PUBLISHER: DelveInsight | PRODUCT CODE: 1160839
PUBLISHER: DelveInsight | PRODUCT CODE: 1160839
DelveInsight's , "Fanconi Anemia - Pipeline Insight, 2022," report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in Fanconi 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.
Fanconi anemia (FA) is a rare genetic disorder, involving all three blood cell lines. It is the most common cause of inherited bone marrow failure characterized by pancytopenia. Additionally, it affects almost all organs of the body. Fanconi anemia is mainly based upon the molecular mechanism involving a defective homologous recombination DNA repair pathway, defects in proteins as well as other enzymes involved in the repair of damaged DNA following various alkylating agents, irradiation, and cytotoxic drugs. It is also referred to as the inherited form of aplastic anemia. The discovery of Fanconi anemia had implications far beyond the disease itself. An extensive study of other bone marrow failure syndromes and chromosome fragility diseases has enhanced the scientific understanding of the bone marrow failure in Fanconi anemia. It is mostly associated with other congenital deformities and is susceptible to hematological and solid tumors. It usually is more common during childhood, with the average age of diagnosis being 7 years. The advancement of molecular genetic studies has helped in the comprehensive study of Fanconi anemia.
"Fanconi Anemia - Pipeline Insight, 2022" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Fanconi Anemia pipeline landscape is provided which includes the disease overview and Fanconi Anemia treatment guidelines. The assessment part of the report embraces, in depth Fanconi 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, Fanconi Anemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
This segment of the Fanconi 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.
Mozafancogene autotemcel (formerly RP L102) is a lentiviral vector-based gene therapy, developed by Rocket Pharmaceuticals for the treatment of Fanconi's. Currently, it is in Phase II stage of clinical trial evaluation.
FP-045 is a potent and highly selective aldehyde dehydrogenase (ALDH2) activator, highly soluble and orally available. ALDH2 is a key mitochondrial regulator of toxic aldehyde metabolism. FP-045 has successfully completed single and multiple ascending dose Phase 1 studies. In vitro, FP-045 increases ALDH2 activity in FANCA-deficient cells and protects FANCA-deficient cells from damage due to exposure to toxic reactive aldehyde such as 4-HNE. FP-045 also decreases TNF-α release from cultured FA lymphocytes. These data support that ALDH2 activity may be important in maintaining the development of blood cells in Fanconi anemia patients and as such may be a useful treatment for patients with incipient bone marrow failure.
This segment of the report provides insights about the different Fanconi Anemia drugs segregated based on following parameters that define the scope of the report, such as:
There are approx. 4+ key companies which are developing the therapies for Fanconi Anemia. The companies which have their Fanconi Anemia drug candidates in the most advanced stage, i.e. phase II include, Rocket Pharmaceuticals.
DelveInsight's report covers around 4+ products under different phases of clinical development like:
Fanconi 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:
Products have been categorized under various Molecule types such as:
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Fanconi Anemia therapeutic drugs key players involved in developing key drugs.
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Fanconi Anemia drugs.