DelveInsight Business Research, LLP: Hemophilia Pipeline Insights Featuring 75+ Companies | Clinical Trial Research Evaluation Report By DelveInsight

Hemophilia pipeline constitutes 75+ key companies continuously working towards developing 80+ Hemophilia treatment therapies, analyzes DelveInsight

LAS VEGAS, Oct. 19, 2022 /PRNewswire/ -- DelveInsight's 'Hemophilia Pipeline Insight - 2022' report provides comprehensive global coverage of available, marketed, and pipeline hemophilia therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the hemophilia pipeline domain.

Key Takeaways from the Hemophilia Pipeline Report

DelveInsight's hemophilia pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for hemophilia treatment.
Leading hemophilia companies such as Intellia tx, Amarna therapeutics, Expression Therapeutics, GC Pharma, Chameleon Biosciences, Pfizer, UBI Pharma, GeneVentiv, Chia Tai Tianqing Pharmaceutical Group, Bayer, ASC Therapeutics, Catalyst Biosciences, Staidson Beijing BioPharmaceuticals, Spark Therapeutics, CSL Behring, Sanofi, Novo Nordisk, Centessa Pharmaceuticals, OPKO Health, Freeline Therapeutics, Spark Therapeutics, Novo Nordisk, Asklepios BioPharmaceutical, Belief Biomed, ASC Therapeutics, uniQure, Sanofi, Bioverativ, and others are evaluating novel hemophilia treatment drugs candidate to improve the treatment landscape.
Key hemophilia pipeline therapies in various stages of development include Hemophilia B Research Project, Hemophilia A Research Project, AMA006, Hemophilia ET3 Research Project, Hemophilia ET8 Research Project, Hemophilia ET9 Research Project, MG1113A, Hemophilia EVADER Research Project, PF-06741086, Giroctocogene fitelparvovec, PF06838435, UB-854, GENV-HEM, GENV-001, TQG203, BAY1093884, ASC618, Marzeptacog Alfa, STSP-0601, SPK-8011, Fitusiran, Concizumab, SerpinPC, MOD-5014, FLT180a, SPK-8011, Mim 8, AskBio009, BBM-H901, PF 06838435, ASC618 and Etranacogene dezaparvovec, Efanesoctocog alfa, and others
In September 2022, Pfizer and Sangamo Therapeutics announced that the Phase III AFFINE study evaluating giroctocogene fitelparvovec, investigational gene therapy for patients with moderately severe to severe hemophilia A, has re-opened recruitment. All trial sites are anticipated to be active by the end of 2022, and a pivotal readout is expected in the first half of 2024.
In June 2022, Freeline Therapeutics Holdings announced that it had begun dosing the second cohort in its Phase I/II B-LIEVE dose confirmation trial of FLT180a in people with hemophilia B.
In May 2022, uniQure announced that the FDA accepted for priority review the Biologics License Application (BLA) submitted for etranacogene dezaparvovec. Priority review of a BLA is reserved for medicines that, if approved, would be potentially significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications.
In March 2022, The Food and Drug Administration (FDA) granted Fast Track designation to ASC618 for the treatment of hemophilia A.
In January 2022, 2seventy bio announced that it has entered into an option and license agreement with Novo Nordisk for joint research and development of an in vivo gene editing treatment for hemophilia A. This agreement builds upon a successful existing multi-year research collaboration between the two companies.
In December 2021, Belief BioMed Group (BBM) announced that it had successfully dosed the first subject in the registrational gene therapy clinical trial by intravenous (IV) infusion of BBM-H901, an adeno-associated virus (AAV) vector expressing factor IX gene for treatment of adult male hemophilia B patients.

Request a sample and discover the recent advances in hemophilia treatment @Hemophilia Pipeline Report

The hemophilia pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage hemophilia products, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the hemophilia pipeline landscape.

Hemophilia Overview

Hemophilia is a genetic bleeding disorder in which the blood fails to clot properly. This can result in both spontaneous and post-injury or surgical bleeding. Blood contains numerous proteins known as clotting factors that can aid in the prevention of bleeding. Hemophilia patients have low levels of either factor VIII (8) or factor IX (9). The amount of factors in a person's blood determines the severity of their hemophilia. The less of the factor there is, the more likely bleeding will occur, which can lead to serious health problems. The hemophilia causes are mainly genetic.

The hemophilia symptoms vary depending on the clotting factor levels. If the clotting factor level is slightly low, you may only bleed after surgery or trauma. A person may bleed for no apparent reason if the deficiency is severe. Clotting factor tests are required for hemophilia diagnosis.

Find out more about hemophilia treatment @Hemophilia Gene Therapy

A snapshot of the Hemophilia Pipeline Drugs mentioned in the report:

Drugs	Company	Phase	MoA	RoA
Etranacogene dezaparvovec	uniQure	Preregistration	Gene transference	Intravenous
Fitusiran	Alnylam Pharmaceuticals	Phase III	Antithrombin III expression inhibitors; Haemostasis stimulants; RNA interference	Subcutaneous
SPK-8011	Spark Therapeutics	Phase III	Factor VIII replacements; Gene transference	Intravenous
Mim8	Novo Nordisk	Phase III	Blood coagulation factor stimulant	Subcutaneous
SerpinPC	Centessa Pharmaceuticals	Phase II	Activated protein C receptor modulators; Serine protease inhibitors	Intravenous
FLT-180a	Freeline Therapeutics	Phase I/II	Factor IX replacements; Gene transference	Intravenous
ASC 618	ASC Therapeutics	Phase I/II	Factor VIII replacements; Gene transference	Parenteral
STSP 0601	Staidson Beijing BioPharmaceuticals	Phase I	Factor X stimulants	Parenteral
GENV-001	GeneVentiv	Preclinical	Gene transference	Unspecified

Learn more about the emerging hemophilia pipeline therapies @Hemophilia Clinical Trials

Hemophilia Therapeutics Assessment

The hemophilia pipeline report proffers an integral view of hemophilia emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

Scope of the Hemophilia Pipeline Report

Coverage: Global
Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Ophthalmic, Oral, Parenteral, Subcutaneous, Topical, Transdermal
Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule, Gene Therapy
Therapeutics Assessment By Mechanism of Action: Activated protein C receptor modulators, Serine protease inhibitors, Antithrombin III expression inhibitors, Haemostasis stimulants, RNA interference, Factor VIII replacements, Gene transference, Factor IX replacements, Factor X stimulants
Key Hemophilia Companies: Intellia tx, Amarna therapeutics, Expressi Ontherapeutics, GC Pharma, Chameleon Biosciences, Pfizer, UBI Pharma, GeneVentiv, Chia Tai Tianqing Pharmaceutical Group, Bayer, ASC Therapeutics, Catalyst Biosciences, Staidson Beijing BioPharmaceuticals, Spark Therapeutics, CSL Behring, Sanofi, Novo Nordisk, Centessa Pharmaceuticals, OPKO Health, Freeline Therapeutics, Spark Therapeutics, Novo Nordisk, Asklepios BioPharmaceutical, Belief Biomed, ASC Therapeutics, uniQure, Sanofi, Bioverativ, and others
Key Hemophilia Pipeline Therapies: Hemophilia B Research Project, Hemophilia A Research Project, AMA006, Hemophilia ET3 Research Project, Hemophilia ET8 Research Project, Hemophilia ET9 Research Project, MG1113A, Hemophilia EVADER Research Project, PF-06741086, PF07055480, PF06838435, UB-854, GENV-HEM, GENV-001, TQG203, BAY1093884, ASC618, Marzeptacog Alfa, STSP-0601, SPK-8011, Fitusiran, Concizumab, SerpinPC, MOD-5014, FLT180a, SPK-8011, Mim 8, AskBio009, BBM-H901, PF 06838435, ASC618 and Etranacogene dezaparvovec, Efanesoctocog alfa, and others

Dive deep into rich insights for drugs for hemophilia, visit @Hemophilia Medications

Table of Contents

1.	Hemophilia Pipeline Report Introduction
2.	Hemophilia Pipeline Report Executive Summary
3.	Hemophilia Pipeline: Overview
4.	Analytical Perspective In-depth Commercial Assessment
5.	Hemophilia Pipeline Therapeutics
6.	Hemophilia Pipeline: Late Stage Products (Pre-registration)
6.1	Etranacogene dezaparvovec: uniQure
7.	Hemophilia Pipeline: Late Stage Products (Phase III)
7.1	Mim8: Novo Nordisk
8.	Hemophilia Pipeline: Mid Stage Products (Phase II)
8.1	SerpinPC: Centessa Pharmaceuticals
9.	Hemophilia Pipeline: Early Stage Products (Phase I)
9.1	STSP 0601: Staidson Beijing BioPharmaceuticals
10.	Hemophilia Pipeline Therapeutic Assessment
11.	Inactive Products in the Hemophilia Pipeline
12.	Company-University Collaborations (Licensing/Partnering) Analysis
13.	Key Companies
14.	Key Products in the Hemophilia Pipeline
15.	Unmet Needs
16.	Market Drivers and Barriers
17.	Future Perspectives and Conclusion
18.	Analyst Views
19.	Appendix

For further information on the hemophilia pipeline therapeutics, reach out @Hemophilia Drugs

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