Seattle company immusoft announced the launch of the first clinical test, in which genetically engineered B cells capable of producing antibodies are used to treat a genetic disease. This experimental method aims to combat mucopolysaccharidosis of type I (MPS I) – a rare hereditary disorder in which an important enzyme that breaks down long-chain sugar in cells is not produced in the patient’s body. The lack of this enzyme leads to serious complications, including clouding of the eyes, breathing problems, cognitive impairment, and issues with the organs.
Traditionally, patients with MPS
Researchers carry out genetic modification of B cells using transposons – DNA sequences that are integrated into the cell of the cell. Unlike other forms of genetic engineering therapy, such as treatment using CRISPR or CAR-T cell therapy, the new Immusoft technique focuses on the use of B cells.
Sean Einsvort, CEO of Immusoft, stated that the first patient who received the experimental therapy in mid-November is feeling well. Unlike other methods of genetic therapy, the procedure for introducing modified B cells can be done on an outpatient basis.
The primary goal of the initial stage of testing is to assess the safety of the method. In the future, it is planned to eliminate the enzyme infusion to test how efficiently the modified B cells perform their task. Researchers also intend to study the longevity of the modified cells in the body.
Clinical tests are being conducted under the leadership of Dr. Paul Orchard from the Medical School of the University of Minnesota. Despite promising prospects, the FDA has expressed concerns about the potential risk of developing cancer, such as lymphoma or leukemia, due to the use of the transposon system for introducing genetic material.