SEL-313 for Ornithine Transcarbamylase Deficiency (OTC)
Selecta is developing a gene therapy product candidate to treat OTC, an inborn error of metabolism that affects one in 15,000-60,000 people worldwide. In patients with OTC, consumption of protein results in the accumulation of undesirable levels of ammonia in the blood, which can cause irreversible brain damage. Patients with the most severe form of the disease today require liver transplantation in early childhood and suffer intellectual disability, developmental delays and reduced life expectancy.
SEL-313 combines an adeno associated virus (AAV)-based gene therapy with Selecta’s SVP-Rapamycin (SEL-110). This combination is designed to enable repeated doses of the gene therapy treatment, which may be required when treating infants and young children with the disease in order to maintain the required expression levels of the missing OTC enzyme over the course of the patient’s lifetime. The treatment also is designed to prevent liver damage that can be associated with cellular immune reactions to gene therapy vectors such as AAV.
To advance the OTC program, Selecta is working with Genethon in Evry, France and the International Centre for Genetic Engineering and Biotechnology (ICGEB) in Trieste, Italy.