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Hemophilia B-Pipeline Review, H1 2018

Published By :

Global Markets Direct

Published Date : Mar 2018

Category :

Pharmaceutical

No. of Pages : 126 Pages

Hemophilia B - Pipeline Review, H1 2018

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Hemophilia B - Pipeline Review, H1 2018, provides an overview of the Hemophilia B (Hematological Disorders) pipeline landscape.

Hemophilia B is a hereditary bleeding disorder caused by a lack of blood clotting factor IX. Symptoms include bleeding into joints and associated pain and swelling, bruising, excessive bleeding following circumcision, gastrointestinal tract and urinary tract hemorrhage and nosebleeds. Treatment includes replacing the defective clotting factor.

Report Highlights

Global Markets Direct's Pharmaceutical and Healthcare latest pipeline guide Hemophilia B - Pipeline Review, H1 2018, provides comprehensive information on the therapeutics under development for Hemophilia B (Hematological Disorders), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

The Hemophilia B (Hematological Disorders) pipeline guide also reviews of key players involved in therapeutic development for Hemophilia B and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Pre-Registration, Phase III, Phase II, Phase I, Preclinical, Discovery and Unknown stages are 1, 3, 10, 2, 11, 1 and 1 respectively. Similarly, the Universities portfolio in Phase II, Preclinical and Discovery stages comprises 1, 4 and 1 molecules, respectively.

Hemophilia B (Hematological Disorders) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. The guide is built using data and information sourced from Global Markets Directs 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. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

Note: Certain content / sections in the pipeline guide may be removed or altered based on the availability and relevance of data.

Scope

- The pipeline guide provides a snapshot of the global therapeutic landscape of Hemophilia B (Hematological Disorders).
- The pipeline guide reviews pipeline therapeutics for Hemophilia B (Hematological Disorders) by companies and universities/research institutes based on information derived from company and industry-specific sources.
- The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages.
- The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities.
- The pipeline guide reviews key companies involved in Hemophilia B (Hematological Disorders) therapeutics and enlists all their major and minor projects.
- The pipeline guide evaluates Hemophilia B (Hematological Disorders) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type.
- The pipeline guide encapsulates all the dormant and discontinued pipeline projects.
- The pipeline guide reviews latest news related to pipeline therapeutics for Hemophilia B (Hematological Disorders)

Reasons to buy

- Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies.
- Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage.
- Find and recognize significant and varied types of therapeutics under development for Hemophilia B (Hematological Disorders).
- Classify potential new clients or partners in the target demographic.
- Develop tactical initiatives by understanding the focus areas of leading companies.
- Plan mergers and acquisitions meritoriously by identifying key players and its most promising pipeline therapeutics.
- Formulate corrective measures for pipeline projects by understanding Hemophilia B (Hematological Disorders) pipeline depth and focus of Indication therapeutics.
- 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.
- Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline.
Table of Contents
List of Tables
List of Figures
Introduction
Global Markets Direct Report Coverage
Hemophilia B - Overview
Hemophilia B - Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Hemophilia B - Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Hemophilia B - Companies Involved in Therapeutics Development
Alnylam Pharmaceuticals Inc
Amarna Therapeutics BV
Bayer AG
Bioverativ Inc
Catalyst Biosciences Inc
China Biologic Products Inc
CSL Ltd
Expression Therapeutics LLC
GC Pharma
Genethon SA
LFB SA
Logicbio Therapeutics Inc
MolMed SpA
Novo Nordisk AS
OPKO Biologics Ltd
Pfizer Inc
Pharming Group NV
Promethera Biosciences SA
RegenxBio Inc
Sangamo Therapeutics Inc
Shire Plc
Spark Therapeutics Inc
UniQure NV
Hemophilia B - Drug Profiles
AMT-060 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
AMT-061 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BAX-335 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BAY-1093884 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
BIVV-002 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CB-2679d - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Cell Therapy to Activate F9 for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
coagulation factor IX (human) - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
concizumab - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
CSL-689 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
fidanacogene elaparvovec - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
fitusiran - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Hematological Disorders - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Coagulation Factor IX for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Coagulation Factor IX for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Coagulation Factor IX for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Gene Therapy to Activate Factor IX for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
HepaStem - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
LR-769 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
marstacimab - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
marzeptacog alfa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
MG-1113A - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
OPK-88005 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Factor IX Replacement for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Recombinant Protein to Inhibit Coagulation Factor XIV for Hemophilia A and Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SB-FIX - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SHP-648 - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Stem Cell Therapy to Activate Coagulation Factor IX for Hemophilia B - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
SVF-VIIa - Drug Profile
Product Description
Mechanism Of Action
R&D Progress
Hemophilia B - Dormant Projects
Hemophilia B - Discontinued Products
Hemophilia B - Product Development Milestones
Featured News & Press Releases
Feb 28, 2018: Sangamo Announces U.K. Authorization Of Clinical Trial Evaluating Zinc Finger Nuclease In Vivo Genome Editing Treatment For Hemophilia B
Feb 05, 2018: Spark Therapeutics to Present Data on its Hemophilia B Drug Candidate SPK-8011 in EAHAD Conference, Spain
Dec 15, 2017: FDA lifts clinical hold on fitusiran
Dec 11, 2017: New Data Show Extended Prophylactic Dosing with ALPROLIX Provides Safe and Effective Protection in People with Severe Hemophilia B
Dec 11, 2017: Spark Therapeutics and Pfizer Announce Longer-term Preliminary Data Showing Consistent and Sustained Factor IX Levels in Hemophilia B at 59th American Society of Hematology (ASH) Annual Meeting and Exposition
Dec 09, 2017: ALPROLIX Demonstrates Higher Tissue Distribution and Retention in Joints Compared to Other Factor IX Molecules in a Preclinical Imaging Study
Dec 06, 2017: One-dose gene therapy produces clotting factor, safely stops bleeding in hemophilia B patients
Dec 06, 2017: Spark Therapeutics and Pfizer Announce Publication in The New England Journal of Medicine of Interim Data from Phase 1/2 Clinical Trial of Investigational Gene Therapy for Hemophilia B
Dec 05, 2017: Spark Therapeutics Announces Presentation on Updated Hemophilia B Clinical Trial at 59th American Society of Hematology Annual Meeting and Exposition
Nov 27, 2017: U.S. Label Update for Bioverativs ALPROLIX Adds Data Reinforcing Efficacy and Safety of Long-Term Prophylactic Treatment in Adults, Adolescents and Children With Hemophilia B
Nov 09, 2017: Alnylam Announces Successful Outcome Following FDA Type A Meeting to Discuss Fitusiran Program in Hemophilia
Nov 01, 2017: Bioverativ to Present New Data on ALPROLIX at ASH 2017 Annual Meeting
Oct 25, 2017: Alprolix approved in the Kingdom of Saudi Arabia for the treatment of hemophilia B
Sep 07, 2017: Alnylam Provides Pipeline Update on Fitusiran Investigational RNAi Therapeutic Programs
Sep 07, 2017: Sanofi Alliance Partner Alnylam Provides Update on Fitusiran Development Program
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer

List of Tables
Number of Products under Development for Hemophilia B, H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Companies, H1 2018 (Contd..1), H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Products under Development by Companies, H1 2018
Products under Development by Companies, H1 2018 (Contd..1), H1 2018
Products under Development by Universities/Institutes, H1 2018
Number of Products by Stage and Target, H1 2018
Number of Products by Stage and Mechanism of Action, H1 2018
Number of Products by Stage and Route of Administration, H1 2018
Number of Products by Stage and Molecule Type, H1 2018
Hemophilia B - Pipeline by Alnylam Pharmaceuticals Inc, H1 2018
Hemophilia B - Pipeline by Amarna Therapeutics BV, H1 2018
Hemophilia B - Pipeline by Bayer AG, H1 2018
Hemophilia B - Pipeline by Bioverativ Inc, H1 2018
Hemophilia B - Pipeline by Catalyst Biosciences Inc, H1 2018
Hemophilia B - Pipeline by China Biologic Products Inc, H1 2018
Hemophilia B - Pipeline by CSL Ltd, H1 2018
Hemophilia B - Pipeline by Expression Therapeutics LLC, H1 2018
Hemophilia B - Pipeline by GC Pharma, H1 2018
Hemophilia B - Pipeline by Genethon SA, H1 2018
Hemophilia B - Pipeline by LFB SA, H1 2018
Hemophilia B - Pipeline by Logicbio Therapeutics Inc, H1 2018
Hemophilia B - Pipeline by MolMed SpA, H1 2018
Hemophilia B - Pipeline by Novo Nordisk AS, H1 2018
Hemophilia B - Pipeline by OPKO Biologics Ltd, H1 2018
Hemophilia B - Pipeline by Pfizer Inc, H1 2018
Hemophilia B - Pipeline by Pharming Group NV, H1 2018
Hemophilia B - Pipeline by Promethera Biosciences SA, H1 2018
Hemophilia B - Pipeline by RegenxBio Inc, H1 2018
Hemophilia B - Pipeline by Sangamo Therapeutics Inc, H1 2018
Hemophilia B - Pipeline by Shire Plc, H1 2018
Hemophilia B - Pipeline by Spark Therapeutics Inc, H1 2018
Hemophilia B - Pipeline by UniQure NV, H1 2018
Hemophilia B - Dormant Projects, H1 2018
Hemophilia B - Discontinued Products, H1 2018

List of Figures
Number of Products under Development for Hemophilia B, H1 2018
Number of Products under Development by Companies, H1 2018
Number of Products under Development by Universities/Institutes, H1 2018
Number of Products by Targets, H1 2018
Number of Products by Stage and Targets, H1 2018
Number of Products by Mechanism of Actions, H1 2018
Number of Products by Stage and Mechanism of Actions, H1 2018
Number of Products by Routes of Administration, H1 2018
Number of Products by Stage and Routes of Administration, H1 2018
Number of Products by Molecule Types, H1 2018
Number of Products by Stage and Molecule Types, H1 2018

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