Hemophilia B - Pipeline Review, H1 2018

  • ID: 4482748
  • Report
  • 126 pages
  • Global Markets Direct
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FEATURED COMPANIES

  • Amarna Therapeutics BV
  • Catalyst Biosciences Inc
  • Genethon SA
  • MolMed SpA
  • Pfizer Inc
  • RegenxBio Inc
  • MORE
Hemophilia B - Pipeline Review, H1 2018

Summary:

This 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:

This 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 the publisher'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 for Purchase:
  • 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.
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Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Amarna Therapeutics BV
  • Catalyst Biosciences Inc
  • Genethon SA
  • MolMed SpA
  • Pfizer Inc
  • RegenxBio Inc
  • MORE
Introduction

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
Note: Product cover images may vary from those shown
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  • 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
Note: Product cover images may vary from those shown
5 of 5
Note: Product cover images may vary from those shown
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