Huntingtin (Huntington Disease Protein or HTT) – Pipeline Review, H2 2017

Global Markets Direct’s, ‘Huntingtin (Huntington Disease Protein or HTT) – Pipeline Review, H2 2017’, provides in depth analysis on Huntingtin (Huntington Disease Protein or HTT) targeted pipeline therapeutics. The report provides comprehensive information complete with analysis by indications, stage of development, 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 Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics development and features dormant and discontinued projects. The report analyses the pipeline products from therapy areas Central Nervous System and Genetic Disorders under development targeting Huntingtin (Huntington Disease Protein or HTT).

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.

*Updated report will be delivered in 48 hours of order confirmation.

Scope

The report provides a snapshot of the global therapeutic landscape for Huntingtin (Huntington Disease Protein or HTT)

The report reviews Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics under development 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 in Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics and enlists all their major and minor projects

The report assesses Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics based on 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 and deals related to Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics

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 the targeted therapy areas and indications for Huntingtin (Huntington Disease Protein or HTT)

Identify the use of drugs for target identification and drug repurposing

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 Huntingtin (Huntington Disease Protein or HTT) development landscape

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

Companies mentioned

AFFiRiS AG

Ionis Pharmaceuticals Inc

Neurimmune Holding AG

nLife Therapeutics SL

PTC Therapeutics Inc

reMYND NV

Shire Plc

UniQure NV

Voyager Therapeutics Inc

Vybion Inc

WAVE Life Sciences Ltd

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Huntingtin (Huntington Disease Protein or HTT) - Overview

Huntingtin (Huntington Disease Protein or HTT) - Therapeutics Development

Products under Development by Stage of Development

Products under Development by Therapy Area

Products under Development by Indication

Products under Development by Companies

Products under Development by Universities/Institutes

Huntingtin (Huntington Disease Protein or HTT) - Therapeutics Assessment

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Huntingtin (Huntington Disease Protein or HTT) - Companies Involved in Therapeutics Development

AFFiRiS AG

Ionis Pharmaceuticals Inc

Neurimmune Holding AG

nLife Therapeutics SL

PTC Therapeutics Inc

reMYND NV

Shire Plc

UniQure NV

Voyager Therapeutics Inc

Vybion Inc

WAVE Life Sciences Ltd

Huntingtin (Huntington Disease Protein or HTT) - Drug Profiles

AMT-130 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Antisense Oligonucleotide to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Antisense Oligonucleotides to Inhibit HTT for Huntington's Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Antisense Oligonucleotides to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Inhibit Huntingtin Protein for Huntington’s Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

INT-41 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

IONIS-HTTRx - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NI-302 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

NLF-HD XXXX - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

P-301905 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Small Molecules to Inhibit Huntingtin Protein for Huntington’s Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Synthetic Peptide to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Vaccine to Target Huntingtin for Huntington's Disease - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

VYHTT-01 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

WVE-120101 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

WVE-120102 - Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Huntingtin (Huntington Disease Protein or HTT) - Dormant Products

Huntingtin (Huntington Disease Protein or HTT) - Product Development Milestones

Featured News & Press Releases

Oct 18, 2017: uniQure Presents New Preclinical Data on AMT-130 in Huntington’s Disease at the ESGCT 25th Anniversary Congress in Berlin

Oct 06, 2017: UniQure Announces FDA Orphan Drug Designation for AMT-130 in Huntingtons disease

Jul 17, 2017: Wave Life Sciences Initiates Phase 1b/2a Clinical Trial: PRECISION-HD1 in Patients with Huntington's Disease

Jul 17, 2017: Wave Life Sciences Announces Initiation of Phase 1b/2a Clinical Trial: PRECISION-HD2 in Patients with Huntington's Disease

Jun 22, 2017: Enrollment in Phase 1/2a Study of IONIS-HTT Rx in Patients with Huntingtons Disease Completed and Open-Label Extension Study to Open in 2H 2017

Jun 01, 2017: Voyager Therapeutics Selects Lead Clinical Candidate for Huntington's Disease

Apr 26, 2017: uniQure Presents New Preclinical Data on AMT-130 in Huntington's Disease at CHDI's 12th Annual Huntington's Disease Therapeutics Conference

Apr 24, 2017: uniQure Announces Presentations at the Upcoming Annual Meeting of The American Society of Gene and Cell Therapy

Apr 04, 2017: uniQure Publishes Data Demonstrating Wide Distribution of AAV5 in the Central Nervous System

Jan 30, 2017: Vybion Huntington's Disease Drug Patent receives Track I Status

Jan 06, 2017: WAVE Life Sciences Provides Update on Lead Huntington’s Disease Program WVE-120101

Jan 06, 2017: WAVE Life Sciences Provides Update on Lead Huntington’s Disease Program WVE-120102

Aug 24, 2016: Novel Gene Therapy Drug for Huntington Disease

Jun 21, 2016: WAVE Life Sciences Receives Orphan Drug Designation from FDA for its Lead Candidate Designed to Treat Huntington’s Disease

Mar 22, 2016: uniQure Announces Preclinical Proof of Concept for Gene Therapy Approach in Huntington’s Disease

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development by Stage of Development, H2 2017

Number of Products under Development by Therapy Areas, H2 2017

Number of Products under Development by Indication, H2 2017

Number of Products under Development by Companies, H2 2017

Products under Development by Companies, H2 2017

Number of Products under Investigation by Universities/Institutes, H2 2017

Products under Investigation by Universities/Institutes, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Stage and Route of Administration, H2 2017

Number of Products by Stage and Molecule Type, H2 2017

Pipeline by AFFiRiS AG, H2 2017

Pipeline by Ionis Pharmaceuticals Inc, H2 2017

Pipeline by Neurimmune Holding AG, H2 2017

Pipeline by nLife Therapeutics SL, H2 2017

Pipeline by PTC Therapeutics Inc, H2 2017

Pipeline by reMYND NV, H2 2017

Pipeline by Shire Plc, H2 2017

Pipeline by UniQure NV, H2 2017

Pipeline by Voyager Therapeutics Inc, H2 2017

Pipeline by Vybion Inc, H2 2017

Pipeline by WAVE Life Sciences Ltd, H2 2017

Dormant Projects, H2 2017

List of Figures

List of Figures

Number of Products under Development by Stage of Development, H2 2017

Number of Products under Development by Therapy Areas, H2 2017

Number of Products under Development by Top 10 Indications, H2 2017

Number of Products by Mechanism of Actions, H2 2017

Number of Products by Stage and Mechanism of Actions, H2 2017

Number of Products by Routes of Administration, H2 2017

Number of Products by Stage and Routes of Administration, H2 2017

Number of Products by Molecule Types, H2 2017

Number of Products by Stage and Molecule Types, H2 2017

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