DelveInsight’s, “Beta thalassemia (B-thal) Pipeline Insight 2023” report provides comprehensive insights about 22+ companies and 22+ pipeline drugs in Beta thalassemia (B-thal) pipeline landscape. It covers the Beta thalassemia pipeline drug profiles, including Beta thalassemia clinical trials and nonclinical stage products. It also covers the Beta thalassemia therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Key Takeaways from the Beta thalassemia Pipeline Report
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Beta Thalassemia Overview
Beta thalassemia is an inherited blood disorder that limits your body’s ability to make beta-globin. Beta-globin is an important protein needed to make hemoglobin and red blood cells. Beta thalassemia can cause the patient to experience anemia symptoms.
Recent Developmental Activities in the Beta thalassemia Treatment Landscape
For further information, refer to the detailed Beta Thalassemia Drugs Launch, Beta Thalassemia Developmental Activities, and Beta Thalassemia News, click here for Beta Thalassemia Ongoing Clinical Trial Analysis
Beta Thalassemia Emerging Drugs Profile
CTX001: CRISPR Therapeutics
CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy in development for patients suffering from β-thalassemia and sickle cell disease. The drug is in Phase I/II clinical evaluation for the treatment of β-thalassemia. CTX001 has been designated an orphan drug in the U.S. and Europe, and given fast track, rare pediatric disease, and regenerative medicine advanced therapy designations in the U.S. for SCD and transfusion-dependent Beta thalassemia (B-thal) (TDT).
VIT-2763: CSL Vifor
VIT-2763 is an orally administered small molecule developed by Vifor Pharma. Intended for daily administration, VIT-2763 has the potential for treating diseases with impaired iron metabolism. Ferroportin is an iron transporter that plays a key role in regulating iron uptake and distribution in the body and thus in controlling iron levels in the blood. At the molecular level, VIT-2763 binds to ferroportin and blocks it to prevent excessive iron release into the blood. Pre-clinical evidence serving as the basis for the clinical development of VIT-2763 revolves around its efficacy for reducing elevated blood and tissue iron levels and for restricting iron uptake in patients suffering from conditions in which iron metabolism is altered.
Vamifeport is currently in phase II development for beta-thalassemia, an inherited rare blood disorder that reduces the production of functional haemoglobin in red blood cells, which can lead to a lack of oxygen in many parts of the body and potentially cause anaemia.
Emeramide: EmeraMed
Emeramide is an antioxidant heavy metal chelator. It prevents methylmercury-induced glutathione (GSH) loss, and cytotoxicity to, isolated mouse aortic endothelial cells when used at a concentration of 50 µM. Emeramide is a lipophilic, di-thiol antioxidant, anti-viral, and metal chelator. Orphan Drug Designations were received for the treatment of mercury toxicity in the EU and US. Pre-clinical safety studies, a Phase I trial, and four Phase II trials have been performed targeting multiple indications. Currently the drug is in phase II for the treatment of beta-thalassaemia.
Beta Thalassemia Pipeline Therapeutics Assessment
There are approx. 22+ key companies which are developing the therapies for Beta-thalassaemia. The companies which have their Beta-thalassaemia drug candidates in the most advanced stage, i.e. pre-registration include, CRISPR Therapeutics.
Find out more about the Beta Thalassemia Diagnosis and Treatment of patients @ Beta Thalassemia Ongoing Clinical Trials Analysis
Scope of the Beta Thalassemia Pipeline Report
Discover more about the list of FDA-approved drugs for Beta Thalassemia @ Beta Thalassemia Treatment Landscape
Table of Content
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