Inherited Metabolic Disorders

Inherited metabolic disorders refer to different types of medical conditions caused by genetic defects that impact metabolism primarily within hepatic cells in the liver. There are hundreds of inherited metabolic disorders, caused by different genetic defects. These often lead to accumulation of substances which are toxic or interfere with normal cellular/organ function, or the reduced ability to synthesize essential compounds. These diseases can be life threatening or strongly debilitating for the people suffering from them, and although some of these diseases can be treated by diet or by enzyme replacement therapies, many of them remain a high unmet medical need, and often progress to organ failure, and death, with a strong impact on life expectancy.

In order to tackle such genetic metabolic diseases, Genespire builds on a novel lentiviral vector technology, the “Immune-Shielded LV” to develop off-the-shelf liver-directed in vivo gene therapies.