BALTIMORE, May 30, 2017 – Elixirgen Therapeutics has successfully concluded its pre-IND meeting with the U.S. Food and Drug Administration (FDA) for inherited bone marrow failure syndromes (IBMFS), including Dyskeratosis Congenita (DC) and Fanconi Anemia (FA). IBMFS such as DC and FA are rare genetic disorders known for their aplastic anemia, which hinders blood cell production in the bone marrow. In DC, bone marrow failure is the primary cause of morbidity, affecting 80-90% of all cases by 30 years old. This program’s treatment paradigm uses Elixirgen Therapeutics’ proprietary ZSCAN4 technology to lengthen the telomeres of the patients. It is the lead indication for Elixirgen Therapeutics’ continued journey to bring ZSCAN4 technology to bear on diseases to improve quality of life through stem cell rejuvenation.
Elixirgen Therapeutics successfully concludes pre-IND meeting with FDA