Aplastic Anemia - Pipeline Insight, 2024

This “Aplastic Anemia - Pipeline Insight, 2024,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Aplastic Anemia 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

Aplastic Anemia Understanding

Aplastic Anemia: Overview

Aplastic anemia (AA) is a life-threatening form of bone marrow failure which, if untreated, is associated with very high mortality. AA refers to pancytopenia in association with bone marrow hypoplasia/aplasia, most often due to immune injury to multipotent hematopoietic stem cells. Aplastic anemia can be moderate, severe or very severe. People with severe or very severe aplastic anemia are at risk for life-threatening infections or bleeding. With prompt and proper care, most people who have aplastic anemia can be successfully treated. The only cure for aplastic anemia is a bone marrow transplant. Aplastic anemia can strike at any age regardless of race or gender. However, it is diagnosed more often in children, young adults and older adults. It also appears more often in Asian-Americans. Each year, between 600 and 900 people in the U.S. learn that they have aplastic anemia.

Aplastic Anemia - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Aplastic Anemia pipeline landscape is provided which includes the disease overview and Aplastic Anemia treatment guidelines. The assessment part of the report embraces, in depth Aplastic Anemia 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, Aplastic Anemia 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 Aplastic Anemia R&D. The therapies under development are focused on novel approaches to treat/improve Aplastic Anemia.

Aplastic Anemia Emerging Drugs Chapters

This segment of the Aplastic Anemia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.

Aplastic Anemia Emerging Drugs

PF-06462700: Pfizer PF-06462700 is an immunosuppressant/immunosuppressive agent. PF-06462700 is being developed by Pfizer for Aplastic Anemia patients and is currently in the phase III stage ofdevelopment.

REGN7257: Regeneron Pharmaceuticals REGN7257is being developed by Regeneron Pharmaceuticals for Aplastic Anemia. It is currently in phase I/II stage of development.

BL-8040: Bio LineRx, Ltd.BL-8040 (Motixafortide) targets CXCR4, a chemokine receptor and a well-validated therapeutic target that is over-expressed in many human cancers, including PDAC. CXCR4 plays a key role in tumor growth, invasion, angiogenesis, metastasis, therapeutic resistance, and CXCR4 overexpression correlated with poor prognosis. A Phase II clinical trial has been completed for BL-8040 for the treatment of AplasticAnemia.

Aplastic Anemia: Therapeutic Assessment

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

Major Players in Aplastic Anemia

There are approx. 8+ key companies which are developing the therapies for Aplastic Anemia. The companies which have their Aplastic Anemia drug candidates in the most advanced stage, i.e. phase III include, Pfizer.

Phases

This report covers around 8+ 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

Aplastic Anemia 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
• Parenteral
• intravenous
• Subcutaneous
• Topical.

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Aplastic Anemia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Aplastic Anemia 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 Aplastic Anemia drugs.

Aplastic Anemia Report Insights

• Aplastic Anemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Aplastic Anemia 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 Aplastic Anemia drugs?
• How many Aplastic Anemia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Aplastic Anemia?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Aplastic Anemia 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 Aplastic Anemia and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Pfizer
• Bio LineRx,Ltd.
• Regeneron Pharmaceuticals
• Gamida Cell
• Elixirgen
• Hangzhou Zede Pharmaceutical Technology
• Cellenkos
• Hemogenyx Pharmaceuticals

Key Products

• PF-06462700
• BL-8040
• REGN7257
• Omidubicel
• EXG 34217
• CN 128
• CK0801
• Hu-PHEC Liver

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