Huntingtin - Pipeline Review, H2 2020
Huntingtin - Pipeline Review, H2 2020
SUMMARY
Huntingtin (Huntington Disease Protein or HTT) - Huntingtin protein is an encoded by the huntingtin gene, also called the HTT or HD (Huntington disease) gene. Huntingtin up regulates the expression of brain derived neurotrophic factor (BDNF) at the transcription level. Huntingtin is primarily associated with vesicles and microtubules. These indicate an important role in cytoskeletal anchoring or transport of mitochondria. The Htt protein is involved in vesicle trafficking as it interacts with HIP1 to mediate endocytosis. Huntingtin also plays an important role in the establishment in epithelial polarity through its interaction with RAB11A.
Huntingtin (Huntington Disease Protein or HTT) pipeline Target constitutes close to 23 molecules. Out of which approximately 20 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Phase III, Phase II, Preclinical and Discovery stages are 1, 3, 11 and 5 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively. Report covers products from therapy areas Central Nervous System and Genetic Disorders which include indications Huntington Disease, Spinocerebellar Ataxia (SCA), Alzheimer's Disease, Kennedy’s Disease (Spinal and Bulbar Muscular Atrophy) and Spinal Muscular Atrophy (SMA).
The latest report Huntingtin - Pipeline Review, H2 2020, outlays 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. It also reviews key players involved in Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics development with respective active and dormant or discontinued projects.
The report is built using data and information sourced from 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.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
SCOPE
SUMMARY
Huntingtin (Huntington Disease Protein or HTT) - Huntingtin protein is an encoded by the huntingtin gene, also called the HTT or HD (Huntington disease) gene. Huntingtin up regulates the expression of brain derived neurotrophic factor (BDNF) at the transcription level. Huntingtin is primarily associated with vesicles and microtubules. These indicate an important role in cytoskeletal anchoring or transport of mitochondria. The Htt protein is involved in vesicle trafficking as it interacts with HIP1 to mediate endocytosis. Huntingtin also plays an important role in the establishment in epithelial polarity through its interaction with RAB11A.
Huntingtin (Huntington Disease Protein or HTT) pipeline Target constitutes close to 23 molecules. Out of which approximately 20 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Phase III, Phase II, Preclinical and Discovery stages are 1, 3, 11 and 5 respectively. Similarly, the universities portfolio in Preclinical and Discovery stages comprises 2 and 1 molecules, respectively. Report covers products from therapy areas Central Nervous System and Genetic Disorders which include indications Huntington Disease, Spinocerebellar Ataxia (SCA), Alzheimer's Disease, Kennedy’s Disease (Spinal and Bulbar Muscular Atrophy) and Spinal Muscular Atrophy (SMA).
The latest report Huntingtin - Pipeline Review, H2 2020, outlays 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. It also reviews key players involved in Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics development with respective active and dormant or discontinued projects.
The report is built using data and information sourced from 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.
Note: Certain content/sections in the pipeline guide may be removed or altered based on the availability and relevance of data.
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
- 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
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
Enzerna 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 designation 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 Nature 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
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
Enzerna 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 designation 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 Nature 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 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
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
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
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
