Table of Content



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
Anima Biotech Inc
Dystrogen Therapeutics SA
Enzer Biosciences LLC
Exicure Inc
F. Hoffmann-La Roche Ltd
Neurimmune Holding AG
Ophidion Inc
Origami Therapeutics Inc
PTC Therapeutics Inc
reMYND NV
Takeda Pharmaceutical Co Ltd
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 Oligonucleotides to Inhibit HTT for Huntington’s Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
C-617 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
DYST-201 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
ENZ-001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Inhibit HTT for Huntington’s disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Inhibit Huntingtin for Huntington Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
INT-41 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NI-302 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Oligonucleotide to Inhibit HTT for Huntington Disease - 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 HTT for Huntington’s Disease - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit Huntingtin Protein for Huntington Disease - 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
Small Molecules to Target HTT 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
TAK-686 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tominersen - 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-003 - 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
Sep 25, 2020: uniQure announces recommendations from data safety monitoring board of phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease
Jun 19, 2020: uniQure announces first two patients treated in phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease
Jun 03, 2020: Neurobiology of Disease publishes encouraging preclinical results of AFFiRiS’ antibody mAB C6-17 to treat Huntington’s disease
Apr 20, 2020: Ionis and partner announce enrollment completion of global Phase 3 GENERATION HD1 study for Huntington’s disease
Feb 27, 2020: Ionis and partner highlight tominersen (formerly known as IONIS-HTTRx and RG6042) data at annual Huntington’s disease drug discovery conference
Feb 27, 2020: uniQure presents multiple new preclinical data on AMT-130 at the CHDI’s 15th Annual Huntington’s Disease Therapeutics Conference
Feb 18, 2020: Wave Life Sciences to present preclinical in vivo and in vitro data for SNP3-targeting Huntington’s Disease program at CHDI Foundation’s 15th Annual HD Therapeutics Conference
Feb 17, 2020: Chugai receives orphan drug desigtion for RG6042 in Huntington’s disease from the MHLW
Dec 30, 2019: Wave Life Sciences announces topline data and addition of higher dose cohort in ongoing Phase 1b/2a PRECISION-HD2 trial in Huntington’s Disease
Dec 19, 2019: uniQure announces publication of preclinical data for AMT-130 in Huntington’s disease supporting non-selective HTT-lowering approach
Sep 27, 2019: uniQure to present update on of AMT-130 at European Huntington Association 2019 Conference
Jul 01, 2019: Sangamo Therapeutics announces ture Medicine publication detailing the activity of disease allele-selective zinc finger proteins in preclinical models of huntington’s disease
May 22, 2019: uniQure strengthens intellectual property portfolio with granted patent claims covering AMT-130 for huntington’s disease
May 07, 2019: uniQure announces featured presentations of new data on AMT-130 at the 2019 American Academy of Neurology Annual Meeting
May 06, 2019: Huntington drug successfully lowers levels of disease-causing protein
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures



Number of Products under Development by Stage of Development, H2 2020
Number of Products under Development by Therapy Areas, H2 2020
Number of Products under Development by Top 10 Indications, H2 2020
Number of Products by Mechanism of Actions, H2 2020
Number of Products by Stage and Mechanism of Actions, H2 2020
Number of Products by Routes of Administration, H2 2020
Number of Products by Stage and Routes of Administration, H2 2020
Number of Products by Top 10 Molecule Types, H2 2020
Number of Products by Stage and Top 10 Molecule Types, H2 2020


List of Tables



Number of Products under Development by Stage of Development, H2 2020
Number of Products under Development by Therapy Areas, H2 2020
Number of Products under Development by Indication, H2 2020
Number of Products under Development by Companies, H2 2020
Products under Development by Companies, H2 2020
Products under Development by Companies, H2 2020 (Contd..1), H2 2020
Number of Products under Investigation by Universities/Institutes, H2 2020
Products under Investigation by Universities/Institutes, H2 2020
Number of Products by Stage and Mechanism of Actions, H2 2020
Number of Products by Stage and Route of Administration, H2 2020
Number of Products by Stage and Molecule Type, H2 2020
Pipeline by AFFiRiS AG, H2 2020
Pipeline by Anima Biotech Inc, H2 2020
Pipeline by Dystrogen Therapeutics SA, H2 2020
Pipeline by Enzerna Biosciences LLC, H2 2020
Pipeline by Exicure Inc, H2 2020
Pipeline by F. Hoffmann-La Roche Ltd, H2 2020
Pipeline by Neurimmune Holding AG, H2 2020
Pipeline by Ophidion Inc, H2 2020
Pipeline by Origami Therapeutics Inc, H2 2020
Pipeline by PTC Therapeutics Inc, H2 2020
Pipeline by reMYND NV, H2 2020
Pipeline by Takeda Pharmaceutical Co Ltd, H2 2020
Pipeline by UniQure NV, H2 2020
Pipeline by Voyager Therapeutics Inc, H2 2020
Pipeline by Vybion Inc, H2 2020
Pipeline by Wave Life Sciences Ltd, H2 2020
Dormant Projects, H2 2020