Huntingtin (Huntington Disease Protein or HTT) – Pipeline Review, H2 2016

Global Markets Direct’s, ‘Huntingtin (Huntington Disease Protein or HTT) – Pipeline Review, H2 2016’, provides in depth analysis on Huntingtin (Huntington Disease Protein or HTT) targeted pipeline therapeutics.

The report provides comprehensive information on the Huntingtin (Huntington Disease Protein or HTT), targeted therapeutics, 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.

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, S.L.

reMYND NV

Shire Plc

UniQure N.V.

Voyager Therapeutics, Inc.

Vybion, Inc.

WAVE Life Sciences Ltd.

Table of Contents

Table of Contents

Table of Contents 2

List of Tables 5

List of Figures 5

Introduction 6

Global Markets Direct Report Coverage 6

Huntingtin (Huntington Disease Protein or HTT) Overview 7

Therapeutics Development 8

Huntingtin (Huntington Disease Protein or HTT) - Products under Development by Stage of Development 8

Huntingtin (Huntington Disease Protein or HTT) - Products under Development by Therapy Area 9

Huntingtin (Huntington Disease Protein or HTT) - Products under Development by Indication 10

Huntingtin (Huntington Disease Protein or HTT) - Pipeline Products Glance 11

Late Stage Products 11

Early Stage Products 12

Huntingtin (Huntington Disease Protein or HTT) - Products under Development by Companies 13

Huntingtin (Huntington Disease Protein or HTT) - Therapeutics Assessment 15

Assessment by Monotherapy/Combination Products 15

Assessment by Mechanism of Action 16

Assessment by Route of Administration 17

Assessment by Molecule Type 19

Huntingtin (Huntington Disease Protein or HTT) - Companies Involved in Therapeutics Development 21

AFFiRiS AG 21

Ionis Pharmaceuticals, Inc. 22

Neurimmune Holding AG 23

nLife Therapeutics, S.L. 24

reMYND NV 25

Shire Plc 26

UniQure N.V. 27

Voyager Therapeutics, Inc. 28

Vybion, Inc. 29

WAVE Life Sciences Ltd. 30

Huntingtin (Huntington Disease Protein or HTT) - Drug Profiles 31

AMT-130 - Drug Profile 31

Product Description 31

Mechanism Of Action 31

R&D Progress 31

Antisense Oligonucleotide 2 to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile 32

Product Description 32

Mechanism Of Action 32

R&D Progress 32

Antisense Oligonucleotide to Inhibit HTT for Huntington's Disease - Drug Profile 33

Product Description 33

Mechanism Of Action 33

R&D Progress 33

Antisense Oligonucleotide to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile 34

Product Description 34

Mechanism Of Action 34

R&D Progress 34

Antisense Oligonucleotides to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile 35

Product Description 35

Mechanism Of Action 35

R&D Progress 35

Gene Therapy to Inhibit Huntingtin Protein for Huntington’s Disease - Drug Profile 36

Product Description 36

Mechanism Of Action 36

R&D Progress 36

INT-41 - Drug Profile 38

Product Description 38

Mechanism Of Action 38

R&D Progress 38

IONIS-HTTRx - Drug Profile 39

Product Description 39

Mechanism Of Action 39

R&D Progress 39

NI-302 - Drug Profile 41

Product Description 41

Mechanism Of Action 41

R&D Progress 41

Small Molecules to Inhibit Huntingtin Protein for Huntington’s Disease - Drug Profile 42

Product Description 42

Mechanism Of Action 42

R&D Progress 42

Vaccine to Target Huntingtin for Huntington's Disease - Drug Profile 43

Product Description 43

Mechanism Of Action 43

R&D Progress 43

VYHTT-01 - Drug Profile 44

Product Description 44

Mechanism Of Action 44

R&D Progress 44

WVE-120101 - Drug Profile 45

Product Description 45

Mechanism Of Action 45

R&D Progress 45

Huntingtin (Huntington Disease Protein or HTT) - Dormant Projects 46

Huntingtin (Huntington Disease Protein or HTT) - Featured News & Press Releases 47

Aug 24, 2016: Novel Gene Therapy Drug for Huntington Disease 47

Jun 21, 2016: WAVE Life Sciences Receives Orphan Drug Designation from FDA for its Lead Candidate Designed to Treat Huntington’s Disease 47

Mar 22, 2016: uniQure Announces Preclinical Proof of Concept for Gene Therapy Approach in Huntington’s Disease 48

Jan 05, 2016: Ionis Pharmaceuticals Receives Orphan Drug Designation from the US FDA for IONIS-HTT Rx for the Treatment of Patients with Huntington Disease 48

Jul 21, 2015: Isis Pharmaceuticals Initiates Clinical Study of ISIS-HTT Rx in Patients With Huntington's Disease 49

Nov 19, 2014: Sangamo BioSciences Presents Study At Society For Neuroscience Meeting Demonstrating In Vivo Reversal Of Huntington's Disease Signs And Symptoms 50

Nov 12, 2013: Sangamo BioSciences Announces Presentation Of First Demonstration Of In Vivo Efficacy Of Novel ZFP Therapeutic For Huntington's Disease 51

Sep 03, 2013: Vybion’s Huntington Drug Neutralizes Critical Disease Driver 52

Oct 17, 2012: Sangamo BioSciences Announces First Presentation Of Data From ZFP Therapeutic For Huntington's Disease At 2012 Annual Meeting Of Society For Neuroscience 53

Appendix 55

Methodology 55

Coverage 55

Secondary Research 55

Primary Research 55

Expert Panel Validation 55

Contact Us 55

Disclaimer 56

List of Tables

List of Tables

Number of Products under Development for, H2 2016 8

Number of Products under Development by Therapy Area, H2 2016 9

Number of Products under Development by Indication, H2 2016 10

Comparative Analysis by Late Stage Development, H2 2016 11

Comparative Analysis by Early Stage Products, H2 2016 12

Number of Products under Development by Companies, H2 2016 13

Products under Development by Companies, H2 2016 14

Assessment by Monotherapy/Combination Products, H2 2016 15

Number of Products by Stage and Mechanism of Action, H2 2016 16

Number of Products by Stage and Route of Administration, H2 2016 18

Number of Products by Stage and Molecule Type, H2 2016 20

Pipeline by AFFiRiS AG, H2 2016 21

Pipeline by Ionis Pharmaceuticals, Inc., H2 2016 22

Pipeline by Neurimmune Holding AG, H2 2016 23

Pipeline by nLife Therapeutics, S.L., H2 2016 24

Pipeline by reMYND NV, H2 2016 25

Pipeline by Shire Plc, H2 2016 26

Pipeline by UniQure N.V., H2 2016 27

Pipeline by Voyager Therapeutics, Inc., H2 2016 28

Pipeline by Vybion, Inc., H2 2016 29

Pipeline by WAVE Life Sciences Ltd., H2 2016 30

Dormant Projects, H2 2016 46

List of Figures

List of Figures

Number of Products under Development for, H2 2016 8

Number of Products under Development by Therapy Area, H2 2016 9

Number of Products under Development by Indication, H2 2016 10

Comparative Analysis by Early Stage Products, H2 2016 12

Assessment by Monotherapy/Combination Products, H2 2016 15

Number of Products by Stage and Mechanism of Actions, H2 2016 16

Number of Products by Routes of Administration, H2 2016 17

Number of Products by Stage and Routes of Administration, H2 2016 18

Number of Products by Molecule Types, H2 2016 19

Number of Products by Stage and Molecule Type, H2 2016 20

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