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Transthyretin (ATTR or Prealbumin or TBPA or TTR) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

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    Report

  • 85 Pages
  • September 2022
  • Region: Global
  • Global Markets Direct
  • ID: 5670533
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Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Transthyretin is a transport protein. It transports thyroid hormones in the plasma and cerebrospinal fluid, and also transports retinol (vitamin A) in the plasma. The diseases caused by mutations include amyloidotic polyneuropathy, euthyroid hyperthyroxinaemia, amyloidotic vitreous opacities, cardiomyopathy, oculoleptomeningeal amyloidosis, meningocerebrovascular amyloidosis and carpal tunnel syndrome.

Transthyretin (ATTR or Prealbumin or TBPA or TTR) pipeline Target constitutes close to 17 molecules. Out of which approximately 16 molecules are developed by companies and remaining by the universities/institutes. The molecules developed by companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical and Discovery stages are 1, 3, 3, 4, 3 and 2 respectively. Similarly, the universities portfolio in Phase III stages comprises 1 molecules, respectively. Report covers products from therapy areas Metabolic Disorders, Ophthalmology, Central Nervous System, Gastrointestinal, Immunology and Infectious Disease which include indications Familial Amyloid Neuropathies, Familial Amyloid Cardiomyopathy, Amyloid Cardiomyopathy, Amyloidosis, Coronavirus Disease 2019 (COVID-19) Associated Acute Respiratory Distress Syndrome, Crohn's Disease (Regional Enteritis), Glaucoma, Juvenile Macular Degeneration (Stargardt Disease), Keratoconjunctivitis Sicca (Dry Eye), Multiple Sclerosis, Rheumatoid Arthritis, Ulcerative Colitis and Unspecified Ophthalmological Disorders.

The latest report Transthyretin - Drugs In Development, 2022, outlays comprehensive information on the Transthyretin (ATTR or Prealbumin or TBPA or TTR) 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 Transthyretin (ATTR or Prealbumin or TBPA or TTR) 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 Transthyretin (ATTR or Prealbumin or TBPA or TTR)
  • The report reviews Transthyretin (ATTR or Prealbumin or TBPA or TTR) 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 Transthyretin (ATTR or Prealbumin or TBPA or TTR) targeted therapeutics and enlists all their major and minor projects
  • The report assesses Transthyretin (ATTR or Prealbumin or TBPA or TTR) 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 Transthyretin (ATTR or Prealbumin or TBPA or TTR) 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 Transthyretin (ATTR or Prealbumin or TBPA or TTR)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 Transthyretin (ATTR or Prealbumin or TBPA or TTR) 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
  • Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Overview
  • Transthyretin (ATTR or Prealbumin or TBPA or TTR) - 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
  • Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Therapeutics Assessment
  • Assessment by Mechanism of Action
  • Assessment by Route of Administration
  • Assessment by Molecule Type
  • Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Companies Involved in Therapeutics Development
  • ADRx Inc
  • Akcea Therapeutics Inc
  • Alexion Pharmaceuticals Inc
  • Alnylam Pharmaceuticals Inc
  • Autotac Bio Inc
  • BridgeBio Pharma Inc
  • BSIM Therapeutics SA
  • Corino Therapeutics Inc
  • Covalent Bioscience Inc
  • Galmed Pharmaceuticals Ltd
  • Novo Nordisk AS
  • Proclara Biosciences Inc
  • Regeneron Pharmaceuticals Inc
  • Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Drug Profiles
  • acoramidis hydrochloride - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • ALNTTRSC-04 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • Amilo-5MER - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • Antibody to Inhibit TTR for Familial Transthyretin Amyloidosis - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • ATC-202 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • doxycycline hyclate - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • eplontersen sodium - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • inotersen sodium - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • Monoclonal Antibody for Familial Amyloid Cardiomyopathy - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • NI-006 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • NPT-189 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • NTLA-2001 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • patisiran - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • PRX-004 - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • Small Molecules to Inhibit Transthyretin for Ophthalmology - Drug Profile
  • Product Description
  • Mechanism Of Action
  • tafamidis meglumine - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • tolcapone - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • vutrisiran - Drug Profile
  • Product Description
  • Mechanism Of Action
  • History of Events
  • Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Dormant Products
  • Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Discontinued Products
  • Transthyretin (ATTR or Prealbumin or TBPA or TTR) - Product Development Milestones
  • Featured News & Press Releases
  • Aug 18, 2022: Alnylam to present data from the APOLLO-B phase 3 study of patisiran in patients with ATTR amyloidosis with cardiomyopathy at the 18th International Symposium on amyloidosis
  • Aug 03, 2022: Alnylam reports positive topline results from APOLLO-B phase 3 study of patisiran in patients with ATTR amyloidosis with cardiomyopathy
  • Jul 22, 2022: Alnylam receives positive CHMP opinion for vutrisiran for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy
  • Jul 20, 2022: Orsini Specialty Pharmacy selected as limited distribution partner for AMVUTTRA (vutrisiran)
  • Jun 24, 2022: Intellia and Regeneron present updated interim data from phase 1 Study of CRISPR-based NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis demonstrating that deep serum TTR reductions remained durable after a single dose
  • Jun 22, 2022: AstraZeneca-Ionis’ eplontersen meets endpoints in Phase III ATTRv-PN trial
  • Jun 13, 2022: Alnylam announces FDA approval of AMVUTTRA (vutrisiran), an RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults
  • Jun 08, 2022: Intellia Therapeutics to present updated interim clinical data from ongoing phase 1 study of NTLA-2001 for the treatment of transthyretin (ATTR) amyloidosis at the International Liver Congress 2022
  • May 23, 2022: Alnylam presents new 18-month results from exploratory cardiac endpoints in HELIOS-A phase 3 study of investigational vutrisiran
  • Apr 04, 2022: Alnylam announces 3-Month extension of review period for New Drug Application for Vutrisiran
  • Apr 03, 2022: BridgeBio Pharma presents updated rsults from phase 2 open-label extension study of Acoramidis in transthyretin amyloid cardiomyopathy (ATTR-CM)
  • Mar 28, 2022: BridgeBio Pharma to present updated results from phase 2 open-label extension study of Acoramidis in Transthyretin Amyloid Cardiomyopathy (ATTR-CM) at the American College of Cardiology (ACC) Annual Scientific Session & Expo
  • Mar 01, 2022: Intellia, Regeneron report positive Phase I transthyretin amyloidosis data
  • Feb 17, 2022: Intellia Therapeutics announces two upcoming investor events in February 2022
  • Jan 24, 2022: Eplontersen granted Orphan Drug Designation in the US for transthyretin amyloidosis
  • 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 ADRx Inc, 2022
  • Pipeline by Akcea Therapeutics Inc, 2022
  • Pipeline by Alexion Pharmaceuticals Inc, 2022
  • Pipeline by Alnylam Pharmaceuticals Inc, 2022
  • Pipeline by Autotac Bio Inc, 2022
  • Pipeline by BridgeBio Pharma Inc, 2022
  • Pipeline by BSIM Therapeutics SA, 2022
  • Pipeline by Corino Therapeutics Inc, 2022
  • Pipeline by Covalent Bioscience Inc, 2022
  • Pipeline by Galmed Pharmaceuticals Ltd, 2022
  • Pipeline by Novo Nordisk AS, 2022
  • Pipeline by Proclara Biosciences Inc, 2022
  • Pipeline by Regeneron Pharmaceuticals Inc, 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 Top 10 Indications, 2022
  • Number of Products by Mechanism of Actions, 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:

  • ADRx Inc
  • Akcea Therapeutics Inc
  • Alexion Pharmaceuticals Inc
  • Alnylam Pharmaceuticals Inc
  • Autotac Bio Inc
  • BridgeBio Pharma Inc
  • BSIM Therapeutics SA
  • Corino Therapeutics Inc
  • Covalent Bioscience Inc
  • Galmed Pharmaceuticals Ltd
  • Novo Nordisk AS
  • Proclara Biosciences Inc
  • Regeneron Pharmaceuticals Inc