Huntingtin (Huntington Disease Protein or HTT) Drugs in Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update
Huntingtin (Huntington Disease Protein or HTT) Drugs in Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update
SUMMARY
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.
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
SUMMARY
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.
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
- 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
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 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
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 Us
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
Products under Development by Companies, 2022 (Contd..1)
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
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
Products under Development by Companies, 2022 (Contd..1)
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
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
