Hemophilia B - Pipeline Insight, 2024

This “Hemophilia B - Pipeline Insight, 2024” report provides comprehensive insights about 15+ companies and 20+ pipeline drugs in Hemophilia B 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.

Geography Covered

• Global coverage

Hemophilia B: Understanding

Hemophilia B: Overview

Hemophilia B is a rare genetic bleeding disorder characterized by a deficiency in factor IX clotting activity, leading to prolonged oozing after injuries, tooth extractions, or surgery, and delayed or recurrent bleeding prior to complete wound healing. The age of diagnosis and frequency of bleeding episodes are related to the level of factor IX clotting activity. In severe cases, individuals may experience frequent spontaneous bleeding episodes, including joint or muscle bleeds, while those with moderate hemophilia B may have less frequent bleeding, typically after minor trauma. Individuals with mild hemophilia B usually do not have spontaneous bleeding episodes but may experience abnormal bleeding during surgery or tooth extractions. The severity of the condition and the frequency of bleeding episodes vary based on the level of factor IX activity, with severe cases usually diagnosed in early childhood and milder cases potentially not being diagnosed until later in life.

The diagnostic tests for hemophilia B include specialized blood coagulation tests to measure the blood's clotting time. Several specific tests are necessary to confirm a diagnosis of hemophilia B, with the initial test being the activated partial thromboplastin time (aPTT). If the results of the aPTT test are abnormal, more specific blood tests must be used to determine if the cause of the abnormal aPTT is due to a factor IX (FIX) deficiency. Additionally, screening tests, clotting factor tests (factor assays), and a complete blood cell count are conducted to diagnose and determine the severity of hemophilia B. Family history of bleeding and inheritance is also considered in the diagnostic process. The severity of hemophilia symptoms varies depending on the clotting factor levels, with clotting factor tests being required for hemophilia diagnosis.

Hemophilia B is typically treated with factor replacement therapy, involving the injection of concentrated factor IX to enable proper blood clotting. Patients with severe hemophilia B may require regular infusions to prevent excessive bleeding, while those with moderate hemophilia B may be on a routine treatment regimen, called prophylaxis, to maintain enough clotting factor in their bloodstream to prevent bleeds. Additionally, gene therapy and other new treatments are being investigated and developed as potential alternative options for individuals with hemophilia B

"Hemophilia B - Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hemophilia B pipeline landscape is provided which includes the disease overview and Hemophilia B treatment guidelines. The assessment part of the report embraces, in depth Hemophilia B commercial assessment and clinical assessment of the pipeline products under development. In the report, 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 B collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Hemophilia B R&D. The therapies under development are focused on novel approaches to treat/improve Hemophilia B

Hemophilia B Emerging Drugs Chapters

This segment of the Hemophilia B report encloses its detailed analysis of various drugs in different stages of clinical development, including Phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Hemophilia B Emerging Drugs

BBM-H901: Belief Biomed

BBM-H901 is indicated for prophylactic treatment of bleeding in adults with hemophilia B. BBM-H901 is designed to restore the production of factor IX (FIX) the blood-clotting protein that is faulty or missing in people with hemophilia B by delivering a functional copy of the F9 gene to liver cells, which are the main producers of clotting factors in the body. Gene therapy components are delivered to liver cells by a modified, harmless adeno-associated virus (AAV) that works as a vehicle. The gene therapy is given as a single intravenous (into-the-vein) infusion. The drug is currently in Phase III stage of clinical trial evaluation for the treatment of hemophilia B.

ISU304: ISU ABXIS

ISU304 (Dalcinonacog alfa, DalcA) is a modified recombinant Factor IX protein for the prophylactic therapy of patients with Hemophilia B which is caused by a deficiency of coagulation FIX. It is designed to be administered subcutaneously and achieves normal FIX activity levels to prevent bleeding of patients with hemophilia B. Currently approved protein replacement therapy for hemophilia B rely on intravenous injection. With a subcutaneous injection, ISU304 is expected to improve the quality of life and treatment convenience in patients with hemophilia B. The drug is in phase II stage of its clinical trial for the treatment of hemophilia B.

TU7710: TiumBio

TU7710 is a bypassing agent treatment for patients with neutralizing antibodies and is an innovative drug candidate with a half-life 7 times greater than conventional treatments through our transferrin fusion gene recombination technology. TU7710 is expected to dramatically improve the convenience and quality of life of hemophilia patients with neutralizing antibodies. Currently, the drug is in phase I stage of its clinical trial for the treatment of Hemophilia B.

Hemophilia B Therapeutic Assessment

This segment of the report provides insights about the different Hemophilia B drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Hemophilia B

There are approx. 15+ key companies which are developing the therapies for Hemophilia B. The companies which have their Hemophilia B drug candidates in the most advanced stage, i.e. Phase III include, Belief Biomed.

Phases

This report covers around 20+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Hemophilia B pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hemophilia B Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Hemophilia B therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hemophilia B drugs.

Hemophilia B Report Insights

• Hemophilia B Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Hemophilia B Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Hemophilia B drugs?
• How many Hemophilia B drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hemophilia B?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hemophilia B therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hemophilia B and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Shanghai Vitalgen BioPharma
• Belief BioMed
• TiumBio
• Jiangsu Gensciences
• Centessa Pharmaceuticals
• Takeda
• Sanofi
• GC Biopharma Corp
• Equilibra Bioscience LLC
• Regeneron Pharmaceuticals
• ISU Abxis

Key Products

• VGB-R04
• BBM-H901
• TU7710
• SS109
• SerpinPC
• AskBio009
• Fitusiran
• MG1113
• SR604
• REGV131
• ISU304

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