DelveInsight’s, “Hemophilia Pipeline Insight 2023” report provides comprehensive insights about 80+ companies and 80+ pipeline drugs in Hemophilia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
In the Hemophilia Pipeline Report, a detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Hemophilia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Key Takeaways from Hemophilia Pipeline Report
To explore more information on the latest breakthroughs in the Hemophilia Pipeline treatment landscape of the report, click here @ Hemophilia Pipeline Outlook
Hemophilia Overview
Hemophilia is usually an inherited bleeding disorder in which the blood does not clot properly. This can lead to spontaneous bleeding as well as bleeding following injuries or surgery. Blood contains many proteins called clotting factors that can help to stop bleeding. People with hemophilia have low levels of either factor VIII (8) or factor IX (9).
Recent Breakthroughs of the Hemophilia Treatment Landscape
For further information, refer to the detailed Hemophilia Unmet Needs, Hemophilia Market Drivers, and Hemophilia Market Barriers, click here for Hemophilia Ongoing Clinical Trial Analysis
Hemophilia Emerging Drugs Profile
SerpinPC, a specific inhibitor of activated protein C (APC), for the treatment of hemophilia A and hemophilia B. SerpinPC has been observed to be well-tolerated in the clinical setting, associated with promising reductions in bleeding rates, and has PK suitable for infrequent subcutaneous dosing without the need for factor replacement. SerpinPC has human genetic target validation and established proof of concept Phase 2a clinical data.
Fitusiran (ALN-AT3) is a subcutaneously administered, investigational RNAi therapeutic targeting antithrombin (AT) in development for the treatment of hemophilia and rare bleeding disorders (RBDs) by their collaborators at Sanofi Genzyme. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Hemophilia.
ASC618 is an AAV8-based gene therapy for the treatment of hemophilia A, affecting approximately 1 of every 5000 live-born males. ASC618 incorporates a novel liver-specific promoter and a bioengineered, codon-optimized B domain-deleted FVIII variant; in preclinical studies, ASC618 exhibits at least a 10-fold increase in the biosynthesis and secretion of FVIII compared with native human FVIII bioengineered gene constructs. ASC618 has the potential to increase durability of clotting factor biosynthesis and secretion by minimizing cellular stress and induction of the unfolded protein response, which may lead to diminished FVIII production from liver cells. ASC Therapeutics will conduct a phase 1/2 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of ASC618. The program received IND clearance from the U.S. Food and Drug Administration.
Hemophilia Pipeline Therapeutics Assessment
There are approx. 80+ key companies which are developing the therapies for Hemophilia. The companies which have their Hemophilia drug candidates in the most advanced stage, i.e. Phase III include, Alnylam Pharmaceuticals.
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Scope of the Hemophilia Pipeline Report
Dive deep into rich insights for drugs for Hemophilia Market Drivers and Hemophilia Market Barriers, click here @ Hemophilia Unmet Needs and Analyst Views
Table of Content
Got Queries? Find out the related information on Hemophilia Mergers and acquisitions, Hemophilia Licensing Activities @ Hemophilia Emerging Drugs, and Recent Trends
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