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Hemophilia B-Pipeline Review, H1 2017

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Hemophilia B-Pipeline Review, H1 2017



Executive Summary

Hemophilia B-Pipeline Review, H1 2017

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Hemophilia B-Pipeline Review, H1 2017, 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 2017, 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, IND/CTA Filed, Preclinical and Discovery stages are 2, 2, 9, 4, 2, 7 and 3 respectively. Similarly, the Universities portfolio in Preclinical and Discovery stages comprises 2 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 Direct's 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 it's 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

Dimension Therapeutics Inc

Genethon SA

Green Cross Corp

MolMed SpA

Novo Nordisk A/S

OPKO Biologics Ltd

Pfizer Inc

Pharming Group NV

Promethera Biosciences SA

RegenxBio Inc

rEVO Biologics 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

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 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

coagulation factor IX (recombinant), pegylated-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

coagulation factor VIIa (recombinant)-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

DTX-101-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 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

HepaStem-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

LR-769-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

MOD-5014-Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PF-06741086-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

SPK-9001-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

Apr 11, 2017: Key Milestone Reached in Catalyst's Subcutaneous Factor IX Program

Mar 31, 2017: FDA posts briefing materials prior to Advisory Committee meeting for nonacog beta pegol, a long-acting factor IX for the treatment of haemophilia B

Mar 30, 2017: Catalyst Biosciences Announces Emergence from Key Patent Opposition Period Supporting Marzeptacog Alfa (activated), Catalyst's Lead Clinical Program

Mar 28, 2017: Catalyst Biosciences Announces IND Approval in South Korea for Next-Generation Subcutaneous Factor IX Program

Mar 23, 2017: Novo Nordisk Receives CHMP Positive opinion For Refixia

Mar 02, 2017: Long-term safety and efficacy extension study data of Alprolix for haemophilia B published in Thrombosis and Haemostasis

Feb 14, 2017: Catalyst Biosciences Highlights Hemophilia Clinical Development Plans at the 19th Annual BIO CEO & Investor Conference

Feb 02, 2017: Long-term safety and efficacy data of extended half-life therapy Alprolix in children with haemophilia B published in The Lancet Haematology

Feb 02, 2017: Alnylam Presents New Data on Fitusiran at EAHAD

Feb 01, 2017: Catalyst Biosciences Announces Positive Preclinical Data of Subcutaneously Dosed Coagulation Factor IX at EAHAD Annual Congress

Feb 01, 2017: Sobi and Bioverativ to reveal new long-term safety and efficacy data of Alprolix at EAHAD

Feb 01, 2017: Catalyst Biosciences Announces Positive Preclinical Data of Subcutaneously Dosed Coagulation Factor VIIa at EAHAD Annual Congress

Jan 26, 2017: Catalyst Biosciences Announces Presentation on investigational coagulation factor CB 2679d at the 2017 Congress of the European Association of Haemophilia and Allied Disorders

Jan 26, 2017: Catalyst Biosciences Announces Data Presentation on Marzeptacog alfa at the 2017 Congress of the European Association of Haemophilia and Allied Disorders

Jan 06, 2017: FDA Accepts For Review LFB's BLA For Its Recombinant Factor VII

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

To know more information on Purchase by Section, please send a mail to sales@kenresearch.com

List of Figures

Number of Products under Development for Hemophilia B, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Number of Products by Targets, H1 2017

Number of Products by Stage and Targets, H1 2017

Number of Products by Mechanism of Actions, H1 2017

Number of Products by Stage and Mechanism of Actions, H1 2017

Number of Products by Routes of Administration, H1 2017

Number of Products by Stage and Routes of Administration, H1 2017

Number of Products by Molecule Types, H1 2017

Number of Products by Stage and Molecule Types, H1 2017

List of Tables

Number of Products under Development for Hemophilia B, H1 2017

Number of Products under Development by Companies, H1 2017

Number of Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Number of Products under Development by Universities/Institutes, H1 2017

Products under Development by Companies, H1 2017

Products under Development by Companies, H1 2017 (Contd..1), H1 2017

Products under Development by Universities/Institutes, H1 2017

Number of Products by Stage and Target, H1 2017

Number of Products by Stage and Mechanism of Action, H1 2017

Number of Products by Stage and Route of Administration, H1 2017

Number of Products by Stage and Molecule Type, H1 2017

Hemophilia B-Pipeline by Alnylam Pharmaceuticals Inc, H1 2017

Hemophilia B-Pipeline by Amarna Therapeutics BV, H1 2017

Hemophilia B-Pipeline by Bayer AG, H1 2017

Hemophilia B-Pipeline by Bioverativ Inc, H1 2017

Hemophilia B-Pipeline by Catalyst Biosciences Inc, H1 2017

Hemophilia B-Pipeline by China Biologic Products Inc, H1 2017

Hemophilia B-Pipeline by CSL Ltd, H1 2017

Hemophilia B-Pipeline by Dimension Therapeutics Inc, H1 2017

Hemophilia B-Pipeline by Genethon SA, H1 2017

Hemophilia B-Pipeline by Green Cross Corp, H1 2017

Hemophilia B-Pipeline by MolMed SpA, H1 2017

Hemophilia B-Pipeline by Novo Nordisk A/S, H1 2017

Hemophilia B-Pipeline by OPKO Biologics Ltd, H1 2017

Hemophilia B-Pipeline by Pfizer Inc, H1 2017

Hemophilia B-Pipeline by Pharming Group NV, H1 2017

Hemophilia B-Pipeline by Promethera Biosciences SA, H1 2017

Hemophilia B-Pipeline by RegenxBio Inc, H1 2017

Hemophilia B-Pipeline by rEVO Biologics Inc, H1 2017

Hemophilia B-Pipeline by Sangamo Therapeutics Inc, H1 2017

Hemophilia B-Pipeline by Shire Plc, H1 2017

Hemophilia B-Pipeline by Spark Therapeutics Inc, H1 2017

Hemophilia B-Pipeline by UniQure NV, H1 2017

Hemophilia B-Dormant Projects, H1 2017

Hemophilia B-Discontinued Products, H1 2017

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Alnylam Pharmaceuticals Inc, Amarna Therapeutics BV, Bayer AG, Bioverativ Inc, Catalyst Biosciences Inc, China Biologic Products Inc, CSL Ltd, Dimension Therapeutics Inc, Genethon SA, Green Cross Corp, MolMed SpA, Novo Nordisk A/S, OPKO Biologics Ltd, Pfizer Inc, Pharming Group NV, Promethera Biosciences SA, RegenxBio Inc, rEVO Biologics Inc, Sangamo Therapeutics Inc, Shire Plc, Spark Therapeutics Inc, UniQure NV


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