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Huntingtin - Pipeline Review, H1 2020

  • ID: 5017482
  • Drug Pipelines
  • March 2020
  • Region: Global
  • 87 pages
  • Global Markets Direct
1h Free Analyst Time

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FEATURED COMPANIES

  • AFFiRiS AG
  • Enzerna Biosciences LLC
  • F. Hoffmann-La Roche Ltd
  • Ophidion Inc
  • reMYND NV
  • Voyager Therapeutics Inc
  • MORE
Huntingtin - Pipeline Review, H1 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 20 molecules. Out of which approximately 17 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 2 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, H1 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
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
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • AFFiRiS AG
  • Enzerna Biosciences LLC
  • F. Hoffmann-La Roche Ltd
  • Ophidion Inc
  • reMYND NV
  • Voyager Therapeutics Inc
  • MORE
  • Introduction
  • 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
  • Dystrogen Therapeutics SA
  • Enzerna Biosciences LLC
  • Exicure Inc
  • F. Hoffmann-La Roche Ltd
  • Neurimmune Holding AG
  • Ophidion 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
  • Huntingtin (Huntington Disease Protein or HTT) - Dormant Products
  • Huntingtin (Huntington Disease Protein or HTT) - Product Development Milestones
  • Featured News & Press Releases
  • 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
  • Apr 29, 2019: Genentech to present on its Huntington’s Disease drug candidate RG-6042 at the 2019 AAN Annual Meeting Showcase Breadth and Promise of Neuroscience Portfolio
  • Apr 15, 2019: uniQure to present data on Gene Therapy for Huntington's Disease at ASGCT 2019
  • Apr 10, 2019: Wave Life Sciences provides timing update on PRECISION-HD clinical programs
  • Apr 10, 2019: Wave Life Sciences provides timing update on WVE-120102 Clinical Program
  • 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 2020
  • Number of Products under Development by Therapy Areas, H1 2020
  • Number of Products under Development by Indication, H1 2020
  • Number of Products under Development by Companies, H1 2020
  • Products under Development by Companies, H1 2020
  • Products under Development by Companies, H1 2020 (Contd..1), H1 2020
  • Number of Products under Investigation by Universities/Institutes, H1 2020
  • Products under Investigation by Universities/Institutes, H1 2020
  • Number of Products by Stage and Mechanism of Actions, H1 2020
  • Number of Products by Stage and Route of Administration, H1 2020
  • Number of Products by Stage and Molecule Type, H1 2020
  • Pipeline by AFFiRiS AG, H1 2020
  • Pipeline by Dystrogen Therapeutics SA, H1 2020
  • Pipeline by Enzerna Biosciences LLC, H1 2020
  • Pipeline by Exicure Inc, H1 2020
  • Pipeline by F. Hoffmann-La Roche Ltd, H1 2020
  • Pipeline by Neurimmune Holding AG, H1 2020
  • Pipeline by Ophidion Inc, H1 2020
  • Pipeline by PTC Therapeutics Inc, H1 2020
  • Pipeline by reMYND NV, H1 2020
  • Pipeline by Takeda Pharmaceutical Co Ltd, H1 2020
  • Pipeline by UniQure NV, H1 2020
  • Pipeline by Voyager Therapeutics Inc, H1 2020
  • Pipeline by Vybion Inc, H1 2020
  • Pipeline by Wave Life Sciences Ltd, H1 2020
  • Dormant Projects, H1 2020
List of Figures
  • Number of Products under Development by Stage of Development, H1 2020
  • Number of Products under Development by Therapy Areas, H1 2020
  • Number of Products under Development by Top 10 Indications, H1 2020
  • Number of Products by Mechanism of Actions, H1 2020
  • Number of Products by Stage and Mechanism of Actions, H1 2020
  • Number of Products by Routes of Administration, H1 2020
  • Number of Products by Stage and Routes of Administration, H1 2020
  • Number of Products by Molecule Types, H1 2020
  • Number of Products by Stage and Molecule Types, H1 2020
Note: Product cover images may vary from those shown
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  • AFFiRiS AG
  • Dystrogen Therapeutics SA
  • Enzerna Biosciences LLC
  • Exicure Inc
  • F. Hoffmann-La Roche Ltd
  • Neurimmune Holding AG
  • Ophidion Inc
  • PTC Therapeutics Inc
  • reMYND NV
  • Takeda Pharmaceutical Co Ltd
  • UniQure NV
  • Voyager Therapeutics Inc
  • Vybion Inc
  • Wave Life Sciences Ltd
Note: Product cover images may vary from those shown
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