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According to the recently published report 'Huntingtin - Drugs In Development, 2022'; Huntingtin (Huntington Disease Protein or HTT) pipeline Target constitutes close to 30 molecules. Out of which approximately 27 molecules are developed by companies and remaining by the universities/institutes. Speak directly to the analyst to clarify any post sales queries you may have.
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 report 'Huntingtin - Drugs In Development, 2022' 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; that are being developed by Companies/Universities.
It also reviews key players involved in Huntingtin (Huntington Disease Protein or HTT) targeted therapeutics development with respective active and dormant or discontinued projects. Currently, The molecules developed by companies in Phase III, Phase II, Preclinical and Discovery stages are 1, 4, 12 and 10 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).
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
Table of Contents
- 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
- Alnylam Pharmaceuticals Inc
- Anima Biotech Inc
- Arvinas Inc
- Atalanta Therapeutics Inc
- Ceptur Therapeutics Inc
- Dystrogen Therapeutics SA
- Enzerna Biosciences LLC
- F. Hoffmann-La Roche Ltd
- Locanabio Inc
- Neurimmune Holding AG
- Novartis AG
- Ophidion Inc
- Origami Therapeutics Inc
- Passage Bio Inc
- Primary Peptides Inc
- PTC Therapeutics Inc
- reMYND NV
- ResQ Biotech
- Takeda Pharmaceutical Co Ltd
- UniQure NV
- Voyager Therapeutics Inc
- Vybion Inc
- Wave Life Sciences Ltd
- Huntingtin (Huntington Disease Protein or HTT) - Drug Profiles
- ALN-HTT - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- AMT-130 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Antisense Oligonucleotides to Inhibit HTT for Huntington's Disease - Drug Profile
- Product Description
- Mechanism Of Action
- Antisense RNAi Oligonucleotide to Inhibit HTT for for Huntington Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- branaplam hydrochloride - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- C-617 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- DYST-201 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- ENZ-001 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Gene Therapy to Inhibit HTT for Huntington Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Gene Therapy to Inhibit HTT for Huntington’s disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Gene Therapy to Inhibit Huntingtin for Huntington Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- HTT - Drug Profile
- Product Description
- Mechanism Of Action
- Huntington Disease - Drug Profile
- Product Description
- Mechanism Of Action
- Huntington's Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- INT-41 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- mHTT - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- NI-302 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- PP-005 - Drug Profile
- Product Description
- Mechanism Of Action
- PTC-518 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Recombinant Peptides to Inhibit Huntingtin for Huntington Disease - Drug Profile
- Product Description
- Mechanism Of Action
- ReS-18H - Drug Profile
- Product Description
- Mechanism Of Action
- Small Molecules to Inhibit HTT for Huntington Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Small Molecules to Inhibit HTT for Huntington’s Disease - Drug Profile
- Product Description
- Mechanism Of Action
- Small Molecules to Inhibit Huntingtin Protein for Huntington Disease - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Small Molecules to Target HTT for Huntington’s Disease - Drug Profile
- Product Description
- Mechanism Of Action
- Synthetic Peptide to Inhibit Huntingtin Protein for Huntington's Disease - Drug Profile
- Product Description
- Mechanism Of Action
- TAK-686 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- tominersen - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Vaccine to Target Huntingtin for Huntington's Disease - Drug Profile
- Product Description
- Mechanism Of Action
- WVE-003 - Drug Profile
- Product Description
- Mechanism Of Action
- History of Events
- Huntingtin (Huntington Disease Protein or HTT) - Dormant Products
- Huntingtin (Huntington Disease Protein or HTT) - Discontinued Products
- Huntingtin (Huntington Disease Protein or HTT) - Product Development Milestones
- Featured News & Press Releases
- Jun 23, 2022: uniQure announces update on low-dose cohort in phase I/II clinical trial of AMT-130 gene therapy for the treatment of Huntington’s disease
- Mar 30, 2022: PTC Therapeutics announces initiation of PIVOT-HD phase 2 clinical trial to evaluate PTC518 in patients with Huntington's Disease
- Mar 21, 2022: uniQure announces completion of patient enrollment in the first two cohorts of its phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease
- Feb 07, 2022: uniQure announces dosing of first patients in European open-label clinical trial of AMT-130 gene therapy in Huntington’s disease
- Jan 18, 2022: Ionis' partner to evaluate tominersen for Huntington's disease in new Phase 2 trial
- Dec 16, 2021: uniQure announces clinical update on first patients in phase I/II clinical trial of AMT-130 gene therapy for the treatment of Huntington’s Disease
- Dec 16, 2021: Novartis receives FDA Fast Track designation for branaplam (LMI070) for the treatment of Huntington’s Disease
- Nov 02, 2021: uniQure announces latest positive recommendation from data safety monitoring board in phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease
- Sep 20, 2021: PTC Therapeutics to host conference call to discuss results of PTC518 phase 1 study for Huntington's disease program
- Sep 09, 2021: Wave Life Sciences announces initiation of dosing in phase 1b/2a SELECT-HD clinical trial of WVE-003 in Huntington’s disease
- Aug 30, 2021: uniQure announces completion of additional patient procedures following positive recommendation from data safety monitoring board in phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease
- Jul 23, 2021: Novartis stops development of branaplam for SMA
- Jun 16, 2021: uniQure announces enrollment of first two patients in second cohort of phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease
- May 27, 2021: uniQure announces positive recommendation to advance phase I/II Clinical Trial of AMT-130 for the treatment of Huntington’s Disease
- Apr 27, 2021: Positive preclinical in vivo results with AFFiRiS’ antibody mAB C6-17 to treat Huntington’s disease to be presented at the 16th Annual Huntington's Disease Therapeutics Conference
- Appendix
- Methodology
- Coverage
- Secondary Research
- Primary Research
- Expert Panel Validation
- Contact the Publisher
- Disclaimer
List of Tables
- Number of Products under Development by Stage of Development, 2022
- Number of Products under Development by Therapy Areas, 2022
- Number of Products under Development by Indication, 2022
- Number of Products under Development by Companies, 2022
- Products under Development by Companies, 2022
- Number of Products under Investigation by Universities/Institutes, 2022
- Products under Investigation by Universities/Institutes, 2022
- Number of Products by Stage and Mechanism of Actions, 2022
- Number of Products by Stage and Route of Administration, 2022
- Number of Products by Stage and Molecule Type, 2022
- Pipeline by AFFiRiS AG, 2022
- Pipeline by Alnylam Pharmaceuticals Inc, 2022
- Pipeline by Anima Biotech Inc, 2022
- Pipeline by Arvinas Inc, 2022
- Pipeline by Atalanta Therapeutics Inc, 2022
- Pipeline by Ceptur Therapeutics Inc, 2022
- Pipeline by Dystrogen Therapeutics SA, 2022
- Pipeline by Enzerna Biosciences LLC, 2022
- Pipeline by F. Hoffmann-La Roche Ltd, 2022
- Pipeline by Locanabio Inc, 2022
- Pipeline by Neurimmune Holding AG, 2022
- Pipeline by Novartis AG, 2022
- Pipeline by Ophidion Inc, 2022
- Pipeline by Origami Therapeutics Inc, 2022
- Pipeline by Passage Bio Inc, 2022
- Pipeline by Primary Peptides Inc, 2022
- Pipeline by PTC Therapeutics Inc, 2022
- Pipeline by reMYND NV, 2022
- Pipeline by ResQ Biotech, 2022
- Pipeline by Takeda Pharmaceutical Co Ltd, 2022
- Pipeline by UniQure NV, 2022
- Pipeline by Voyager Therapeutics Inc, 2022
- Pipeline by Vybion Inc, 2022
- Pipeline by Wave Life Sciences Ltd, 2022
- Dormant Projects, 2022
- Discontinued Products, 2022
List of Figures
- Number of Products under Development by Stage of Development, 2022
- Number of Products under Development by Therapy Areas, 2022
- Number of Products under Development by Indications, 2022
- Number of Products by Stage and Mechanism of Actions, 2022
- Number of Products by Routes of Administration, 2022
- Number of Products by Stage and Routes of Administration, 2022
- Number of Products by Molecule Types, 2022
- Number of Products by Stage and Molecule Types, 2022
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- AFFiRiS AG
- Alnylam Pharmaceuticals Inc
- Anima Biotech Inc
- Arvinas Inc
- Atalanta Therapeutics Inc
- Ceptur Therapeutics Inc
- Dystrogen Therapeutics SA
- Enzerna Biosciences LLC
- F. Hoffmann-La Roche Ltd
- Locanabio Inc
- Neurimmune Holding AG
- Novartis AG
- Ophidion Inc
- Origami Therapeutics Inc
- Passage Bio Inc
- Primary Peptides Inc
- PTC Therapeutics Inc
- reMYND NV
- ResQ Biotech
- Takeda Pharmaceutical Co Ltd
- UniQure NV
- Voyager Therapeutics Inc
- Vybion Inc
- Wave Life Sciences Ltd
