Fanconi’s anemia is an inherited genetic disorder that affects the bone marrow and results in the decreased production of all types of blood cells. The symptoms associated with the disease include decreased blood cell number, bone problems, vitiligo, deafness, eye problems, short height, small head, small testicles and genital changes. The disease can be diagnosed by bone marrow biopsy, complete blood count, hearing test, CT scan and MRI. The disease can be treated by growth factors, hormone therapy, antibiotics and blood transfusions. According to the data published by National Organization for Rare Disorders, the incidence rate of Fanconi’s anemia is estimated to be around 1 in 136,000 births. This condition is more common among people of Ashkenazi Jewish descent, the Roma population of Spain, and black South Africans. Abeona Therapeutics Inc. is in the process of developing ABO-301 for the treatment of Fanconi's anemia as a gene therapy. Some of the companies having a pipeline of Fanconi's anemia include Elixirgen Therapeutics, LLC, and Aldea Pharmaceuticals.
The report provides a comprehensive understanding of the pipeline activities covering all drug candidates under various stages of development, with detailed analysis of pipeline and clinical trials. Pipeline analysis of drugs by phases includes product description and development activities, including information about clinical results, designations, collaborations, licencing, grants, technology and others.