Table of Content


List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Familial Amyloid Neuropathies - Overview
Familial Amyloid Neuropathies - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Familial Amyloid Neuropathies - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Familial Amyloid Neuropathies - Companies Involved in Therapeutics Development
ADRx Inc
Akcea Therapeutics Inc
Alnylam Pharmaceuticals Inc
BSIM Therapeutics SA
Corino Therapeutics Inc
Eidos Therapeutics Inc
Precision Biosciences Inc
Proclara Biosciences Inc
Prothe Corp Plc
Regeneron Pharmaceuticals Inc
Familial Amyloid Neuropathies - Drug Profiles
Antibody to Inhibit TTR for Familial Transthyretin Amyloidosis - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BBP-265 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CLR-01 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
doxycycline hyclate - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
eplontersen sodium - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Transthyretin For Familial Amyloid Neuropathies - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NPT-189 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
NTLA-1001 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
patisiran - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
PRX-004 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Small Molecules to Inhibit Transthyretin Related Amyloid Protein for Familial Amyloidotic Polyneuropathy - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
tolcapone - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
vutrisiran - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Familial Amyloid Neuropathies - Dormant Projects
Familial Amyloid Neuropathies - Discontinued Products
Familial Amyloid Neuropathies - Product Development Milestones
Featured News & Press Releases
Dec 06, 2018: Eidos Therapeutics Appoints William Lis to Board of Directors
Nov 13, 2017: European Medicines Agency (EMA) Grants Alnylam Accelerated Assessment of Patisiran for Patients with Hereditary ATTR (hATTR) Amyloidosis
Nov 02, 2017: Sanofi and Alnylam Present Positive Complete Results from APOLLO Phase 3 study of Investigatiol Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy
Sep 20, 2017: Arbutus’ LNP Licensee Alnylam Announces Positive Phase 3 Results for LNP-Ebled Patisiran Program
Sep 20, 2017: Sanofi and Alnylam Report Positive Topline Results from APOLLO Phase 3 Study of Patisiran in Hereditary ATTR (hATTR) Amyloidosis Patients with Polyneuropathy
Apr 26, 2017: Alnylam Reports Fil 24-Month Results from Phase 2 Open-Label Extension Study of Patisiran, an Investigatiol Ri Therapeutic in Development for the Treatment of Hereditary ATTR (hATTR) Amyloidosis
Oct 10, 2016: Data Monitoring Committee Recommends Continuation of APOLLO Phase 3 Clinical Trial of Patisiran for Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN)
Jul 01, 2016: Alnylam Reports New Results on patisiran at the XV Intertiol Symposium on Amyloidosis
Jun 27, 2016: Alnylam to Report New Patisiran Results at the XV Intertiol Symposium on Amyloidosis
Apr 20, 2016: Alnylam Reports Complete 18-Month Data from Ongoing Phase 2 Open-Label Extension Study of Patisiran, an Investigatiol Ri Therapeutic Targeting Transthyretin for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy (hATTR-PN)
Feb 01, 2016: Alnylam Completes Enrollment in APOLLO Phase 3 Study with Patisiran, an Investigatiol Ri Therapeutic for Patients with Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
Nov 03, 2015: Alnylam Reports Positive Results from Ongoing Phase 2 Open-Label Extension Studies for Patisiran in Development for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
Sep 28, 2015: Alnylam and Collaborators Report First-Ever Evidence that Patisiran Reduces Pathogenic, Misfolded Transthyretin Monomers and Oligomers in TTR-Mediated Amyloidosis Patients with Familial Amyloidotic Polyneuropathy
Jul 20, 2015: Alnylam Initiates Phase 3 Open Label Extension Study with Patisiran, an Investigatiol Ri Therapeutic Targeting Transthyretin for the Treatment of TTR-Mediated Amyloidosis
Apr 21, 2015: Alnylam Reports 12-Month Clinical Data from Phase 2 Open Label Extension Study of Patisiran, an Investigatiol Ri Therapeutic for Patients with Familial Amyloidotic Polyneuropathy
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer



List of Figures


Number of Products under Development for Familial Amyloid Neuropathies, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Number of Products by Top 10 Targets, H1 2020
Number of Products by Stage and Top 10 Targets, H1 2020
Number of Products by Top 10 Mechanism of Actions, H1 2020
Number of Products by Stage and Top 10 Mechanism of Actions, H1 2020
Number of Products by Top 10 Routes of Administration, H1 2020
Number of Products by Stage and Top 10 Routes of Administration, H1 2020
Number of Products by Top 10 Molecule Types, H1 2020
Number of Products by Stage and Top 10 Molecule Types, H1 2020


List of Tables


Number of Products under Development for Familial Amyloid Neuropathies, H1 2020
Number of Products under Development by Companies, H1 2020
Number of Products under Development by Universities/Institutes, H1 2020
Products under Development by Companies, H1 2020
Products under Development by Universities/Institutes, H1 2020
Number of Products by Stage and Target, H1 2020
Number of Products by Stage and Mechanism of Action, H1 2020
Number of Products by Stage and Route of Administration, H1 2020
Number of Products by Stage and Molecule Type, H1 2020
Familial Amyloid Neuropathies - Pipeline by ADRx Inc, H1 2020
Familial Amyloid Neuropathies - Pipeline by Akcea Therapeutics Inc, H1 2020
Familial Amyloid Neuropathies - Pipeline by Alnylam Pharmaceuticals Inc, H1 2020
Familial Amyloid Neuropathies - Pipeline by BSIM Therapeutics SA, H1 2020
Familial Amyloid Neuropathies - Pipeline by Corino Therapeutics Inc, H1 2020
Familial Amyloid Neuropathies - Pipeline by Eidos Therapeutics Inc, H1 2020
Familial Amyloid Neuropathies - Pipeline by Precision Biosciences Inc, H1 2020
Familial Amyloid Neuropathies - Pipeline by Proclara Biosciences Inc, H1 2020
Familial Amyloid Neuropathies - Pipeline by Prothena Corp Plc, H1 2020
Familial Amyloid Neuropathies - Pipeline by Regeneron Pharmaceuticals Inc, H1 2020
Familial Amyloid Neuropathies - Dormant Projects, H1 2020
Familial Amyloid Neuropathies - Discontinued Products, H1 2020