Huntingtin (Huntington Disease Protein or HTT) - Pipeline Review, H1 2018
Huntingtin (Huntington Disease Protein or HTT) - Pipeline Review, H1 2018
SUMMARY
Huntingtin (Huntington Disease Protein or HTT) pipeline Target constitutes close to 15 molecules. Out of which approximately 13 molecules are developed by companies and remaining by the universities/institutes.
The latest report Huntingtin - Pipeline Review, H1 2018, 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.
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. The molecules developed by companies in Phase II, Preclinical and Discovery stages are 3, 7 and 3 respectively.
Similarly, the universities portfolio in Preclinical and Discovery stages comprises 1 and 1 molecules, respectively. Report covers products from therapy areas Central Nervous System and Genetic Disorders which include indications Huntington Disease, Alzheimer's Disease, Kennedy’s Disease (Spinal and Bulbar Muscular Atrophy) and Spinocerebellar Ataxia (SCA).
Furthermore, this report also reviews key players involved in Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics development with respective active and dormant or discontinued projects. Driven by 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) pipeline Target constitutes close to 15 molecules. Out of which approximately 13 molecules are developed by companies and remaining by the universities/institutes.
The latest report Huntingtin - Pipeline Review, H1 2018, 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.
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. The molecules developed by companies in Phase II, Preclinical and Discovery stages are 3, 7 and 3 respectively.
Similarly, the universities portfolio in Preclinical and Discovery stages comprises 1 and 1 molecules, respectively. Report covers products from therapy areas Central Nervous System and Genetic Disorders which include indications Huntington Disease, Alzheimer's Disease, Kennedy’s Disease (Spinal and Bulbar Muscular Atrophy) and Spinocerebellar Ataxia (SCA).
Furthermore, this report also reviews key players involved in Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics development with respective active and dormant or discontinued projects. Driven by 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
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
F. Hoffmann-La Roche Ltd
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
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
Dec 13, 2017: New drug could halt progression of Huntingtons disease
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 Huntington’s 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
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
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
F. Hoffmann-La Roche Ltd
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
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
Dec 13, 2017: New drug could halt progression of Huntingtons disease
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 Huntington’s 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
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, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indication, H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Number of Products under Investigation by Universities/Institutes, H1 2018
Products under Investigation by Universities/Institutes, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Pipeline by AFFiRiS AG, H1 2018
Pipeline by F. Hoffmann-La Roche Ltd, H1 2018
Pipeline by Neurimmune Holding AG, H1 2018
Pipeline by nLife Therapeutics SL, H1 2018
Pipeline by PTC Therapeutics Inc, H1 2018
Pipeline by reMYND NV, H1 2018
Pipeline by Shire Plc, H1 2018
Pipeline by UniQure NV, H1 2018
Pipeline by Voyager Therapeutics Inc, H1 2018
Pipeline by Vybion Inc, H1 2018
Pipeline by WAVE Life Sciences Ltd, H1 2018
Dormant Projects, H1 2018
Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indication, H1 2018
Number of Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018
Number of Products under Investigation by Universities/Institutes, H1 2018
Products under Investigation by Universities/Institutes, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Pipeline by AFFiRiS AG, H1 2018
Pipeline by F. Hoffmann-La Roche Ltd, H1 2018
Pipeline by Neurimmune Holding AG, H1 2018
Pipeline by nLife Therapeutics SL, H1 2018
Pipeline by PTC Therapeutics Inc, H1 2018
Pipeline by reMYND NV, H1 2018
Pipeline by Shire Plc, H1 2018
Pipeline by UniQure NV, H1 2018
Pipeline by Voyager Therapeutics Inc, H1 2018
Pipeline by Vybion Inc, H1 2018
Pipeline by WAVE Life Sciences Ltd, H1 2018
Dormant Projects, H1 2018
LIST OF FIGURES
Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indications, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
COMPANIES MENTIONED
AFFiRiS AG
F. Hoffmann-La Roche Ltd
Neurimmune Holding AG
nLife Therapeutics SL
PTC Therapeutics Inc
reMYND NV
Shire Plc
UniQure NV
Voyager Therapeutics Inc
Vybion Inc
WAVE Life Sciences Ltd
Number of Products under Development by Stage of Development, H1 2018
Number of Products under Development by Therapy Areas, H1 2018
Number of Products under Development by Indications, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018
COMPANIES MENTIONED
AFFiRiS AG
F. Hoffmann-La Roche Ltd
Neurimmune Holding AG
nLife Therapeutics SL
PTC Therapeutics Inc
reMYND NV
Shire Plc
UniQure NV
Voyager Therapeutics Inc
Vybion Inc
WAVE Life Sciences Ltd
