapy for Cerebral Adrenoleukodystrophy at SSIEM 2018 Symposium
May 23, 2018: FDA Grants Breakthrough Therapy Desigtion to Lenti-D for the Treatment of Cerebral Adrenoleukodystrophy
Apr 05, 2018: Magenta Therapeutics Announces First Patient Transplanted with MGTA-456 in Phase 2 Study in Inherited Metabolic Disorders
Dec 04, 2017: Orpheris Appoints Neil Warma to Board of Directors
Nov 29, 2017: Orpheris Obtains FDA Agreement On Single, Seamless Phase 1/2/3 Trial to Registration for Its Lead Drug Candidate, OP-101, as a Treatment for Childhood Cerebral Andrenoleukodystrophy (ccALD)
Nov 27, 2017: Orpheris Receives Orphan Drug Desigtion from the U.S. FDA for its Lead Drug Candidate, OP-101, for the Treatment of ccALD
Oct 23, 2017: Viking Therapeutics Presents Results from Proof-of-Concept Study of VK0214 in In Vivo Model of X-Linked Adrenoleukodystrophy at the 87th Annual Meeting of the American Thyroid Association
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Number of Products under Development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex), 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 Routes of Administration, H1 2020
Number of Products by Stage and Top 10 Routes of Administration, H1 2020
Number of Products by Molecule Types, H1 2020
Number of Products by Stage and Molecule Types, H1 2020


List of Tables


Number of Products under Development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex), 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
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Applied Genetic Technologies Corp, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by bluebird bio Inc, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Magenta Therapeutics Inc, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by MedDay SA, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Minoryx Therapeutics sl, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by NeuroVia Inc, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Orpheris Inc, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Poxel SA, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by SOM Biotech SL, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by SwanBio Therapeutics Ltd, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Pipeline by Viking Therapeutics Inc, H1 2020
Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) - Dormant Projects, H1 2020