Huntingtin (Huntington Disease Protein or HTT) Drugs in Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players, 2022 Update

Pages: 85 Published: June 30, 2022 Report Code: GDGMDHC22115TDB

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.

* Global Markets Direct, is a research brand owned and operated by GlobalData.

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

Key Players

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

Table of Contents

Table of Contents

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

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

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

List of Figures

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

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