Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline Review, H1 2020

Global Markets Direct’s, ‘Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) – Pipeline Review, H1 2020’, provides an overview of the Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex), 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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) 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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex)

– The report reviews pipeline therapeutics for Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) 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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) therapeutics and enlists all their major and minor projects

– The report assesses Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) 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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex)

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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex)

- 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 Adrenoleukodystrophy (Adrenomyeloneuropathy/ Schilder-Addison Complex) 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

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

Table of Contents

Table of Contents

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) designation 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 investigational Lenti-D gene therapy for CALD and initial data from observational 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 Designation 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 Designation 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 Tables

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

List of Figures

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

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