Hemophilia B (Factor IX Deficiency) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Hemophilia B (Factor IX Deficiency) Drugs in Development by Stages, Target, MoA, RoA, Molecule Type and Key Players, 2022 Update

Summary

Global Markets Direct's latest Pharmaceutical and Healthcare disease pipeline guide Hemophilia B – Drugs In Development, 2022, 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 – Drugs In Development, 2022, 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 (Factor IX Deficiency) 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 2, 8, 7, 2, 12, 5 and 1 respectively. Similarly, the Universities portfolio in IND/CTA Filed, Preclinical and Discovery stages comprises 1, 6 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.


Introduction
Global Markets Direct Report Coverage
Hemophilia B (Factor IX Deficiency) – Overview
Hemophilia B (Factor IX Deficiency) – Therapeutics Development
Pipeline Overview
Pipeline by Companies
Pipeline by Universities/Institutes
Products under Development by Companies
Products under Development by Universities/Institutes
Hemophilia B (Factor IX Deficiency) – Therapeutics Assessment
Assessment by Target
Assessment by Mechanism of Action
Assessment by Route of Administration
Assessment by Molecule Type
Hemophilia B (Factor IX Deficiency) – Companies Involved in Therapeutics Development
AlphaMab Co Ltd
Amarna Therapeutics BV
ASC Therapeutics Inc
Belief Biomed Inc
Biocad
Catalyst Biosciences Inc
Centessa Pharmaceuticals Plc
Chameleon Biosciences Inc
Chengdu Rongsheng Pharmaceutical Co Ltd
Chia Tai Tianqing Pharmaceutical Group Co Ltd
CSL Ltd
Daiichi Sankyo Co Ltd
Expression Therapeutics Inc
Freeline Therapeutics Holdings Plc
GC Pharma
GeneLeap Biotech
GeneVentiv Therapeutics Inc
Intellia Therapeutics Inc
LFB SA
Logicbio Therapeutics Inc
Novo Nordisk AS
OPKO Health Inc
Pfizer Inc
RegenxBio Inc
Sanofi
Sigilon Therapeutics Inc
SK Plasma Co Ltd
Staidson BioPharma Inc
ToolGen Inc
UBI Pharma Inc
UniQure NV
Zhengzhou Gensciences Inc
Hemophilia B (Factor IX Deficiency) – Drug Profiles
AMA-005 – Drug Profile
ANB-2 – Drug Profile
ASC-519 – Drug Profile
BBM-001 – Drug Profile
BIVV-002 – Drug Profile
CB-2679dGT – Drug Profile
coagulation factor IX (human) – Drug Profile
coagulation factor IX (recombinant) – Drug Profile
coagulation factor IX (recombinant), glycopegylated LA – Drug Profile
coagulation factor VIIa (recombinant) – Drug Profile
concizumab – Drug Profile
dalcinonacog alfa LA – Drug Profile
DS-7411 – Drug Profile
ET-9 – Drug Profile
etranacogene dezaparvovec – Drug Profile
fidanacogene elaparvovec – Drug Profile
fitusiran – Drug Profile
Fusion Protein to Replace Factor IX for Hemophilia B – Drug Profile
Gene Therapy for Hematological Disorders – Drug Profile
Gene Therapy to Activate Coagulation Factor IX for Hemophilia B – Drug Profile
Gene Therapy to Activate F9 for Hemophilia B – Drug Profile
Gene Therapy to Activate Factor IX for Hemophilia B – Drug Profile
GENV-HEM – Drug Profile
GLB-2001 – Drug Profile
Hemophilia B – Drug Profile
KN-054 – Drug Profile
lanacogene vosiparvovec – Drug Profile
LB-101 – Drug Profile
marstacimab – Drug Profile
MG-1113A – Drug Profile
OPK-88005 – Drug Profile
Protein for Hemophilia A and B – Drug Profile
SerpinPC – Drug Profile
SIG-003 – Drug Profile
SKP-0150 – Drug Profile
STSP-0601 – Drug Profile
TG-LBP – Drug Profile
verbrinacogene setparvovec – Drug Profile
VGBR-04 – Drug Profile
Hemophilia B (Factor IX Deficiency) – Dormant Projects
Hemophilia B (Factor IX Deficiency) – Discontinued Products
Hemophilia B (Factor IX Deficiency) – Product Development Milestones
Featured News & Press Releases
May 24, 2022: uniQure announces FDA acceptance of Biologics License Application for Etranacogene Dezaparvovec under priority Review
Mar 28, 2022: EMA initiates review of CSL Behring’s gene therapy for Haemophilia B
Feb 04, 2022: Final analysis of pivotal HOPE-B study demonstrates durable and sustained therapeutic effect of etranacogene dezaparvovec gene therapy in hemophilia B - Data presented at EAHAD 2022
Dec 15, 2021: CSL Behring receives accelerated CHMP assessment for Etranacogene Dezaparvovec for European patients living with hemophilia B
Dec 14, 2021: Data from two phase 3 studies demonstrating fitusiran significantly reduced bleeds in people with hemophilia A or B, with or without inhibitors, were featured at ASH’s plenary and late-breaking sessions
Dec 09, 2021: uniQure and CSL Behring announce primary endpoint achieved in HOPE-B pivotal trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B
Nov 01, 2021: GeneVentiv receives Orphan Drug Designation (ODD) for GENV-HEM for the treatment of hemophilia A or B with or without inhibitors
Jul 19, 2021: Catalyst Biosciences presents data on dalcinonacog alfa LA at the International Society for Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress
Jul 06, 2021: Catalyst Biosciences announces presentations on DalcA at the International Society on Thrombosis and Haemostasis
Jul 02, 2021: uniQure announces presentations on AMT-061 at upcoming International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress
Jul 02, 2021: Sanofi to present on Fitusiran at ISTH 2021
Jun 22, 2021: uniQure announces positive 52-week clinical data from HOPE-B pivotal trial of Etranacogene Dezaparvovec gene therapy in patients with hemophilia B and provides regulatory update
May 13, 2021: uniQure presents HOPE-B clinical data demonstrating clinical benefit in Hemophilia B patients with pre-existing antibodies to AAV5 vector
Apr 26, 2021: uniQure announces FDA removes clinical hold on Hemophilia B gene therapy program
Apr 19, 2021: Catalyst Biosciences announces publication of CB 2679d preclinical FIX gene therapy data in Blood, the Journal of the American Society of Hematology
Appendix
Methodology
Coverage
Secondary Research
Primary Research
Expert Panel Validation
Contact Us
Disclaimer
List of Tables
Table 1: Number of Products under Development for Hemophilia B (Factor IX Deficiency), 2022
Table 2: Number of Products under Development by Companies, 2022
Table 3: Number of Products under Development by Companies, 2022 (Contd..1)
Table 4: Number of Products under Development by Universities/Institutes, 2022
Table 5: Products under Development by Companies, 2022
Table 6: Products under Development by Companies, 2022 (Contd..1)
Table 7: Products under Development by Companies, 2022 (Contd..2)
Table 8: Products under Development by Universities/Institutes, 2022
Table 9: Number of Products by Stage and Target, 2022
Table 10: Number of Products by Stage and Mechanism of Action, 2022
Table 11: Number of Products by Stage and Route of Administration, 2022
Table 12: Number of Products by Stage and Molecule Type, 2022
Table 13: Hemophilia B (Factor IX Deficiency) – Pipeline by AlphaMab Co Ltd, 2022
Table 14: Hemophilia B (Factor IX Deficiency) – Pipeline by Amarna Therapeutics BV, 2022
Table 15: Hemophilia B (Factor IX Deficiency) – Pipeline by ASC Therapeutics Inc, 2022
Table 16: Hemophilia B (Factor IX Deficiency) – Pipeline by Belief Biomed Inc, 2022
Table 17: Hemophilia B (Factor IX Deficiency) – Pipeline by Biocad, 2022
Table 18: Hemophilia B (Factor IX Deficiency) – Pipeline by Catalyst Biosciences Inc, 2022
Table 19: Hemophilia B (Factor IX Deficiency) – Pipeline by Centessa Pharmaceuticals Plc, 2022
Table 20: Hemophilia B (Factor IX Deficiency) – Pipeline by Chameleon Biosciences Inc, 2022
Table 21: Hemophilia B (Factor IX Deficiency) – Pipeline by Chengdu Rongsheng Pharmaceutical Co Ltd, 2022
Table 22: Hemophilia B (Factor IX Deficiency) – Pipeline by Chia Tai Tianqing Pharmaceutical Group Co Ltd, 2022
Table 23: Hemophilia B (Factor IX Deficiency) – Pipeline by CSL Ltd, 2022
Table 24: Hemophilia B (Factor IX Deficiency) – Pipeline by Daiichi Sankyo Co Ltd, 2022
Table 25: Hemophilia B (Factor IX Deficiency) – Pipeline by Expression Therapeutics Inc, 2022
Table 26: Hemophilia B (Factor IX Deficiency) – Pipeline by Freeline Therapeutics Holdings Plc, 2022
Table 27: Hemophilia B (Factor IX Deficiency) – Pipeline by GC Pharma, 2022
Table 28: Hemophilia B (Factor IX Deficiency) – Pipeline by GeneLeap Biotech, 2022
Table 29: Hemophilia B (Factor IX Deficiency) – Pipeline by GeneVentiv Therapeutics Inc, 2022
Table 30: Hemophilia B (Factor IX Deficiency) – Pipeline by Intellia Therapeutics Inc, 2022
Table 31: Hemophilia B (Factor IX Deficiency) – Pipeline by LFB SA, 2022
Table 32: Hemophilia B (Factor IX Deficiency) – Pipeline by Logicbio Therapeutics Inc, 2022
Table 33: Hemophilia B (Factor IX Deficiency) – Pipeline by Novo Nordisk AS, 2022
Table 34: Hemophilia B (Factor IX Deficiency) – Pipeline by OPKO Health Inc, 2022
Table 35: Hemophilia B (Factor IX Deficiency) – Pipeline by Pfizer Inc, 2022
Table 36: Hemophilia B (Factor IX Deficiency) – Pipeline by RegenxBio Inc, 2022
Table 37: Hemophilia B (Factor IX Deficiency) – Pipeline by Sanofi, 2022
Table 38: Hemophilia B (Factor IX Deficiency) – Pipeline by Sigilon Therapeutics Inc, 2022
Table 39: Hemophilia B (Factor IX Deficiency) – Pipeline by SK Plasma Co Ltd, 2022
Table 40: Hemophilia B (Factor IX Deficiency) – Pipeline by Staidson BioPharma Inc, 2022
Table 41: Hemophilia B (Factor IX Deficiency) – Pipeline by ToolGen Inc, 2022
Table 42: Hemophilia B (Factor IX Deficiency) – Pipeline by UBI Pharma Inc, 2022
Table 43: Hemophilia B (Factor IX Deficiency) – Pipeline by UniQure NV, 2022
Table 44: Hemophilia B (Factor IX Deficiency) – Pipeline by Zhengzhou Gensciences Inc, 2022
Table 45: Hemophilia B (Factor IX Deficiency) – Dormant Projects, 2022
Table 46: Hemophilia B (Factor IX Deficiency) – Dormant Projects, 2022 (Contd..1)
Table 47: Hemophilia B (Factor IX Deficiency) – Discontinued Products, 2022
List of Figures
Figure 1: Number of Products under Development for Hemophilia B (Factor IX Deficiency), 2022
Figure 2: Number of Products under Development by Companies, 2022
Figure 3: Number of Products under Development by Universities/Institutes, 2022
Figure 4: Number of Products by Targets, 2022
Figure 5: Number of Products by Stage and Targets, 2022
Figure 6: Number of Products by Mechanism of Actions, 2022
Figure 7: Number of Products by Stage and Mechanism of Actions, 2022
Figure 8: Number of Products by Routes of Administration, 2022
Figure 9: Number of Products by Stage and Routes of Administration, 2022
Figure 10: Number of Products by Molecule Types, 2022
Figure 11: Number of Products by Stage and Molecule Types, 2022

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