Wilson Disease – Drugs in Development, 2021

Global Markets Direct’s, ‘Wilson Disease – Drugs in Development, 2021’, provides an overview of the Wilson Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Wilson Disease, 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 Wilson Disease 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 Wilson Disease

– The report reviews pipeline therapeutics for Wilson Disease 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 Wilson Disease therapeutics and enlists all their major and minor projects

– The report assesses Wilson Disease 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 Wilson Disease

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 Wilson Disease

- 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 Wilson Disease 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

Alexion Pharmaceuticals Inc

Aligen Therapeutics SL

Arbor Pharmaceuticals, LLC

Deep Genomics Inc

DepYmed Inc

Enveda Biosciences Inc

Generation Bio Co

GMP-Orphan SA

Krisani Bio Sciences Pvt Ltd

LambdaGen Therapeutics

Nobelpharma Co Ltd

Ultragenyx Pharmaceutical Inc

Vivet Therapeutics SAS

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Wilson Disease – Overview

Wilson Disease – Therapeutics Development

Pipeline Overview

Pipeline by Companies

Products under Development by Companies

Wilson Disease – Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Wilson Disease – Companies Involved in Therapeutics Development

Alexion Pharmaceuticals Inc

Aligen Therapeutics SL

Arbor Pharmaceuticals, LLC

Deep Genomics Inc

DepYmed Inc

Enveda Biosciences Inc

Generation Bio Co

GMP-Orphan SA

Krisani Bio Sciences Pvt Ltd

LambdaGen Therapeutics

Nobelpharma Co Ltd

Ultragenyx Pharmaceutical Inc

Vivet Therapeutics SAS

Wilson Disease – Drug Profiles

CM-1186 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

DG-12P1 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

DPM-1001 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy for Wilson’s Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate ATP7B for Wilson Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate ATP7B for Wilson Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

KBHD-001 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

methanobactin – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules for Wilson Disease – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

tiomolibdate choline – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

trientine tetrahydrochloride – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

UX-701 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

VTX-801 – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

zinc acetate – Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Wilson Disease – Dormant Projects

Wilson Disease – Product Development Milestones

Featured News & Press Releases

Oct 18, 2021: Ultragenyx initiates Cyprus2+, a pivotal clinical trial evaluating UX701 gene therapy for the treatment of Wilson disease

Sep 02, 2021: Orphalan announces FDA acceptance for filing of New Drug Application (NDA) for trientine tetrahydrochloride (TETA 4HCl) for the treatment of Wilson’s Disease

Aug 27, 2021: Alexion’s drug meets primary goal in Phase III Wilson disease trial

Jun 25, 2021: Orphalan announces positive top line data with Trientine Tetrahydrochloride for maintenance patients with Wilson’s disease

Jan 21, 2021: Ultragenyx announces FDA clearance of Investigational New Drug (IND) application for UX701, a new gene therapy for the treatment of Wilson disease

Dec 09, 2020: Ultragenyx announces Orphan Drug Designation for UX701 for the treatment of Wilson disease

Jun 27, 2018: Progress toward improved Wilson's disease drug

Jun 01, 2018: Wilson Therapeutics to present data from WTX101 Phase 2 extension study at 4th Congress of the European Academy of Neurology

Apr 12, 2018: Promising preliminary long-term data for WTX101 in Wilson Disease presented at EASL Annual Meeting

Mar 28, 2018: Preliminary Long-Term Data For WTX101 In Wilson Disease Accepted As A Late-Breaker Presentation At EASL Annual Meeting

Feb 16, 2018: First patient enrolled in pivotal Phase 3 FOCuS trial evaluating WTX101 for the treatment of Wilson Disease

Dec 14, 2017: WTX101 Granted Fast Track Designation by the U.S. FDA for the Treatment of Wilson Disease

Oct 23, 2017: Wilson Therapeutics reaches agreement with the FDA and EMA to initiate pivotal Phase 3 FOCuS study with WTX101 in Wilson Disease

Oct 20, 2017: Promising Preliminary Long-term Data For WTX101 In Wilson Disease Highlighted at The Liver Meeting

Oct 06, 2017: Phase 2 Clinical Trial Data for WTX101 Published in The Lancet Gastroenterology & Hepatology

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 Wilson Disease, 2021

Number of Products under Development by Companies, 2021

Products under Development by Companies, 2021

Number of Products by Stage and Target, 2021

Number of Products by Stage and Mechanism of Action, 2021

Number of Products by Stage and Route of Administration, 2021

Number of Products by Stage and Molecule Type, 2021

Wilson Disease – Pipeline by Alexion Pharmaceuticals Inc, 2021

Wilson Disease – Pipeline by Aligen Therapeutics SL, 2021

Wilson Disease – Pipeline by Arbor Pharmaceuticals, LLC, 2021

Wilson Disease – Pipeline by Deep Genomics Inc, 2021

Wilson Disease – Pipeline by DepYmed Inc, 2021

Wilson Disease – Pipeline by Enveda Biosciences Inc, 2021

Wilson Disease – Pipeline by Generation Bio Co, 2021

Wilson Disease – Pipeline by GMP-Orphan SA, 2021

Wilson Disease – Pipeline by Krisani Bio Sciences Pvt Ltd, 2021

Wilson Disease – Pipeline by LambdaGen Therapeutics, 2021

Wilson Disease – Pipeline by Nobelpharma Co Ltd, 2021

Wilson Disease – Pipeline by Ultragenyx Pharmaceutical Inc, 2021

Wilson Disease – Pipeline by Vivet Therapeutics SAS, 2021

Wilson Disease – Dormant Projects, 2021

List of Figures

List of Figures

Number of Products under Development for Wilson Disease, 2021

Number of Products under Development by Companies, 2021

Number of Products by Targets, 2021

Number of Products by Stage and Targets, 2021

Number of Products by Mechanism of Actions, 2021

Number of Products by Stage and Mechanism of Actions, 2021

Number of Products by Routes of Administration, 2021

Number of Products by Stage and Routes of Administration, 2021

Number of Products by Molecule Types, 2021

Number of Products by Stage and Molecule Types, 2021

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