Huntingtin – Pipeline Review, H2 2020

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Huntingtin – Pipeline Review, H2 2020


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 23 molecules. Out of which approximately 20 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 5 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, H2 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.


– 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

Companies Mentioned:
Anima Biotech Inc
Dystrogen Therapeutics SA
Enzerna Biosciences LLC
Exicure Inc
F. Hoffmann-La Roche Ltd
Neurimmune Holding AG
Ophidion Inc
Origami Therapeutics Inc
PTC Therapeutics Inc
Takeda Pharmaceutical Co Ltd
UniQure NV
Voyager Therapeutics Inc
Vybion Inc
Wave Life Sciences Ltd

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