Synlogic Proprietary Synthetic Biotic Receives FDA Orphan Drug Designation for treatment of urea cycle disorders
Synlogic has got the U.S. Food and Drug Administration (FDA) orphan drug designation to it's program involving a live Escherichia coli Nissle bacterium modified to assimilate ammonia for treatment of urea cycle disorders. Synlogic is a company based in Cambridge, Massachusetts, pioneering the development of a novel class of therapeutics, called synthetic biotics, based on synthetic biology and microbiome
Some rare (estimated incidence of 1 in 8500 births http://www.nucdf.org/index.htm ) but serious mutations lead to accumulation of ammonia in the blood which causes severe neurological symptoms and damage. Depending on the severity of the enzimatic deficit these conditions could remain undiagnosed even until the adulthood when this disorder could be responsible of stroke-like symptoms, episodes of lethargy, and delirium (https://en.wikipedia.org/wiki/Urea_cycle_disorder).
Synlogic's SYNB1020, is currently in preclinical development and the company plans to file an investigational new drug application (IND) with the FDA for the potential treatment of UCDs in the first quarter of 2017.